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368 Study Matches
Study of Tinengotinib VS. Physician's Choice a Treatment of Subjects With FGFR-altered in Cholangiocarcinoma (FIRST-308)
This study is a Phase III, Randomized, Controlled, Global Multicenter Study to Evaluate the Efficacy and Safety of Oral Tinengotinib versus Physician's Choice in Subjects with Fibroblast Growth Factor Receptor (FGFR)-altered, Chemotherapy- and FGFR Inhibitor-Refractory/Relapsed Cholangiocarcinoma
Call 833-722-6237
canceranswerline@utsouthwestern.edu
David Hsieh
ALL
18 Years and over
PHASE3
NCT05948475
STU-2024-0123
Inclusion Criteria:
• ≥ 18 years of age at the time of signing the informed consent form (ICF).
• Histologically or cytologically confirmed CCA/adenocarcinoma of biliary origin with radiological evidence of unresectable or metastatic disease.
• Documentation of FGFR2 fusion/rearrangement gene status
• Subjects must have received at least one line of prior chemotherapy and exactly one FDA approved FGFR inhibitor.
Exclusion Criteria:
• Prior receipt of two or more FGFR inhibitors, either approved or investigational drugs.
• Subjects with known brain or central nervous system (CNS) metastases that have radiologically or clinically progressed in the 28 days prior to initiation of therapy. Subjects with asymptomatic brain/CNS metastases or treated brain/CNS metastases that have been clinically stable for 14 days on steroids without escalation of steroids are eligible for enrollment.
• Subjects with a known concurrent malignancy that is progressing or requires active treatment. Exceptions include basal cell carcinoma of the skin, carcinoma in situ of the cervix, or other noninvasive or indolent malignancy, including those that have previously undergone potentially curative therapy.
• Subjects who have received prior systemic therapy or investigational study drug ≤ 5 half-lives or 14 days, whichever is shorter, prior to starting the study drug or who have not recovered (grade ≤ 1 or at pretreatment baseline except tolerable grade 2 alopecia, fatigue/asthenia, and neuropathy due to trauma) from adverse events (AEs) of prior therapy.
• Concurrent anticancer therapy including chemo-, immune-, or radiotherapy. Hormone therapy may be allowed with Sponsor approval.
• Subjects who have received wide field radiotherapy ≤ 4 weeks or limited field radiation for palliation ≤ 2 weeks prior to starting the study drug or who have not recovered from AEs of prior therapy.
• Subjects with uncontrolled hypertension (defined as blood pressure of ≥ 150 mm Hg systolic and/or ≥ 90 mm Hg diastolic despite adequate treatment with antihypertensive medications at screening)
DRUG: Tinengotinib 8 mg, DRUG: Tinengotinib 10 mg, DRUG: Physician's Choice
Cholangiocarcinoma, Other Digestive Organ
Refractory/Relapsed Cholangiocarcinoma
UT Southwestern; Parkland Health & Hospital System
Sequential Treatment of Cabozantinib for Advanced Renal Cell Carcinoma (RCC)
The goal of this clinical trial is to learn about the effects of a higher dose of ncabozantinib in patients with advanced renal cell carcinoma who have progressed on or after receiving cabozantinib treatment.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Tian Zhang
ALL
18 Years and over
PHASE2
NCT05931393
STU-2023-0439
Inclusion Criteria:
• Patients with advanced RCC (defined as locally advanced unresectable or metastatic) of any histology who progressed on/after cabozantinib monotherapy in any line of treatment. Patient must have cabozantinib sensitive disease (prior treatment with cabozantinib ≥ 6 months)
• Ability to tolerate prior cabozantinib at 60mg PO daily with manageable toxicity profile at the respective doses, at investigator discretion
• Prior PD-1 inhibitor/PD-L1 inhibitor allowed
• Evidence of measurable disease per RECIST 1.1
• For up to 5 patients opting into on-treatment biopsy, one of the following must be met:
• Archival tissue confirmed to be available and obtained within 30 days of informed consent as well as willingness to undergo an on-treatment biopsy at 12 weeks (+/- 7 days). OR
• Willingness to undergo a baseline biopsy prior to Cycle 1 Day1, as well as an on-treatment biopsy at 12 weeks (+/- 7 days).
• Age ≥ 18 at time of consent
• ECOG performance status ≤ 2
• Capable of understanding and complying with the protocol requirements and must have signed the informed consent document
• No washout period is needed for cabozantinib, minimum of 4 weeks or 4 half-lives washout, whichever is shorter, for other standard or experimental anti-cancer therapies.
• Recovery to baseline or ≤ Grade 1 National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 5 from toxicities related to any prior treatments, unless AE(s) are clinically nonsignificant and/or stable on supportive therapy
• Adequate organ and marrow function, based upon meeting all of the following laboratory criteria within 14 days before first dose of study treatment:
• Absolute neutrophil count (ANC) ≥ 1500/µL without granulocyte colony-stimulating factor (G-CSF) support
• White blood cell (WBC) count ≥ 2500/µL
• Platelets ≥ 100,000/µL without transfusion
• Hemoglobin ≥ 9 g/dL (≥ 90 g/L) (transfusion acceptable per investigator discretion)
• Alanine transaminase (ALT), AST and alkaline phosphatase (ALP) ≤ 3 x ULN. ALP ≤ 5x ULN with documented bone metastases
• Total bilirubin ≤ 1.5 x ULN (for subjects with Gilbert's disease ≤ 3x ULN)
• Serum albumin ≥ 2.8 g/dl
• Prothrombin (PT)/international normalized ratio (INR) or partial thromboplastin time (PTT) test \< 1.3x the laboratory ULN
• Serum creatinine ≤ 1.5x ULN or calculated creatinine clearance ≥ 40mL/min (≥ 0.675mL/sec) using the Cockcroft-Gault equation: * Males: (140 - age) x weight (kg)/(serum creatinine \[mg/dL\] × 72) * Females: \[(140 - age) x weight (kg)/(serum creatinine \[mg/dL\] × 72)\] × 0.85
• Urine protein/creatinine ratio (UPCR) ≤1 mg/mg (≤113.2 mg/mmol), or 24h urine protein ≤1 g
• Sexually active fertile subjects and their partners must agree to use medically accepted methods of contraception (e.g., barrier methods, including male condom, female condom, or diaphragm with spermicidal gel) during the course of the study and for 4 months after the last dose of cabozantinib Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
• Female subjects are considered to be of childbearing potential unless one of the following criteria is met: * documented permanent sterilization (hysterectomy, bilateral salpingectomy, or bilateral oophorectomy), or * documented postmenopausal status (defined as 12 months of amenorrhea in a woman \> 45 years-of-age in the absence of other biological or physiological causes.
• In addition, females \< 55 years-of-age must have a serum follicle stimulating hormone (FSH) level \> 40 mIU/mL to confirm menopause.
Exclusion Criteria:
• Radiation therapy for bone metastasis within 2 weeks or any other radiation therapy within 4 weeks before first dose of study treatment. Systemic treatment with radionuclides within 6 weeks before first dose of study treatment. Subjects with clinically relevant ongoing complications from prior radiation therapy are not eligible
• Known brain metastases or cranial epidural disease unless adequately treated with radiotherapy and/or surgery (including radiosurgery) and stable for 1) at least 2 weeks after radiotherapy or 2) at least 4 weeks after major surgery (e.g., removal or biopsy of brain metastasis) prior to first dose of study treatment. Subjects must have complete wound healing from major surgery or minor surgery before first dose of study treatment. Eligible subjects must be neurologically asymptomatic and without corticosteroid treatment for the brain metastasis at the time of first dose of study treatment
• Concomitant anticoagulation with coumarin agents (e.g., warfarin), direct thrombin inhibitors (e.g., dabigatran), direct factor Xa inhibitor betrixaban, or platelet inhibitors (e.g., clopidogrel). Allowed anticoagulants are the following: 1) prophylactic use of low-dose aspirin for cardio-protection (per local applicable guidelines) and low-dose low molecular weight heparins (LMWH). 2) Therapeutic doses of LMWH or anticoagulation with direct factor Xa inhibitors rivaroxaban, edoxaban, or apixaban in subjects without known brain metastases who are on a stable dose of the anticoagulant for at least 1 week before first dose of study treatment without clinically significant hemorrhagic complications from the anticoagulation regimen or the tumor
• The subject has uncontrolled, significant intercurrent or recent illness including, but not limited to, the following conditions:
• Cardiovascular disorders: 1) congestive heart failure New York Heart Association Class 3 or 4, unstable angina pectoris, serious cardiac arrhythmias; 2) uncontrolled hypertension defined as sustained blood pressure (BP) \> 140 mm Hg systolic or \> 90 mm Hg diastolic despite optimal antihypertensive treatment within 1 week of treatment; 3) stroke (including transient ischemic attack \[TIA\]), myocardial infarction (MI), or other ischemic event, or thromboembolic event (e.g., deep venous thrombosis \[DVT\], pulmonary embolism \[PE\]) within 6 months before first dose of study treatment. Note: subjects with a diagnosis of incidental, subsegmental PE or DVT within 6 months are allowed if stable, asymptomatic, and treated with a stable dose of permitted anticoagulation (see exclusion criterion #3.2.4) for at least 1 week before first dose of study treatment
• Gastrointestinal (GI) disorders including those associated with a high risk of perforation or fistula formation, including 1) the subject has evidence of tumor invading the GI tract, active peptic ulcer disease, inflammatory bowel disease (e.g., Crohn's disease), diverticulitis, cholecystitis, symptomatic cholangitis or appendicitis, acute pancreatitis, acute obstruction of the pancreatic duct or common bile duct, or gastric outlet obstruction; 2) abdominal fistula, GI perforation, bowel obstruction, or intra-abdominal abscess within 6 months before first dose of study treatment. Note: Complete healing of an intra-abdominal abscess must be confirmed before first dose of study treatment
• Clinically significant hematuria, hematemesis, hemoptysis, or other history of significant bleeding (e.g., pulmonary hemorrhage) within 6 weeks before first dose of study treatment. (Clinically significant hematuria defined by needing transfusion; clinically significant hematemesis or hemoptysis defined by needing hospital admission)
• Cavitating pulmonary lesion(s) or known endotracheal or endobronchial disease manifestation. Cavitary pulmonary lesions are allowed if not symptomatic.
• Lesions invading or encasing any major blood vessels
• Other clinically significant disorders that would preclude safe study participation
• Serious non-healing wound/ulcer/bone fracture
• Moderate to severe hepatic impairment (Child-Pugh B or C).
• Acute COVID-19 infection - clinical recovery from COVID-19 infection at least 14 days prior to enrollment allowed.
• Major surgery (e.g., laparoscopic nephrectomy, GI surgery, removal or biopsy of brain metastasis) within 2 weeks before first dose of study treatment. Minor surgeries within 10 days before first dose of study treatment. Subjects must have complete wound healing from major surgery or minor surgery before first dose of study treatment. Subjects with clinically relevant ongoing complications from prior surgery are not eligible
• Corrected QT interval calculated by the Fridericia formula (QTcF) \> 500 ms per electrocardiogram (ECG) within 14 days before first dose of study treatment. Furthermore, subjects with a history of additional risk factors for torsades de pointes (e.g., long QT syndrome) are also excluded. Note: If a single ECG shows a QTcF with an absolute value \> 500 ms, two additional ECGs at intervals of approximately 3 min must be performed within 30 min after the initial ECG, and the average of these three consecutive results for QTcF will be used to determine eligibility.
• Pregnant or lactating females
• Inability to swallow tablets
• Previously identified allergy or hypersensitivity to components of the study treatment formulations or history of severe infusion-related reactions to monoclonal antibodies. Subjects with rare hereditary problems of galactose intolerance, the Lapp lactase deficiency or glucose-galactose malabsorption are also excluded
• Another malignancy within 2 years prior to first dose of study treatment that requires active treatment, except for locally curable cancers that have been apparently cured, such as basal or squamous cell skin cancer, superficial bladder cancer, Gleason 6 prostate cancer, or carcinoma in situ of cervix or breast
DRUG: Cabozantinib 80 MG
RCC, Renal Cell Carcinoma, Kidney
UT Southwestern
A Study of AAV2-hAQP1 Gene Therapy in Participants With Radiation-Induced Late Xerostomia (AQUAx2)
This study will assess the efficacy and safety of bilateral intra-parotid administration of AAV2-hAQP1 in adults with Grade 2 or Grade 3 radiation-induced late xerostomia.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Thomas Schlieve
ALL
18 Years and over
PHASE2
NCT05926765
STU-2023-0369
Inclusion Criteria:
* Completed beam radiation therapy for head and neck cancer at least 3 years prior to the first screening visit
* No history of recurrent head and neck cancer, parotid gland cancer, or a second primary cancer, except for treated basal cell or squamous cell carcinoma of the skin or in situ cervical carcinoma
* An unstimulated whole saliva flow rate (mL/min) \>0 (i.e., at least one drop of saliva in the collection tube)
* A stimulated whole saliva flow rate (mL/min) within a specified range after mechanical stimulation by chewing
* Average screening XQ Total Score at or above a specified threshold
* No evidence of head and neck cancer, defined as a negative otolaryngology exam and a negative computed tomography (CT) scan of the head, neck, and chest with contrast. If a participant has had a magnetic resonance imaging (MRI) study, CT scan, positron emission tomography (PET), or fluorodeoxyglucose-positron emission tomography (FDG-PET) scan of the head, neck, and chest within 6 months of signing the informed consent form (and at least 3 years after the completion of radiotherapy), then that scan may be used for eligibility determination and a CT scan at screening will not be required. If the CT of the neck captures images from the forehead down to the neck, no CT of the head is required.
* Either received treatment with one or more prescription sialagogues and elected to discontinue therapy or, in consultation with their physician, elected not to initiate such treatment
* Participants taking a prescription sialagogue (specifically, pilocarpine or cevimeline) must stop that medication at least 2 weeks prior to Screening and be willing to refrain from taking such medications for the duration of the study
* Participants who require medication for an underlying medical condition that is known to affect salivary output must be on stable doses of such medications for at least one month prior to the first screening visit
Exclusion Criteria:
* History of recurrent head and neck cancer, parotid gland cancer, or a second primary cancer, except for treated basal cell or squamous cell carcinoma of the skin or in situ cervical carcinoma
* History of systemic autoimmune disease affecting the salivary glands (e.g., Sjogren's disease)
* Currently using systemic immunosuppressive medication(s) (e.g., corticosteroids or biologics) or treated with one within 4 weeks of the first screening visit. Note: Topical, inhaled, or intranasal corticosteroids are permitted.
* Active viral infection with Epstein-Barr virus (EBV), defined as a positive anti-VCA IgM. In the event a potential participant has a positive anti-VCA IgM, they may be rescreened 2-4 months later at which time a positive Epstein-Barr Virus Nuclear Antigen (EBNA) will be considered as evidence of resolved EBV infection.
* Evidence of active Hepatitis C virus (HCV) infection
* Evidence of human immunodeficiency virus (HIV) infection
* Diagnosis of myasthenia gravis
* Personal or family history of acute or chronic angle-closure glaucoma (ACG), or at increased risk of developing ACG, or had prophylactic treatment to reduce the risk of developing ACG
* Known allergy or hypersensitivity to glycopyrrolate
* Current smokers or history of smoking within the preceding 3 years (includes vaping with tobacco additives)
* Current alcohol misuse or a history of the same within the preceding 3 years, as defined by local guidance. In North America, an average intake for men of more than 14 drinks per week, and for women more than 7 drinks per week, consistent with the US National Institute of Alcohol Abuse and Alcoholism. In the UK, an average intake of more than 14 units per week for both men and women, consistent with the UK Chief Medical Officers' Low Risk Drinking Guidelines.
* Poorly controlled diabetes (hemoglobin A1c \>7%) GENETIC: AAV2-hAQP1 Concentration 1, GENETIC: AAV2-hAQP1 Concentration 2, OTHER: Placebo, GENETIC: AAV2-hAQP1 Concentration 3, GENETIC: AAV2-hAQP1 Concentration 4
Grade 2 and 3 Late Xerostomia Caused by Radiotherapy for Cancers of the Upper Aerodigestive Tract, Excluding the Parotid Glands, Lip, Oral Cavity and Pharynx
UT Southwestern
Fontan Udenafil Exercise Longitudinal Assessment Trial - 2 (FUEL-2)
This study will evaluate the clinical efficacy and safety of udenafil, an orally administered, potent and selective inhibitor of PDE5, versus placebo for the treatment of adolescent who have had the Fontan procedure.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Ana.Garzon@childrens.com
Matthew Lemler
ALL
12 Years to 18 Years old
PHASE3
NCT05918211
STU-2024-1065
Inclusion Criteria:
• Males and females with Fontan physiology who are 12 to less than 19 years of age at enrollment.
• Participant consent or parental/guardian consent and participant assent.
• Participant fluency in primary language of country in which study is being conducted.
• Current antiplatelet or anticoagulant therapy.
Exclusion Criteria:
• Height \< 132 cm.
• Weight \< 40 kg.
• Hospitalization for acute decompensated heart failure within the last 12 months.
• Current intravenous inotropic drugs.
• Undergoing evaluation for heart transplantation or listed for transplantation.
• Diagnosis of active protein losing enteropathy or plastic bronchitis within the last 3 years, or a history of liver cirrhosis.
• Known Fontan baffle obstruction, branch pulmonary artery stenosis, or pulmonary vein stenosis resulting in a mean gradient of \> 4 mmHg between the regions proximal and distal to the obstruction as measured by either catheterization or echocardiography, obtained prior to screening for the trial.
• Single lung physiology with greater than 80% flow to one lung.
• Failure to achieve maximal exertion (defined as RER \< 1.10) on screening/baseline exercise test.
• Peak minute oxygen consumption (VO2) less than 45% or ≥ 80% of predicated for age and gender at enrollment.
• Severe ventricular dysfunction assessed qualitatively by clinical echocardiography within 6 months prior to enrollment.
• Severe valvar regurgitation, ventricular outflow obstruction, or severe aortic arch obstruction assessed by clinical echocardiography within six months prior to enrollment.
• History of significant renal (serum creatinine \> 2.0), hepatic (serum AST and/or ALT \> 3 times upper limit of normal), gastrointestinal or biliary disorders that could impair absorption, metabolism or excretion of orally administered medications.
• Inability to complete exercise testing at baseline screening.
• Subjects with a pacemaker whose heart rate at peak exercise is controlled by the extrinsic pacemaker as opposed to a native atrial rhythm.
• History of PDE-5 inhibitor use within 12 months prior to enrollment. (Treatment is defined as chronic therapy as opposed to a single dose.)
• History of any other medication for treatment of pulmonary hypertension within 3 months before study onset.
• Known intolerance to oral udenafil.
• Frequent use of medications or other substances that inhibit or induce CYP3A4.
• Current use of alpha-blockers or nitrates.
• Ongoing or planned participation in another research protocol that would either prevent successful completion of planned study testing or invalidate its results.
• Noncardiac medical, psychiatric, and/or social disorder that would prevent successful completion of planned study testing or would invalidate its results.
• Cardiac care, ongoing or planned, at a non-study center that would impede study completion.
• For females: Pregnancy at the time of screening, pregnancy planned before study completion, or refusal to use an acceptable method of contraception for study duration if sexually active.
• Unable to abstain or limit intake of grapefruit juice and grapefruit containing drinks during the duration of the trial.
• Refusal to provide written informed consent/assent.
• In the opinion of the investigator, the subject is likely to be non-compliant with the study protocol.
• History of clinically significant thromboembolic event, in the option of the site Principal Investigator, that may put the subject at increased risk of a subsequent event while participating in the study.
• Coronavirus disease 2019 (COVID-19) vaccination or symptoms of COVID-19 infection within 7 days of Visit 1.
• Not taking antiplatelet or anticoagulant therapy.
DRUG: Udenafil, DRUG: Placebo
Single Ventricle Heart Disease
Fontan, Maximal Oxygen Consumption, Work Rate, VO2 at VAT, Ventilatory Efficiency
Children’s Health
Disitamab Vedotin With Pembrolizumab vs Chemotherapy in Previously Untreated Urothelial Cancer Expressing HER2
This study will enroll participants with urothelial cancer (UC). UC can include cancer of the bladder, kidney, or the tubes that carry pee through the body (ureter, urethra). This study will try to find out if the drugs disitamab vedotin with pembrolizumab works better than platinum-containing chemotherapy to treat patients with UC. This study will also test what side effects happen when participants take these drugs together. A side effect is anything a drug does to the body besides treating the disease. Participants in this study will have cancer that has spread through the body (metastatic) or spread near where it started (locally advanced). In this study, there are 2 different groups. Participants will be assigned to a group randomly. Participants in the disitamab vedotin arm will get the study drug disitamab vedotin once every two weeks and pembrolizumab once every 6 weeks. Participants in the standard of care arm will get gemcitabine once a week for 2 weeks with either cisplatin or carboplatin once every 3 weeks.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Waddah Arafat
ALL
18 Years to old
PHASE3
NCT05911295
STU-2023-0936
Inclusion Criteria:
* Histopathological confirmation of locally advanced unresectable or metastatic urothelial carcinoma (LA/mUC), including UC originating from the renal pelvis, ureters, bladder, or urethra.
* Measurable disease by investigator assessment per RECIST v1.1.
* Participant must not have received prior systemic therapy for LA/mUC. Exception will be made for neoadjuvant or adjuvant therapy, if disease recurrence/progression occurred more than 12 months after the last dose of therapy.
* Eligible to receive cisplatin- or carboplatin-containing chemotherapy.
* Able to provide archived formalin-fixed paraffin-embedded tumor tissue blocks from a muscle-invasive or metastatic UC lesion or biopsy of metastatic UC prior to treatment initiation. If archival tissue is not available a newly obtained baseline biopsy of an accessible tumor lesion is required within 28 days of cycle 1 day 1.
* HER2 expression of 1+ or greater on immunohistochemistry (IHC).
* Eastern Cooperative Oncology Group (ECOG) performance score of 0, 1, or 2 within 7 days prior to randomization.
Exclusion Criteria:
* Known hypersensitivity to disitamab vedotin, cisplatin, carboplatin, gemcitabine, or pembrolizumab or any of their components.
* History of severe/life threatening immune-related adverse event (irAE) with PD-(L)1 inhibitors are excluded.
* Central nervous system (CNS) and/or leptomeningeal metastasis. Participants with treated CNS metastases are permitted if all of the following are met.
* CNS metastases have been clinically stable for at least 4 weeks and baseline scans show no evidence of new or worsening CNS metastasis.
* Participant is on a stable dose of ≤ 10 mg/day of prednisone or equivalent for at least 2 weeks.
* History of or active autoimmune disease that has required systemic treatment in the past 2 years.
* Prior treatment with an agent directed to another stimulatory or co-inhibitory T cell receptor (including but not limited to CD137 agonists, CAR-T cell therapy, CTLA-4 inhibitors, or OX-40 agonists).
* Prior solid organ or bone marrow transplantation.
* Pleural effusion or ascites with symptoms or requiring symptomatic treatment.
* Estimated life expectancy \<12 week
* Prior treatment with an MMAE agent or anti-HER2 therapy DRUG: disitamab vedotin, DRUG: pembrolizumab, DRUG: gemcitabine, DRUG: cisplatin, DRUG: carboplatin
Urothelial Carcinoma, Urinary Bladder
Urothelial Cancer, LA/mUC, Bladder Cancer, HER2 Overexpression, HER2 Amplification, HER2, Seattle Genetics
UT Southwestern
Nuwiq for Perioperative Management Of Patients With Haemophilia A on Emicizumab Regular Prophylaxis Study (NuPOWER)
Recombinant factor VIII for the prevention of bleeding in patients with severe haemophilia A undergoing major surgery while receiving emicizumab prophylaxis
Call 214-648-5005
studyfinder@utsouthwestern.edu, susan.corley@childrens.com
Jessica Garcia
MALE
12 Years to old
PHASE4
NCT05935358
STU-2023-0793
Inclusion Criteria:
* Severe haemophilia A (FVIII activity \[FVIII:C\] \<1%) according to medical history
* Male patients at least 12 years of age
* Previous treatment with any FVIII product(s) for at least 150 exposure days
* On regular prophylaxis with emicizumab for at least 1 month prior to a scheduled major elective surgery requiring FVIII treatment
* Freely given written informed consent of the patient, or parent/legal representative where applicable, obtained in accordance with local regulations
Exclusion Criteria:
* Coagulation disorder other than haemophilia A
* Present or past FVIII inhibitor (≥0.6 Bethesda units \[BU\]/mL) according to medical history
* Severe liver or kidney disease (alanine aminotransferase \[ALT\] and/or aspartate aminotransferase \[AST\] levels \>5 times the upper limit of normal; or creatinine \>120 μmol/L)
* Known hypersensitivity to Nuwiq's active substance or its excipients (sucrose, sodium chloride, calcium chloride dihydrate, arginine hydrochloride, sodium citrate dihydrate, poloxamer 188)
* Already had surgery in this study
* Current participation in another interventional clinical trial
* Treatment with any investigational medicinal product (IMP) within 30 days prior to screening visit DRUG: Nuwiq
Severe Hemophilia A, Other Hematopoietic
Children’s Health
Mitoquinone/mitoquinol Mesylate As Oral and Safe Postexposure Prophylaxis for Covid-19
Adults who do not have major health, kidney, gastrointestinal disease will be randomized to receive oral mitoquinone/mitoquinol mesylate (Mito-MES) versus placebo to prevent the development and progression of COVID-19 after high-risk exposure to a person with confirmed SARS-CoV-2 infection.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Theodoros.Kelesidis@UTSouthwestern.edu
Theodoros Kelesidis
ALL
18 Years to 65 Years old
PHASE2
NCT05886816
STU-2023-0524
Inclusion Criteria:
Age 18-65 years old Asymptomatic (no symptoms of viral infection) on study entry High risk exposure without use of masks to confirmed case of COVID-19 Members in a household one of which is a confirmed case of COVID-19 Negative baseline SARS-COV-2 diagnostic test
Exclusion Criteria:
* Women with variations in physiological functions due to hormones that may effect immune function and (transgender, pregnant, breastfeeding)
* Specific significant clinical diseases \[cardiovascular disease (such as coronary artery/vascular disease), heart disease (such as congestive heart failure, cardiomyopathy, atrial fibrillation), lung disease (such as chronic obstructive pulmonary disease, asthma, bronchiectasis, pulmonary fibrosis, pleural effusions), kidney disease (glomerular filtration rate or GFR less than 60 ml/min/1.73 m2), liver disease (such as cirrhosis, hepatitis), major immunosuppression (such as history of transplantation, uncontrolled HIV infection, cancer on active chemotherapy\] based on history. Participants with well controlled HIV (CD4 count \> 500 cells/mm\^3 and HIV viral load \< 50 copies/ml) and people with remote history of cancer not on active treatment will be allowed to participate.
* History of known gastrointestinal disease (such as gastroparesis) that may predispose patients to nausea
* History of auto-immune diseases
* Chronic viral hepatitis
* Use of systemic immunomodulatory medications (e.g. steroids) within 4 weeks of enrollment
* Any participant who has received any investigational drug within 30 days of dosing
* History of underlying cardiac arrhythmia
* History of severe recent cardiac or pulmonary event
* A history of a hypersensitivity reaction to any components of the study drug or structurally similar compounds including Coenzyme Q10 and idebenone
* Unable to swallow tablets
* Use of any investigational products within 4 weeks of enrollment
* Any other clinical condition or prior therapy that, in the opinion of the investigator, would make the patient unsuitable for the study or unable to comply with the study requirements.
* Eligible for other FDA approved treatment for post-exposure prophylaxis against SARS-CoV-2
* Use of Coenzyme Q10 or Vitamin E \< 120 days from enrollment DRUG: Mitoquinone/mitoquinol mesylate, OTHER: Placebo
SARS-CoV Infection, COVID-19, Lung/Thoracic, Nose
SARS-CoV Infection, COVID-19, Post-exposure prophylaxis
UT Southwestern; Parkland Health & Hospital System
Clinical Study of Ivonescimab for First-line Treatment of Metastatic NSCLC Patients
This is a Phase 3 Randomized, double-blind, Multiregional Study of Ivonescimab Combined with Chemotherapy Versus Pembrolizumab Combined with Chemotherapy for the First-line Treatment of Metastatic Non-small Cell Lung Cancer. The primary endpoint is overall survival and progression free survival assessed by investigator. The key secondary endpoints include response and safety.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Mitchell von Itzstein
ALL
18 Years to old
PHASE3
NCT05899608
STU20251014
Inclusion Criteria:
* Age ≥ 18 years old at the time of enrollment
* Eastern Cooperative Oncology Group (ECOG) performance status score of 0 or 1
* Expected life expectancy ≥ 3 months
* Metastatic (Stage IV) NSCLC
* Histologically or cytologically confirmed squamous or non-squamous NSCLC
* Recorded measurement of the Tumor Proportion Score (TPS) or Tumor Cells (TC) for PD-L1 expression, irrespective of the PD-L1 expression, prior to randomization
* At least one measurable noncerebral lesion according to RECIST 1.1
* No prior systemic treatment for metastatic NSCLC
Exclusion Criteria:
* Histologic or cytopathologic evidence of the presence of small cell lung carcinoma
* Known actionable genomic alterations (EGFR, ALK, ROS1, and BRAF V600E) or genes for which first-line approved therapies are available.
* For non-squamous histology patients, actionable driver mutation testing results are required before randomization.
* Has received any prior therapy for NSCLC in the metastatic setting
* Tumor invasion, encasement of organs (e.g. pericardium, heart, trachea, esophagus, central bronchi), or major blood vessels (e.g aorta, central veins), if poses a significant increased risk of bleeding. BIOLOGICAL: Ivonescimab Injection, BIOLOGICAL: Pembrolizumab Injection
Non-Small Cell Lung Cancer, Lung/Thoracic
UT Southwestern; Parkland Health & Hospital System
Testing the Anti-cancer Drug Erdafitinib for Brain Cancers That Have Returned or Progressed Following Treatment
This phase II trial tests how well erdafitinib works in controlling IDH-wild type (WT), FGFR-TACC gene fusion positive gliomas that have come back after a period of improvement (recurrent) or that are growing, spreading, or getting worse (progressive). Erdafitinib is in a class of medications called kinase inhibitors. It works by blocking the action of an abnormal FGFR protein that signals tumor cells to multiply. This may help keep tumor cells from growing and may kill them. Giving erdafitinib may help to slow the growth of, or to shrink, tumor cells in patients with recurrent or progressive IDH-wild type gliomas with FGFR-TACC gene fusion.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Michael Youssef
ALL
18 Years to old
PHASE2
NCT05859334
STU20250708
Inclusion Criteria:
* Patient must be \>= 18 years of age
* Patient must have histologically confirmed IDH-WT gliomas (grade 2-4) as per World Health Organization (WHO) 2016 or 2021 classification
* Tumor tissue should be positive for FGFR-TACC gene fusion as per any local next generation sequencing (NGS) (Clinical Laboratory Improvement Act \[CLIA\]-approved) assay described in background section
* The disease should be recurrent or progressive glioma after initial anti-tumor treatment with at least 1 line of treatment including surgical resection, radiation therapy and/or chemotherapy
* For patients with WHO grade 3 or 4 glioma and progressive disease \< 12 weeks after completion of chemoradiotherapy, progression can be defined by the following set of criteria:
* New enhancement outside of the radiation field (beyond the high-dose region or 80% isodose line)
* If there is unequivocal evidence of viable tumor on histopathologic sampling (e.g., solid tumor areas. i.e., \> 70% tumor cell nuclei in areas), high or progressive increase in Ki-67 proliferation index compared with prior biopsy, or evidence for histologic progression or increased anaplasia in tumor)
* For patients with WHO grade 3 or 4 glioma and progressive disease \>= 12 weeks after completion of chemoradiotherapy, progression can be defined by the following set of criteria:
* New contrast-enhancing lesion outside of radiation field on decreasing, stable, or increasing doses of corticosteroids
* Increase by \>= 25% in the sum of the products of perpendicular diameters between the first post-radiotherapy scan, or a subsequent scan with smaller tumor size, and the scan at 12 weeks or later on stable or increasing doses of corticosteroids
* For patients receiving antiangiogenic therapy, significant increase in T2/fluid attenuated inversion recovery (FLAIR) non-enhancing lesion may also be considered progressive disease. The increased T2/FLAIR must have occurred with the patient on stable or increasing doses of corticosteroids compared with baseline scan or best response after initiation of therapy and not be a result of comorbid events (e.g., effects of radiation therapy, demyelination, ischemic injury, infection, seizures, postoperative changes, or other treatment effects)
* For patients with WHO grade 2 glioma progression is defined by any one of the following:
* Development of new lesions or increase of enhancement (radiological evidence of malignant transformation)
* A 25% increase of the T2 or FLAIR non-enhancing lesion on stable or increasing doses of corticosteroids compared with baseline scan or best response after initiation of therapy, not attributable to radiation effect or to comorbid events
* There must be measurable disease (enhancing or non-enhancing as per Response Assessment in Neuro-Oncology \[RANO\] or RANO-low-grade glioma \[LGG\] criteria), as evaluated on pre-treatment MRI
* Patient understands the procedures and investigational nature of the study drug and agrees to comply with study requirements by providing written informed consent
* Patient must have Eastern Cooperative Oncology Group (ECOG) performance status =\< 2 (or Karnofsky \>= 60%)
* Absolute neutrophil count \>= 1000/uL
* Hemoglobin \> 8 g/dL (Patients are allowed to be transfused to this level)
* Platelets \>= 100 x 10\^9/L
* Serum total bilirubin =\< 1.5 x upper limit of normal (ULN), unless considered due to Gilbert's disease or disease involvement following approval by the medical monitor
* Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT) (serum glutamic pyruvic transaminase \[SGPT\]) =\< 3 x institutional ULN
* Creatinine clearance \> 30 mL/min (patients with mild or moderate renal impairment) based on the Cockroft-Gault glomerular filtration rate (GFR) estimation
* Patient must have normal serum phosphate level as per local laboratory parameters. (Medical management allowed)
* Patient must be recovered from any clinically relevant toxic effects of any prior surgery, radiotherapy, or chemotherapy treatment with temozolomide
* Human immunodeficiency virus (HIV)-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial
* For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated
* Patients with a history of hepatitis C virus (HCV) infection must have been treated and cured. For patients with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load
* Patients should be New York Heart Association Functional Classification class 2B or better
* The effects of erdafitinib on the developing human fetus are unknown. For this reason and because FGFR inhibitors are known to be teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation, and one month after completion of erdafitinib administration. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and one month after completion of erdafitinib administration
* Ability to understand and the willingness to sign a written informed consent document. Legally authorized representatives may sign and give informed consent on behalf of study participants
Exclusion Criteria:
* Patients who are receiving any other investigational agents
* History of allergic reactions attributed to compounds of similar chemical or biologic composition to erdafitinib
* Patients requiring any medications or substances that are moderate CYP2C9 inducers and strong CYP3A4 inducers are ineligible. Because the lists of these agents are constantly changing, it is important to regularly consult a frequently-updated medical reference. As part of the enrollment/informed consent procedures, the patient will be counseled on the risk of interactions with other agents, and what to do if new medications need to be prescribed or if the patient is considering a new over-the-counter medicine or herbal product
* Patients with uncontrolled intercurrent illness
* Pregnant women are excluded from this study because erdafitinib is an FGFR inhibitor agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with erdafitinib, breastfeeding should be discontinued if the mother is treated with erdafitinib
* Current central serous retinopathy (CSR) or retinal pigment epithelial detachment of any grade
* Corrected QT interval (QTc) prolongation as confirmed by electrocardiography (ECG) at screening (Fridericia; QTc \> 480 milliseconds)
* Patients who have previously received FGFR inhibitors PROCEDURE: Biospecimen Collection, DRUG: Erdafitinib, PROCEDURE: Magnetic Resonance Imaging, PROCEDURE: Optical Coherence Tomography
Recurrent Glioma, Recurrent WHO Grade 2 Glioma, Recurrent WHO Grade 3 Glioma, Recurrent WHO Grade 4 Glioma, Brain and Nervous System
UT Southwestern
A Safety and Efficacy Study of HCB101, Fc-fusion Protein Targeting SIRPα-CD47 Pathway, in Solid or Hematological Tumors
The purpose of this study is to find out whether IV injection of HCB101 is an effective treatment for different types of advanced solid tumors or relapsed and refractory non-Hodgkin lymphoma and what side effects (unwanted effects) may occur in subjects aged 18 years old and above.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Tian Zhang
ALL
18 Years and over
PHASE1
NCT05892718
STU-2023-1031
Inclusion Criteria:
• Able to understand and willing to sign the ICF.
• Male and female subjects of ≥18 years of age.
• Histologically/cytologically confirmed, locally advanced solid tumor: subjects with histologically or cytologically confirmed advanced solid tumors refractory to standard therapy, or for which no standard treatment exists or non-Hodgkin lymphoma, relapsed or refractory to at least 2 prior lines of therapy.
• For subjects with advanced solid tumor - must have at least 1 measurable lesion as defined by Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 at baseline.
• For subjects with non-Hodgkin lymphoma - must have non-Hodgkin lymphoma that is measurable or assessable for response per Lugano Classification (with 2016 refinement).
• Must have ECOG performance status of 0 to 2 at Screening.
• Able to provide tumor tissue samples.
• Have life expectancy of ≥12 weeks.
Exclusion Criteria:
• With known history of hypersensitivity to any components of HCB101.
• Known active or untreated CNS metastases and/or carcinomatous meningitis.
• Have undergone a major surgery or radical radiotherapy or palliative radiotherapy or have used a radioactive drug that is not completed at least 2 weeks prior to the first dose of HCB101.
• Clinically significant cardiovascular condition.
• Any previous treatment-related toxicities which have not recovered to ≤ Grade 1 as evaluated by National Cancer Institute, Common Terminology Criteria for Adverse Events (NCI CTCAE) version 5.0 or baseline, except alopecia and anemia.
• With known inherited or acquired bleeding disorder or bleeding diathesis. .
• Have RBC transfusion within 4 weeks prior to Screening.
• With a previously documented diagnosis of hemolytic anemia or Evans Syndrome in the last 3 months.
• Any investigational or approved systemic cancer therapy.
• Active use of vitamin K antagonist anticoagulant like warfarin. Use of low molecular weight heparin and factor Xa inhibitors will be permitted on case by case basis. There will be no restriction for daily aspirin ≤ 81 mg/QD.
• Have used herbal medication within 14 days prior to the first dose of HCB101.
• Have received any treatment targeting the CD47 or SIRPα pathway.
• Have other malignancies requiring treatment within 2 years prior to the first dose of HCB101.
• Participation in another clinical study with an investigational product administered in the last 14 days prior to receiving the first dose of HCB101.
• An investigational device used within 28 days prior to the first dose of HCB101.
• Positive for hepatitis B, active hepatitis C infections, positive for HIV, or known active or latent tuberculosis.
• Known to have a history of alcoholism or drug abuse.
DRUG: HCB101
Advanced Solid Tumor, Refractory Non-Hodgkin Lymphoma, Anus, Bones and Joints, Breast - Female, Breast - Male, Cervix, Colon, Corpus Uteri, Esophagus, Eye and Orbit, Hodgkins Lymphoma, Kaposis sarcoma, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Multiple Myeloma, Non-Hodgkins Lymphoma, Ovary, Pancreas, Prostate, Rectum, Small Intestine, Stomach, Thyroid, Urinary Bladder
Immunotherapy, CD47, SIRPα, Solid Tumor, Lymphoma
UT Southwestern; Parkland Health & Hospital System
Safety and Efficacy of Epcoritamab With Gemcitabine, Dexamethasone, and Cisplatin (GDP) Salvage Chemotherapy in Relapsed Refractory Large B-cell Lymphoma
Subjects with relapsed large cell lymphoma will receive 3 cycles of combination therapy consisting of GDP and epcoritamab. Each cycle will last 21 days. GDP consists of gemcitabine 1000 mg/m2 IV on Days 1 and 8, cisplatin 75 mg/m2 IV on Day 1, and dexamethasone 40 mg orally on Days 1 through 4. Epcoritamab will be administered subcutaneously (SC) on Days 1, 8, and 15. Patients will receive granulocyte colony stimulating factor (G-CSF) between Day 8 through Day 10 of each cycle of combination therapy. Patients will then undergo radiology imaging for disease assessment. Patients may proceed to SCT(autologous or allogeneic) or CAR T-cell therapy or epcoritamab monotherapy upon completion of Cycle 3 per investigator discretion. The rationale for subjects not proceeding to autoSCT or CAR T-cell therapy will be captured in the eCRFs. Patients who do not undergo SCT or CAR T-cell therapy may have the option to receive study treatment with epcoritamab monotherapy following completion of Cycle 3. Epcoritamab monotherapy will be offered to selected subjects who become ineligible to undergo SCT or CAR T-cell therapy (such as social situation, change in subject decision). The decision to offer epcoritamab monotherapy will be per investigator's discretion. However, subjects must have demonstrated a response to the combination therapy (partial remission or complete remission) per disease assessment scans prior to offering epcoritamab monotherapy. Epcoritamab monotherapy should begin 2 weeks following Cycle 3 Day 15. Monotherapy will consist of epcoritamab 48 mg administered subcutaneously on Days 1 and 15 of each 28 day cycle for Cycle 4 to Cycle 9 or until unacceptable toxicity, or disease progression per the Lugano Criteria.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Elif Yilmaz
ALL
18 Years to old
PHASE2
NCT05852717
STU-2024-1048
Inclusion Criteria:
• Written informed consent and HIPAA authorization for release of personal health information prior to registration. NOTE: HIPAA authorization may be included in the informed consent or obtained separately.
• Age ≥ 18 years at the time of consent.
• ECOG Performance Status of 0-2 within 28 days prior to registration.
• Histological confirmed CD20+ relapsed large cell lymphoma according to the 5th edition of the WHO classification of the hematolymphoid tumors and the 2022 international consensus classification of mature lymphoid neoplasms including de-novo and transformed from prior indolent B-cell NHL such as follicular lymphoma, or marginal zone lymphoma (33, 34). NOTE: Subjects with high-grade B-cell lymphoma (HGBCL), NOS subtype, and high-grade B-cell lymphoma with c-MYC, Bcl2 and/or Bcl6 rearrangements (double or triple hit lymphoma) are eligible. Patients with primary mediastinal B-cell lymphoma, and T-cell histiocyte-rich B-cell lymphoma, primary cutaneous diffuse large B-cell lymphoma, leg type, Intravascular large B-cell lymphoma, Epstein-Barr virus-positive diffuse large B-cell lymphoma, NOS, Diffuse large B-cell lymphoma associated with chronic inflammation, and ALK-positive large B-cell lymphoma are eligible. Patients with Burkitt lymphoma or lymphoplasmacytic lymphoma are not eligible.
• Positron emission tomography (PET) positive measurable disease with at least 1 node having the longest diameter (LDi) greater than (\>) 1.5 centimeter (cm) or 1 extranodal lesion with LDi \>1 cm (per the Lugano Criteria 2014).
• Have received at least 1 prior line of systemic therapy for the treatment of large cell lymphoma. NOTE: Prior radiation therapy or systemic corticosteroids will not be considered a line of therapy.
• Must have had relapsed or refractory disease following standard frontline chemotherapy. Refractory disease is defined as large cell lymphoma not achieving complete remission, progressing or relapsing within 6 months after first-line chemotherapy based on PET/CT per the Lugano criteria. Relapsed disease is defined as disease that recurs beyond 6 months after completion of initial chemotherapy based on PET/CT per the Lugano criteria.
• Patients must be deemed eligible to proceed with stem cell transplantation (autologous or allogeneic) or CAR T-cell therapy per treating physician discretion. Patients being considered for allogeneic stem cell transplant may be eligible.
• Archival tissue obtained within 2 years of signing consent is required if available and will be identified at screening and shipped prior to Cycle 2 Day 1. If archival tissue is not available, fresh tissue from a standard of care biopsy is required. If a subject does not have archival tissue or is not undergoing a standard of care biopsy, they are not eligible for the trial. NOTE: A pre-treatment fresh tissue core or excisional biopsy at screening is preferred which should be considered standard of care.
• Demonstrate adequate organ function. All screening labs to be obtained within 21 days prior to registration. \*Patients with bone marrow involvement will be eligible to participate in the study but must meet hematologic parameters.
• Life expectancy of ≥ 6 months, as determined by the enrolling physician or protocol designee.
• Females subjects of childbearing potential must have a negative urine or serum pregnancy test within 24 hours prior to study treatment. If a urine test is done and it is positice ir cannot be confirmed as negative, a serum pregnancy test will be required.
• Female subjects of childbearing potential and male subjects must be willing to abstain from penile-vaginal intercourse or to use an effective method(s) of contraception.
• As determined by the enrolling physician or protocol designee, ability of the subject to understand and comply with study procedures for the entire length of the study.
Exclusion Criteria:
• Previous treatment with gemcitabine, cisplatin, and epcoritamab or other bispecific T-cell engager antibody (BiTE) such aas glofitamab, mosunetuzumab, or odronextamab.
• Known active central nervous system or meningeal involvement by large cell lymphoma at time of screening. Patients diagnosed with CNS disease who achieved and maintained CNS CR at the time of relapse are eligible. Lumbar puncture must be done in this case prior to study entry to demonstrate CNS CR status. Tests to investigate CNS involvement are required otherwise only if clinically indicated (i.e. disease suspected on basis of symptoms or other findings).
• Contraindication to any drug contained in the combination therapy regimen (GDP).
• Known hypersensitivity or allergic reaction to epcoritamab or its' excipients.
• Use of any standard or experimental anti-large cell lymphoma therapy (including nonpalliative radiation, chemotherapy, immunotherapy, radio-immunotherapy, or any other anticancer therapy) \< 14 days prior to C1D1. NOTE: Prednisone up to 50 mg or equivalent for 5 days is permitted; palliative radiation is permitted only if on non-target lesions).
• Major surgery \< 14 days of Cycle 1 Day 1.
• Neuropathy Grade ≥ 2 (CTCAE v.5.0).
• Patients with a history of other malignancies, except adequately treated non-melanoma skin cancer, non-invasive superficial bladder cancer, curatively treated in-situ cancer of the cervix, DCIS of the breast, localized low grade prostate cancer (up to Gleason score 6), or other solid tumours curatively treated with no evidence of disease for at least 3 years.
• Active bacterial, viral, fungal, mycobacterial, parasitic, or other infection (excluding fungal infections of nail beds) requiring systemic treatment within 7 days prior to the first dose of study treatment. Prophylactic antibacterial, antiviral, and antifungal agents are allowed.
• Active HIV infection. NOTE: Testing for HIV antibody is required at the time of screening. Those with positive HIV antibody will require HIV viral load by PCR testing. Patients with detectable viral load will not be eligible for the study. Those with positive antibody but undetectable viral load and CD4 \>200 will be eligible.
• Testing for hepatitis B (HBV) and hepatitis C virus (HCV) is required at screening. Hepatitis B testing will consist of Hepatitis B surface Antigen (HBsAg), Hepatitis B Core Antibody (HBcAb) and Hepatitis Surface Antibody (HBsAb). Hepatitis C testing will consist of Hepatitis C Antibody (HCAb). Subjects with a history of chronic hepatitis B virus (HBV) infection must have an undetectable HBV viral load on suppressive therapy, if indicated. Subjects with evidence of prior HBV but who are PCR-negative are permitted in the trial but should receive prophylactic antiviral therapy. Subjects with a history of hepatitis C virus (HCV) infection must have been treated. For patients with HCV infection who are currently on treatment, the HCV viral load must be undetectable to be eligible for this trial. Subjects who received treatment for HCV that was intended to eradicate the virus may participate if hepatitis C RNA levels are undetectable.
• Pregnant or breastfeeding (NOTE: breast milk cannot be stored for future use while the mother is being treated on study).
• Any life-threatening illness, medical condition, or organ system dysfunction which, in the Investigator's opinion, could compromise the subject's safety, or being compliant with the study procedures.
PROCEDURE: AutoSCT OR CAR T-cell Therapy, DRUG: GDP, DRUG: Epcoritamab
Large Cell Lymphoma, Diffuse, Relapsed Cancer, Refractory Cancer, Non-Hodgkins Lymphoma
UT Southwestern
A Study With Tovorafenib (DAY101) as a Treatment Option for Progressive, Relapsed, or Refractory Langerhans Cell Histiocytosis
This phase II trial tests the safety, side effects, best dose and activity of tovorafenib (DAY101) in treating patients with Langerhans cell histiocytosis that is growing, spreading, or getting worse (progressive), has come back (relapsed) after previous treatment, or does not respond to therapy (refractory). Langerhans cell histiocytosis is a type of disease that occurs when the body makes too many immature Langerhans cells (a type of white blood cell). When these cells build up, they can form tumors in certain tissues and organs including bones, skin, lungs and pituitary gland and can damage them. This tumor is more common in children and young adults. DAY101 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Using DAY101 may be effective in treating patients with relapsed or refractory Langerhans cell histiocytosis.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Erin Butler
ALL
180 Days to 22 Years old
PHASE2
NCT05828069
STU-2023-0818
Inclusion Criteria:
* 180 days- \< 22 years (at time of study enrollment)
* Patient must have a body surface area of ≥ 0.3 m\^2
* Patients with progressive, relapsed, or recurrent LCH with measurable disease at study entry
* Patients must have had histologic verification of LCH (from either original diagnosis or relapse/progression) at the time of study entry (must be obtained within 28 days prior to enrollment and start of protocol therapy) (repeat if necessary)
* Tissue confirmation of relapse is recommended but not required
* Pathology report must be submitted for central confirmation of diagnosis within 7 days of enrollment.
* Formalin-fixed paraffin-embedded (FFPE) blocks or unstained slides (initial diagnosis and/or subsequent biopsies) will be required for retrospective central confirmation of diagnosis and molecular studies
* Patients with mixed histiocytic disorders (e.g. LCH with juvenile xanthogranuloma) may be included
* Patients must have measurable disease, documented by radiographic imaging (LCH- specific response criteria (must be obtained within 28 days prior to enrollment and start of protocol therapy) (repeat if necessary).
* Patients must have progressive or refractory disease or experience relapse after at least one previous systemic treatment strategy
* Pathogenic somatic mutation detected in genes encoding tyrosine kinase receptors (CSFR1, ERBB3 or ALK), RAS or RAF (may be from original or subsequent biopsy or peripheral blood/bone marrow aspirate). Clinical mutation reports may include quantitative polymerase chain reaction (PCR) (e.g. BRAFV600E) and/or Sanger or next generation sequencing. Immunohistochemistry (e.g. VE1 antibody for BRAFV600E) alone is not sufficient
* Participant must be able to take an enteral dose and formulation of medication. Study medication is only available as an oral suspension or tablet, which may be taken by mouth or other enteral route such as nasogastric, jejunostomy, or gastric tube
* Karnofsky \>= 50% for patients \> 16 years of age and Lansky \>= 50% for patients =\< 16 years of age
* Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1 or 2. Use Karnofsky for patients \> 16 years of age and Lansky for patients =\< 16 years of age
* Myelosuppressive chemotherapy: Patients must not have received within 14 days of entry onto this study
* Investigational agent or any other anticancer therapy not defined above: Patients must not have received any investigational agent or any other anticancer therapy (including MAPK pathway inhibitor) for at least 14 days prior to planned start of tovorafenib (DAY101)
* Radiation therapy (RT): Patient must not have received RT within 2 weeks after the last dose fraction of RT
* Patients must have fully recovered from any prior surgery
* Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, targeted inhibitor, and/or radiotherapy with toxicities reduced to grade 1 or less (Common Terminology Criteria for Adverse Events \[CTCAE\] version 5.0)
* Steroids: =\< 0.5 mg/kg/day of prednisone equivalent (maximum 20 mg/day) averaged during the month prior to study enrollment is permissible
* Strong inducers or inhibitors of CYP2C8 are prohibited for 14 days before the first dose of tovorafenib (DAY101) and from planned administration for the duration of study participation
* Medications that are breast cancer resistant protein (BCRP) substrates that have a narrow therapeutic index are prohibited for 14 days before the first dose of tovorafenib (DAY101) and for the duration of study participation
* Peripheral absolute neutrophil count (ANC) \>= 750/uL unless secondary to bone marrow involvement, in such cases bone marrow involvement must be documented (must be performed within 7 days prior to enrollment, must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment)
* Platelet count \>= 75,000/uL (unsupported/without transfusion within the past 7 days) (must be performed within 7 days prior to enrollment, must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment)
* Patients with marrow disease must have platelet count of \>= 75,000/uL (transfusion support allowed) and must not be refractory to platelet transfusions. Bone marrow involvement must be documented
* Hemoglobin \>= 8 g/dL (unsupported/without transfusion within the past 7 days). Patients with marrow disease must have hemoglobin \>= 8 g/dL (transfusion support allowed). Bone marrow involvement must be documented
* Hematopoietic growth factors: At least 14 days after the last dose of a long-acting growth factor (e.g., Neulasta \[registered trademark\]) or 7 days for short-acting growth factor
* A serum creatinine based on age/sex as follows (must be performed within 7 days prior to enrollment, must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment)
* Age: 6 months to \< 1 year; Maximum Serum Creatinine (mg/dL):= 0.5 mg/dl (male and female)
* Age: 1 to \< 2 years; Maximum Serum Creatinine (mg/dL): = 0.6 mg/dl (male and female)
* Age: 2 to \< 6 years; Maximum Serum Creatinine (mg/dL): = 0.8 mg/dl (male and female)
* Age: 6 to \< 10 years; Maximum Serum Creatinine (mg/dL): = 1.0 mg/dl (male and female)
* Age: 10 to \< 13 years; Maximum Serum Creatinine (mg/dL): = 1.2 mg/dl (male and female)
* 13 to \< 16 years; Maximum Serum Creatinine (mg/dL): = 1.5 mg/dl (male) and 1.4 mg/dl (female)
* Age: \>= 16 years; Maximum Serum Creatinine (mg/dL): = 1.7 mg/dl (male) and 1.4 mg/dl (female)
* OR- a 24 hour urine creatinine clearance \>= 50 mL/min/1.73 m\^2
* OR- a glomerular filtration rate (GFR) \>= 50 mL/min/1.73 m\^2. GFR must be performed using direct measurement with a nuclear blood sampling method OR direct small molecule clearance method (iothalamate or other molecule per institutional standard)
* Note: Estimated GFR (eGFR) from serum creatinine, cystatin C or other estimates are not acceptable for determining eligibility
* Bilirubin (sum of conjugated + unconjugated) =\< 1.5 x upper limit of normal (ULN) for age (must be performed within 7 days prior to enrollment, must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment)
* Alanine aminotransferase (ALT) =\< 3 x ULN for age (must be performed within 7 days prior to enrollment, must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment)
* Serum albumin \>= 2 g/dl must be performed within 7 days prior to enrollment, must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment)
* For patients with liver disease caused by their histiocytic disorder (as evaluated on radiographic imaging or biopsy): patients may be enrolled with abnormal bilirubin, aspartate aminotransferase (AST), ALT and albumin with documentation of histiocytic liver disease
* Fractional shortening (FS) of \>= 25% or ejection fraction of \>= 50%, as determined by echocardiography or multigated acquisition scan (MUGA) within 28 days prior to study enrollment. Depending on institutional standard, either FS or left ventricular ejection fraction (LVEF) is adequate for enrollment if only one value is measured; if both values are measured, then both values must meet criteria above (must be obtained within 28 days prior to enrollment and start of protocol therapy) (repeat if necessary)
* No evidence of dyspnea at rest, no exercise intolerance, and a pulse oximetry \> 94% if there is clinical indication for determination; unless it is due to underlying pulmonary LCH
* Central Nervous System Function Defined As:
* Patients with seizure disorder may be enrolled if well controlled
* Central nervous system (CNS) toxicity =\< Grade 2
* Human immunodeficiency virus (HIV) infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial unless antiretroviral therapy interacts with the metabolism of tovorafenib (DAY101) and cannot safely be changed to antivirals that do not interact with study medication
Exclusion Criteria:
* LCH arising along with other hematologic malignancy (e.g. mixed LCH with acute lymphoblastic leukemia) or any history of non-histiocytic malignancy
* Disease scenarios as below will be excluded
* Skin-limited disease
* Gastrointestinal (GI) tract involvement only (those that have disease that can be determined by endoscopic biopsies only)
* LCH-associated neurodegeneration (LCH-ND) without parenchymal lesions or other systemic lesions
* Patients with activating mutations in MAP2K1 are not eligible for this study due to drug target specificity. Mutation status will be submitted to study team within 7 days of enrollment
* Refractory nausea and vomiting, malabsorption, or external biliary shunt that would preclude adequate absorption of tovorafenib (DAY101)
* Uncontrolled systemic bacterial, viral, or fungal infection
* Major surgical procedure or significant traumatic injury within 14 days prior to study enrollment, or anticipation of need for major surgical procedure during the course of the study. Placement of a vascular access device or minor surgery is permitted within fourteen (14) days of study enrollment (provided that the wound has healed)
* History of significant bowel resection that would preclude adequate absorption or other significant malabsorptive disease
* Ophthalmologic considerations: Patients with known significant ophthalmologic conditions or known risk factors for retinal vein occlusion (RVO) or central serous retinopathy (CSR) are not eligible
* History of solid organ or hematopoietic bone marrow transplantation
* Clinically significant active cardiovascular disease, or history of myocardial infarction, or deep vein thrombosis/pulmonary embolism within 6 months prior to enrollment, ongoing cardiomyopathy, or current prolonged QT interval \> 440 ms based on triplicate electrocardiogram (ECG) average
* History of Grade \>= 2 CNS hemorrhage or history of any CNS hemorrhage within 28 days of study entry
* History of any drug reaction with eosinophilia and systemic symptoms (DRESS) syndrome or Stevens Johnsons syndrome (SJS) or who are allergic to tovorafenib (DAY101) or any of its components
* CTCAE version (V). 5.0 Grade 3 symptomatic creatinine kinase (CPK) elevation ( \> 5 x ULN)
* Female patients who are pregnant are ineligible. A pregnancy test is required for female patients of childbearing potential
* Lactating females who plan to breastfeed their infants are ineligible
* Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation are ineligible. Women of childbearing potential must use non-hormonal contraception during tovorafenib treatment and for at least 28 days after the last dose. Men should use effective contraception and must not father a child while taking tovorafenib and for 14 days after the last dose PROCEDURE: Biospecimen Collection, PROCEDURE: Bone Marrow Aspiration, PROCEDURE: Bone Marrow Biopsy, PROCEDURE: Computed Tomography, PROCEDURE: Echocardiography Test, PROCEDURE: FDG-Positron Emission Tomography and Computed Tomography Scan, PROCEDURE: Lumbar Puncture, PROCEDURE: Multigated Acquisition Scan, DRUG: Tovorafenib
Recurrent Langerhans Cell Histiocytosis, Refractory Langerhans Cell Histiocytosis, Bones and Joints, Other Skin, Brain and Nervous System, Liver, Lung/Thoracic, Other Hematopoietic, Small Intestine
Children’s Health
The Rhythm Evaluation for AntiCoagulaTion With Continuous Monitoring of Atrial Fibrillation (REACT-AF)
REACT-AF is a multicenter prospective, randomized, open-label, blinded endpoint (PROBE design), controlled trial comparing the current Standard Of Care (SOC) of continuous Direct Oral Anticoagulation (DOAC) use versus time-delimited (1 month) DOAC guided by an AF-sensing Smart Watch (AFSW) in participants with a history of paroxysmal or persistent Atrial Fibrillation (AF) and low-to-moderate stroke risk.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Vukile.Mlambo@utsouthwestern.edu
Mark Link
ALL
22 Years to 85 Years old
PHASE3
NCT05836987
STU-2023-0473
Inclusion Criteria:
• 22-85 years of age.
• English speaking participants. Spanish-only speakers may be included in the future at select sites appropriately translated.
• History of non-permanent atrial fibrillation.
• CHA2DS2-VASC score of 1-4 for men and 2-4 for women without prior stroke or Transient Ischemic Attack (TIA), The CHA2DS2-VASc score is a point-based system used to stratify the risk of stroke in Atrial Fibrillation (AF) patients. The acronym CHA2DS2-VASc stands for congestive heart failure, hypertension, age ≥75 (doubled), diabetes, stroke (doubled), vascular disease, age 65 to 74 and sex category (female). Congestive heart failure defined as: The presence of signs and symptoms of either right (elevated central venous pressure, hepatomegaly, dependent edema) or left ventricular failure (exertional dyspnea, cough, fatigue, orthopnea, paroxysmal nocturnal dyspnea, cardiac enlargement, rales, gallop rhythm, pulmonary venous congestion) or both, confirmed by non-invasive or invasive measurements demonstrating objective evidence of cardiac dysfunction and/or ejection fraction \< 40%.
• The participant is on a DOAC at the time of screening and willing to stay on DOAC for duration of study.
• Willing and able to comply with the protocol, including: * Possession of a smart watch-compatible smart phone (iPhone that supports the latest shipping iOS) with a cellular service plan * Be willing to wear the smart watch for the suggested minimum of 14 hours a day * Expected to be within cellular service range at least 80% of the time
• Willing and able to discontinue DOAC
• The participant is willing and able to provide informed consent.
Exclusion Criteria:
• Valvular or permanent atrial fibrillation.
• Current treatment with warfarin and unwilling or unable to take a DOAC.
• The participant is a woman who is pregnant or nursing.
• The participant is being treated with chronic aspirin, another anti-platelet agent, or chronic NSAIDS outside of current medical guidelines (e.g., primary stroke prevention in patients with atrial fibrillation, primary prevention of cardiovascular events, pain relief, fever, gout) and is unwilling or unable to discontinue use for the study duration.
• Existing cardiac rhythm device or indication for a permanent pacemaker, Implantable Cardioverter-Defibrillator (ICD) or Cardiac Resynchronization Therapy (CRT) device or planned insertable cardiac monitor. Insertable cardiac monitors are permitted unless they are being used to guide anticoagulation treatment.
• Known or suspected symptomatic or asymptomatic atrial fibrillation lasting ≥ 1 hour/month over the last 3 months.
• Any documented single AF episode lasting ≥ 1 hour on standard of care or study-provided external cardiac monitor of \> 6 days duration performed within 45 days prior to randomization. Shorter monitoring durations may be acceptable for inclusion at the discretion of the site PI based on the totality of monitoring data and approval of the study PI.
• Ablation for AF within the last 2 months.
• Prior or anticipated left atrial appendage occlusion or ligation.
• Mechanical prosthetic valve(s) or severe valve disease.
• Hypertrophic cardiomyopathy.
• Participant needs DOAC for reasons other than preventing stroke or arterial embolism resulting from AF (i.e., preventing Deep Vein Thrombosis (DVT) or PE) or needs permanent OAC (i.e., congenital heart defects, prosthetic heart valve).
• Participants deemed high risk for non-cardioembolic stroke (i.e., significant carotid artery disease defined as stenosis \> 75%) based on the investigator's discretion.
• The participant is enrolled, has participated within the last 30 days, or is planning to participate in a concurrent drug and/or device study during the course of this clinical trial. Co-enrollment in concurrent trials is only allowed with documented pre-approval from the study manager; there is no concern that co-enrollment could confound the results of this trial.
• The participant has a tattoo, birthmark, or surgical scar over the dorsal wrist area on the ipsilateral side that the AFSW may be worn.
• The participant has a tremor on their ipsilateral side that the AFSW may be worn.
• Any concomitant condition that, in the investigator's opinion, would not allow safe participation in the study (e.g., drug addiction, alcohol abuse).
• Known hypersensitivity or contraindication to direct oral anticoagulants.
• Documented prior stroke (ischemic or hemorrhagic) or transient ischemic attack.
• Reversible causes of AF (e.g., cardiac surgery, pulmonary embolism, untreated hyperthyroidism). AF ablation does not constitute reversible AF.
• \> 5% burden of premature atrial or ventricular depolarizations on pre-enrollment cardiac monitoring.
• History of atrial flutter that has not been treated with ablation (participants in atrial flutter and have been ablated are eligible for enrollment).
• Stage 4 or 5 chronic kidney disease.
• Conditions associated with an increased risk of bleeding: * Major surgery in the previous month * Planned surgery or intervention in the next three months that would require cessation of anticoagulation \> 2 weeks. * History of intracranial, intraocular, spinal, retroperitoneal, or atraumatic intra- articular bleeding * Gastrointestinal hemorrhage within the past year unless the cause has been permanently eliminated (e.g., by surgery) * Symptomatic or endoscopically documented gastroduodenal ulcer disease in the previous 30 days * Hemorrhagic disorder or bleeding diathesis * Need for anticoagulant treatment for disorders other than AF * Uncontrolled hypertension (Systolic Blood Pressure \>180 mmHg and/or Diastolic Blood Pressure \>100 mmHg)
DEVICE: AFSW Guided DOAC, DRUG: Continuous DOAC therapy
Atrial Fibrillation, Heart
Atrial Fibrillation, Anticoagulation, AF-sensing Smart Watch, Ischemic Stroke, Systemic Embolism
UT Southwestern
A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace) (PALace)
This is a 10-year multi-center, global, observational study to further characterize the safety profile of pegvaliase, including hypersensitivity reactions, long-term safety and tolerability, and the effectiveness of the additional risk minimization measures (aRMMs) (European Union (EU) only) in subjects receiving pegvaliase for the treatment of PKU. Subjects for whom a clinical decision has been made that they will receive pegvaliase to treat their PKU within 30 days following the date of enrollment (incident-users) or have previously started treatment with pegvaliase at the date of enrollment (prevalent-users) are eligible for participation in this study.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Juana.Luevano@UTSouthwestern.edu
Markey McNutt
All
Not specified
NCT05813678
STU-2023-0263
Inclusion Criteria:
• Documented diagnosis of PKU per local standard of care
• Currently receiving or planned to receive pegvaliase treatment within 30 days after the date of enrollment, including subjects who previously received pegvaliase as part of the clinical development program and have completed study participation.
• Subject (or legally authorized representative) is willing and able to provide written informed consent after the nature of the study has been explained and prior to any data collection.
Exclusion Criteria:
• Subject has any kind of disorder that, in the opinion of the investigator, may compromise the ability of the subject to give written informed consent and/or comply with any aspect of the study.
• Currently participating in an interventional study of any investigational product, device, or procedure
• Previously enrolled in this study (eg, subjects who have been withdrawn from the study and wish to participate again at a later date)
• German subjects <16 years if age
Drug: Pegvaliase
Phenylketonuria (PKU), Other Endocrine System
Observational, Safety Study, Pegvaliase, Palynziq, PKU, Phenylketonuria, Phase 4
UT Southwestern; Children’s Health
A Study to Compare Iberdomide Maintenance Versus Lenalidomide Maintenance Therapy Following Autologous Stem Cell Transplant in Participants With Newly Diagnosed Multiple Myeloma
The purpose of this study is to compare the effectiveness of iberdomide maintenance to lenalidomide maintenance therapy after autologous stem cell transplantation (ASCT) in participants with newly diagnosed multiple myeloma (NDMM).
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Larry Anderson
ALL
18 Years and over
PHASE3
NCT05827016
STU-2023-0468
Inclusion Criteria
* Confirmed diagnosis of symptomatic multiple myeloma (MM).
* Eastern Cooperative Oncology Group performance status (ECOG) score of 0, 1, or 2.
* Received 3 to 6 cycles of an induction therapy that includes a proteasome inhibitor (PI) and immunomodulatory (IMiD) \[eg, bortezomib thalidomide and dexamethasone (VTd), lenalidomide, bortezomib and dexamethasone (RVd)\] with or without a CD38 monoclonal antibody, daratumumab + bortezomib/thalidomide/dexamethasone \[D-VTd\] and daratumumab + bortezomib/ lenalidomide/dexamethasone \[D-VRd\]), or VCd / daratumumab + bortezomib/cyclophosphamide/dexamethasone \[D-VCd\], and followed by a single or tandem autologous stem cell transplantation (ASCT). Post-stem cell transplant consolidation is permitted.
* Participants within 12 months (single transplant) or 15 months (tandem transplant) from initiation of induction therapy who achieved at least a partial response (PR) after autologous stem cell transplantation (ASCT) with or without consolidation, according to International Myeloma Working Group (IMWG 2016) criteria.
Exclusion Criteria
* Progressive disease or clinical relapse (as defined by IMWG response criteria) following ASCT with or without consolidation or is not responsive to primary therapy.
* Smoldering myeloma, solitary plasmacytoma or nonsecretory myeloma.
* Known central nervous system/meningeal involvement of MM.
* Prior history of malignancies, other than MM, unless the participant has been free of the disease for ≥ 5 years.
* Other protocol-defined Inclusion/Exclusion criteria apply.
DRUG: Iberdomide, DRUG: Lenalidomide
Multiple Myeloma
Multiple Myeloma, Iberdomide, CC-220, Lenalidomide, MM, NDMM, Maintenance, Autologous stem cell transplant, ASCT, CELMoD, Cereblon Modulators
UT Southwestern
Trifecta-Lung cfDNA-MMDx Study
Demonstrate the relationship between dd-cfDNA levels and HLA antibodies in blood transplant recipient and Demonstrate the Molecular Microscope® (MMDx) Diagnostic System results in indication and protocol biopsies from lung transplants.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Rhoda.AnnohGordon@UTSouthwestern.edu
Irina Timofte
ALL
18 Years to old
NCT05837663
STU-2024-0425
Inclusion Criteria:
Adult, Older adult
Exclusion Criteria:
Patients will be excluded from the study if they decline participation Are unable to give informed consent. Recipients of multiple organs, cancer patients and pregnant women Lung Transplantation
Donor derived cfDNA, Gene expression, Lung transplant biopsy, Blood
UT Southwestern
Long Term Efficacy and Safety of Orlistat for Type 1 Hyperlipoproteinemia
Type I hyperlipoproteinemia (T1HLP, also known as familial chylomicronemia syndrome or FCS) is a rare diseasewhere the blood triglycerides (fats) are very high. It is caused by lack of certain enzymes and proteins in the bodythat are important in disposing circulating fats from blood. Treatment of T1HLP patients who have very high levels of blood fats (≥ 1,000 mg/dL) is challenging as conventional triglyceride-lowering medications, such as fibrates and fishoil, are ineffective. The purpose of this trial is to study the long-term efficacy and safety of orlistat for reducing blood triglyceride levels in patients with T1HLP.
Call 214-648-5005
studyfinder@utsouthwestern.edu, CHANDNA.VASANDANI@UTSouthwestern.edu
Abhimanyu Garg
ALL
8 Years to 70 Years old
PHASE2
NCT05816343
STU-2019-0776
* Type I hyperlipoproteinemia confirmed by bi-allelic disease-causing variants in any one of the T1HLP genes (LPL, APOC2, APOA5, LMF1, GPIHBP1, or GCKR).
* Subjects who have a fasting TG greater than or equal to 750 mg/dL at the end of run-in period of 8 weeks will be eligible for randomization.
* Age ≥ 8 years
* Well controlled diabetes mellitus with hemoglobin A1c \< 8%
* Off orlistat for a period of 2 months
Exclusion Criteria:
* Secondary hypertriglyceridemia due to diabetes, renal disease, , alcoholism and drug therapy such as estrogens and estrogen analogues, steroids, HIV-1 protease inhibitors, retinoic acid derivatives, interferons, or lasparaginase.
* On lomitapide or participating in clinical trial of volanesorsen and olezarsen
* On cyclosporine
* Having serum TSH outside of the normal range if on levothyroxine supplementation.
* Use of levothyroxine to suppress TSH in individuals with thyroid cancer.
* Pregnant or lactating women
* Significant liver disease (elevated transaminases \> 2 times upper limit of normal)
* Alcohol abuse (\> 7 drinks or 84 g per week for women and \> 14 drinks or 168 g per week for men)
* Severe anemia (hematocrit \< 24%)
* Illicit drug use (cocaine, marijuana, LSD, etc.)
* Major surgery in the past three months
* Congestive heart failure
* Serum creatinine greater than 2.5 mg/dL
* Cancer within the past five years
* Gastrointestinal surgery in the past
* Current therapy with anti-coagulants, digoxin and anti-arrhythmics
* Chronic malabsorption syndromes
* Cholestasis
* Acute illnesses such as acute pancreatitis in the last 8 weeks
* Previous history of renal calcium oxalate stones DRUG: Orlistat, DRUG: Placebo
Type 1 Hyperlipoprotenemia
UT Southwestern; Parkland Health & Hospital System
Pembrolizumab vs. Observation in People With Triple-negative Breast Cancer Who Had a Pathologic Complete Response After Chemotherapy Plus Pembrolizumab
The phase III trial compares the effect of pembrolizumab to observation for the treatment of patients with early-stage triple-negative breast cancer who achieved a pathologic complete response after preoperative chemotherapy in combination with pembrolizumab. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. This trial may help researchers determine if observation will result in the same risk of cancer coming back as pembrolizumab after surgery in triple-negative breast cancer patients who achieve pathologic complete response after preoperative chemotherapy with pembrolizumab.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Heather McArthur
ALL
18 Years and over
PHASE3
NCT05812807
STU-2023-0742
Inclusion Criteria:
* Age \>= 18 years
* Eastern Cooperative Oncology Group (ECOG) Performance Status 0-2
* Triple Negative Breast Cancer:
* Patients with a history of stage T1cN1-2 or T2-4N0-2 breast cancer according to the primary tumor-regional lymph node anatomic staging criteria of the American Joint Committee on Cancer (AJCC), 8th edition as determined by the investigator in radiologic assessment, clinical assessment or both
* Patients must have no residual invasive disease in the breast or lymph nodes after the completion of neoadjuvant therapy. Residual ductal carcinoma in situ (DCIS) is allowed. Isolated tumor cells are considered node-negative
* Estrogen (ER) and progesterone (PR) =\< 10%; HER2-negative by American Society of Clinical Oncology (ASCO)/College of American Pathologists (CAP) guidelines (immunohistochemistry \[IHC\] and fluorescence in situ hybridization \[FISH\])
* If invasive disease was present in both breasts, participation in the study is permitted as long as the eligibility criteria are met for both tumors/breasts
* Patients must have received neoadjuvant chemotherapy in combination with pembrolizumab for a minimum of 6 cycles. All systemic chemotherapy and ICI therapy should have been completed preoperatively
* An interval of no more than 12 weeks between the completion date of the final surgery and the date of randomization
\* Note: Adjuvant radiation can be given on study. If given, it is encouraged to be given concurrently with pembrolizumab, per investigator discretion. Treatment with adjuvant pembrolizumab is strongly discouraged prior to participation in this trial, but if administered (e.g., if patients are awaiting pathology results), pembrolizumab may be administered for up to 6 weeks post-surgery and must be completed prior to registration
* Use of investigational anti-cancer agents must be discontinued at time of registration
* Adequate excision: Surgical removal of all clinically evident disease in the breast and lymph nodes as follows:
* Breast surgery: Total mastectomy or breast-conserving surgery with histologically negative margins, including no ink on tumor for DCIS, at the time of excision
\*\* For patients who undergo breast-conserving surgery, the margins of the resected specimen must be histologically free of ductal carcinoma in-situ (DCIS) as determined by the local pathologist. If pathologic examination demonstrates DCIS at the line of resection, additional operative procedures may be performed to obtain clear margins. If DCIS is still present at the resected margin after re-excision(s), the patient must undergo total mastectomy to be eligible. Patients with margins positive for classic lobular carcinoma in situ (LCIS) are eligible without additional resection
* Lymph node surgery:
* For a patient with clinically N0 disease, a sentinel lymph node biopsy should have been performed at time of surgical evaluation, and if pathologically node positive, the patient is no longer eligible. Isolated tumor cells are considered node-negative
* For a patient with clinically N1 disease at diagnosis (with positive results from a fine-needle aspiration, core biopsy, or sentinel node biopsy performed prior to preoperative therapy) additional surgical evaluation of the axilla following preoperative therapy is required
\*\*\* If they become cN0 (no palpable adenopathy), then a sentinel lymph node biopsy could have been performed at time of surgery (axillary dissection would also be permitted); if the sentinel lymph node biopsy is positive, the patient is no longer eligible
* If sentinel node biopsy performed before preoperative therapy was negative, no additional surgical evaluation of the axilla is required after preoperative therapy. If sentinel node biopsy performed before preoperative therapy was positive, an ALND is required after preoperative therapy
* If the only sentinel node identified by isotope scan is in the internal mammary chain, surgical evaluation of the axilla is still required
* If sentinel node evaluation after preoperative therapy is negative, no further additional surgical evaluation of the axilla is required
* Axillary dissection without sentinel node evaluation is permitted as the initial or sole axillary evaluation after preoperative therapy
* If breast-conserving surgery was performed but patient will not be receiving breast radiation, the patient is not eligible
* Not pregnant and not nursing, because this study involves an agent whose genotoxic, mutagenic and teratogenic effects on the developing fetus and newborn are unknown. Therefore, for women of childbearing potential only, a negative serum or urine pregnancy test done =\< 7 days prior to randomization is required
* Absolute neutrophil count (ANC) \>= 1,000/mm\^3
* Platelet Count \>= 100,000/mm\^3
* Estimated glomerular filtration rate (eGFR) \>= 15 mL/min/1.73m\^2
* Total Bilirubin =\<1.5 x upper limit of normal (ULN)
\* Patients with Gilbert's disease with a total bilirubin =\< 2.5 x ULN and direct bilirubin within normal limits are permitted
* Aspartate aminotransferase (AST) serum aspartate aminotransferase \[SGOT\] / alanine aminotransferase (ALT) serum glutamic pyruvic transaminase \[SGPT\] =\< 3 x institutional ULN
* Patients must be willing to provide tumor tissue from the diagnostic core biopsy. If inadequate tumor tissue is available, patients are still eligible to participate in the trial
* Patients with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial
* Patients with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association Functional Classification. To be eligible for this trial, patients should be class 2B or better
* Human immunodeficiency virus (HIV)-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months prior to registration are eligible for this trial
Exclusion Criteria:
* No stage IV (metastatic) breast cancer
* No history of any prior (ipsi- or contralateral) invasive breast cancer. Prior DCIS is allowed
* No evidence of recurrent disease following preoperative therapy and surgery
* No known active liver disease, e.g. due to hepatitis B virus (HBV), hepatitis C virus (HCV), autoimmune hepatic disorders, or sclerosing cholangitis
* No history of intolerance, including Grade 3 or 4 infusion reaction or hypersensitivity to pembrolizumab or murine proteins or any components of the product
\* Note: Prior immune-related adverse events (irAEs) are allowed if they resolved and the patient tolerated subsequent therapy without requiring chronic steroids for the irAE
* No medical conditions that require chronic systemic steroids (\>10 mg prednisone daily or equivalent) or any other form of immunosuppressive medications and has required such therapy in the last two years. Replacement therapy (e.g. thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic therapy
* Patients who are unable or unwilling to comply with the requirements of the protocol per investigator assessment are not eligible BIOLOGICAL: Pembrolizumab, OTHER: Patient Observation, PROCEDURE: Biopsy, PROCEDURE: Biospecimen Collection, OTHER: Questionnaire Administration, OTHER: Quality-of-Life Assessment
Anatomic Stage I Breast Cancer AJCC v8, Anatomic Stage II Breast Cancer AJCC v8, Anatomic Stage III Breast Cancer AJCC v8, Early Stage Triple-Negative Breast Carcinoma, Breast - Female, Breast - Male
UT Southwestern; Parkland Health & Hospital System
Replacing Invasive Cystoscopy With Urine Testing for Non-muscle Invasive Bladder Cancer Surveillance (ReplaceCysto)
The purpose of this research is to determine whether bladder cancer monitoring can be improved by replacing some cystoscopy procedures with urine testing. Specifically, this study examines whether there are any differences in urinary symptoms, discomfort, number of invasive procedures, anxiety, complications, cancer recurrence or cancer progression when some cystoscopy procedures are replaced with urine testing.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Yair Lotan
ALL
18 Years and over
NA
NCT05796375
STU-2023-0996
Inclusion Criteria:
• Aged 18 years or older
• History of low grade intermediate-risk non-muscle invasive bladder cancer, defined as most recent pathology report showing any of the following: * multifocal low grade non-invasive urothelial carcinoma of any size * solitary low grade non-invasive urothelial carcinoma greater than 3cm in size * recurrent low grade non-invasive urothelial carcinoma
• Stated willingness to comply with all study procedures and availability for the duration of the study
• No evidence for recurrence at cystoscopy ≤6 months after most recent tumor resection
• Ability to consent in English or Spanish
Exclusion Criteria:
• History of total cystectomy of the bladder.
• History of urinary diversion (e.g., neo-bladder, colon pouch, or ileal conduit).
• History of muscle-invasive bladder tumor.
• Pregnancy or lactation.
• History of urothelial carcinoma of the ureter or renal pelvis status post endoscopic treatment or with evidence of recurrent upper tract disease (inclusion allowed if status post nephroureterectomy and recurrence free at time of inclusion)
• Anatomic constraints making cystoscopy impossible (e.g., history of urethrectomy, obliterated urethra secondary to stricture).
• Inability to provide a voided urine sample.
PROCEDURE: Cystoscopy, DIAGNOSTIC_TEST: Bladder EpiCheck urine test, DIAGNOSTIC_TEST: Xpert Bladder Cancer Monitor urine test
Non-muscle-invasive Bladder Cancer, Urinary Bladder
Bladder, Cancer, Urology, Cystoscopy, Xpert, EpiCheck, Urine test
UT Southwestern
The GORE VBX FORWARD Clinical Study: A Comparison of the GORE® VIABAHN® VBX Balloon Expandable Endoprosthesis to Bare Metal Stenting for Patients With Complex Iliac Occlusive Disease
The objective of this prospective, multicenter, randomized, controlled clinical trial is to demonstrate the superiority of the VBX Device for primary patency when compared to bare metal stenting in complex iliac occlusive disease.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Antonio.SolanoAvendano@UTSouthwestern.edu
Melissa Kirkwood
ALL
18 Years and over
NA
NCT05811364
STU-2023-0754
Inclusion Criteria:
* Age ≥ 18 years at time of informed consent signature
* Informed Consent Form (ICF) is signed by the subject
* Subject can comply with protocol requirements, including follow-up
* Patient has symptomatic claudication, rest pain, or minor tissue loss (Rutherford Category 2-5)
* Patient has de novo or restenotic lesion(s) found in the common and/or external iliac artery(ies)
* Patient has: Unilateral or bilateral single or multiple lesions (\>50% stenosis or chronic total occlusion) each between 4 and 11 cm in length
* Patient has a target vessel diameter visually estimated to be approximately between 5 mm and 13 mm
* Patient has a sufficient (\<50% stenotic) common femoral artery and at least one sufficient (\<50% stenotic) femoral artery (deep or superficial).
* Patient has at least one sufficient (\<50% stenotic) infrapopliteal run-off vessel.
Exclusion Criteria:
* Life expectancy \<1 year
* Patient is pregnant at time of informed consent.
* Patient has a known allergy to stent or stent graft components (including nitinol, stainless steel, or heparin).
* Patient has severe chronic renal insufficiency (serum creatinine level \> 2.5mg/dL) and not undergoing hemodialysis.
* Patient has evidence of a systemic infection.
* Patient has a known intolerance to antithrombotic medications that prevent compliance with study or control device Instructions for Use.
* Patient has had vascular catheterization of the lower extremities within 30 days of randomization (excluding diagnostic angiograms for the study procedure).
* Patient has previous stenting in the iliac arteries.
* Patient has previous surgical bypass in the target limb.
* Patient is currently participating in another investigative clinical study unless received written approval by the sponsor.
* Patient has a lesion requiring drug-coated balloon angioplasty, atherectomy, lithotripsy, or any ablative device to facilitate stent delivery.
* Patient has an abdominal aortic artery lesion or aneurysm.
* Patient has a lesion that requires stent placement within 2 cm of the inguinal ligament.
* Patient has isolated common iliac artery stenosis that can be treated with a single device (i.e., common iliac artery stenosis that does not require kissing stents or extend into the external iliac artery).
* Patient has outflow disease that requires concomitant interventions (i.e. common femoral endarterectomy or femoral / tibial revascularization). DEVICE: Stenting of the Common and/or External Iliac Arteries with the GORE® VIABAHN® VBX Balloon Expandable Endoprosthesis, DEVICE: Stenting of the Common and/or External Iliac Arteries with Bare Metal Stent
Aortoiliac Occlusive Disease, Peripheral Arterial Disease, Other Hematopoietic
UT Southwestern
Study of JANX008 in Subjects With Advanced or Metastatic Solid Tumor Malignancies
This study is a first-in-human (FIH), Phase 1/1b, open-label, multicenter dose escalation and dose expansion study to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary anti-tumor activity of JANX008 in adult subjects with advanced or metastatic carcinoma expressing EGFR.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Tian Zhang
ALL
18 Years to 100 Years old
PHASE1
NCT05783622
STU-2023-0808
Inclusion Criteria:
* Subjects ≥18 years of age at the time of signing informed consent
* Histologically or cytologically documented locally advanced or metastatic NSCLC, SCCHN, CRC, RCC, SCLC, PDAC, TNBC
* Progressed or was intolerant to all available therapies known to confer clinical benefit appropriate for the tumor type
* Adequate organ function
* At least 1 measurable lesion per RECIST 1.1
Exclusion Criteria:
* Treatment with anti-cancer therapy within 28 days or ≤5 elimination half-lives, whichever is earlier, before enrollment
* Prior treatment with EGFR-targeted bispecific T cell engager or CAR-T cell therapy
* Prior treatment with CD3 engaging bispecific antibodies
* Clinically significant cardiovascular diseases
* Active clinically significant infection (bacterial, viral, fungal, mycobacteria, or other)
* On supplemental oxygen
* Any medical condition or clinical laboratory abnormality likely to interfere with assessment of safety or efficacy of study treatment DRUG: JANX008
Non-Small Cell Lung Cancer, Renal Cell Carcinoma, Squamous Cell Carcinoma of the Head and Neck, Colorectal Carcinoma, Small Cell Lung Cancer, Pancreatic Ductal Adenocarcinoma, Triple-Negative Breast Cancer, Colon, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Lung/Thoracic, Rectum
UT Southwestern
Phase 1/2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD) (FORTITUDE)
A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
Call 214-648-5005
studyfinder@utsouthwestern.edu, Jonathan.Thompson@UTSouthwestern.edu
Jaya Trivedi
All
18 Years to 65 Years old
Phase 1/Phase 2
NCT05747924
STU-2022-1177
Inclusion Criteria:
• FSHD1 or FSHD2 diagnosis confirmed by documented genetic testing (testing provided by Sponsor)
• Ambulatory and able to walk 10 meters (with or without assistive devices such as one cane, walking stick or braces)
• At least 1 muscle region suitable for biopsy (testing provided by Sponsor)
• Muscle weakness in both upper and lower body, as determined by Investigator
Exclusion Criteria:
• Diagnosed with congenital or infantile FSHD
• Pregnancy, intent to become pregnant within 9 months after last planned dose of Study Drug, or active breastfeeding
• Unwilling or unable to continue to comply with contraceptive requirements
• Body mass index (BMI) >35.0 kg/m2 at Screening
• History of muscle biopsy within 30 days of the screening biopsy or planning to undergo any nonstudy muscle biopsies over the duration of the study
• History of bleeding disorders, significant keloid, or other skin or muscle conditions (e.g., severe muscle wasting) that, in the opinion of the Investigator, makes the participant unsuitable for serial muscle biopsy
• Anticipated survival less than 2 years
• Blood or plasma donation within 16 weeks of Study Day 1
• Any contraindication to MRI
• Any abnormal lab values, conditions or diseases that, in the opinion of the investigator or Sponsor, would make the participant unsuitable for the study or could interfere with participation or completion of the study
• Treatment with any investigative medication within 1 month (or 5 half-lives of the drug, whichever is longer) of Screening
Drug: AOC 1020, Drug: Placebo
FSHD, FSHD1, FSHD2, FMD, FMD2, Fascioscapulohumeral Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy Type 1, Fascioscapulohumeral Muscular Dystrophy Type 2, Dystrophies, Facioscapulohumeral Muscular, Dystrophy, Facioscapulohumeral Muscular, Facioscapulohumeral Muscular Dystrophy 1, Facioscapulohumeral Muscular Dystrophy 2, Facio-Scapulo-Humeral Dystrophy, Atrophy, Facioscapulohumeral, Atrophies, Facioscapulohumeral, Facioscapulohumeral Atrophy, Muscular Dystrophies, Muscular Dystrophy, Facioscapulohumeral, FSH Muscular Dystrophy, Landouzy Dejerine Dystrophy, Landouzy-Dejerine Muscular Dystrophy, Dystrophies, Landouzy-Dejerine, Dystrophy, Landouzy-Dejerine, Landouzy-Dejerine Syndrome, Muscular Dystrophy, Landouzy Dejerine, Progressive Muscular Dystrophy, FSH
FORTITUDE, Avidity, Avidity Biosciences, AOC 1020
UT Southwestern
ARTEMIS: Ravulizumab to Protect Patients With CKD From CSA-AKI and MAKE (ARTEMIS)
The primary objective of this study is to assess the efficacy of a single dose of ravulizumab IV compared with placebo in reducing the risk of the clinical consequences of AKI (MAKE) at 90 days in adult participants with CKD who undergo non-emergent cardiac surgery with CPB.
Call 214-648-5005
studyfinder@utsouthwestern.edu, salina.shrestha@utsouthwestern.edu
Michael Jessen
All
18 Years to 90 Years old
Phase 3
NCT05746559
STU-2023-0186
Inclusion Criteria:
• Participant weighs ≥ 30 kg
• Planned non-emergent sternotomy with CPB procedure for the following surgeries:
• Multi-vessel CABG
• Valve replacement or repair; ascending aorta surgery permitted if combined with aortic valve replacement/repair
• Combined CABG and valve surgery; inclusion of single-vessel CABG when combined with valve replacement/repair is permitted
• Known CKD for at least 90 days (CKD Stage 3A, 3B, or 4)
Exclusion Criteria:
• Emergency or salvage cardiac surgery is expected at screening or randomization, as assessed by the Investigator.
• Single-vessel CABG without valve surgery is planned.
• Off-pump surgery is planned (eg, surgery without CPB).
• Recipient of a solid organ or bone marrow transplantation.
• Cardiogenic shock, hemodynamic instability, use of intra-aortic balloon pump, extracorporeal membrane oxygenation, or left ventricular assist device within 72 hours of randomization.
• Active systemic bacterial, viral, or fungal infection within 14 days prior to randomization.
• History of unexplained, recurrent infection.
• Any use of KRT or presence of AKI within 30 days of randomization
• Use of any complement inhibitors, or plasmapheresis or plasma exchange within the year prior to Screening, or planned use during the course of the study.
• Participant is not willing to be vaccinated against N meningitidis or is unwilling to receive prophylactic treatment with appropriate antibiotics, if needed
• History of or unresolved N meningitidis infection.
Drug: Placebo, Drug: Ravulizumab
Chronic Kidney Disease, CKD, Cardiac Disease, Cardiopulmonary Bypass, Kidney
Chronic Kidney Disease, CKD, Cardiac Disease, cardiopulmonary bypass
UT Southwestern
Neutrophil and Monocyte Deactivation Via the SeLective CytopheretIc Device - A Randomized Clinical Trial in Acute Kidney Injury (NEUTRALIZE-AKI)
This randomized, controlled, pivotal study is intended to determine whether up to ten sequential 24-hour treatments with the Selective Cytopheretic Device (SCD) will improve survival in patients with Acute Kidney Injury (AKI) requiring continuous kidney replacement therapy (CKRT) when compared to CKRT alone (standard of care). This study is further intended to determine whether SCD therapy will reduce the duration of maintenance dialysis secondary to AKI. This study will enroll approximately 200 subjects across 30 US sites. Participants will be patients in an intensive care unit (ICU) setting with a diagnosis of AKI requiring CKRT.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Bandana.Poudel@UTSouthwestern.edu
Tamim Hamdi
ALL
18 Years and over
NA
NCT05758077
STU-2024-0432
Inclusion Criteria:
* Admitted to an ICU requiring CKRT:
• Must have AKI stage 2 or greater at the time of CKRT initiation.
• Must have been on CKRT for at least 12 hours but no greater than 48 hours at the time of enrollment. * At least 18 years of age but not older than 80 at the time of enrollment. * One additional life-threatening organ dysfunction present. * Acceptable vascular access for CKRT to include adequate lumen size and length of catheters. * Initial (non-binding) commitment to maintaining current level of care for at least 96 hours. * C-Reactive Protein \>3.5 mg/dl.
Exclusion Criteria:
* Not expected to survive next 24 hours.
* Anticipated transition to comfort measures or hospice in next 4 days.
* Terminal condition whereby the patient is not expected to survive 28 days or any condition in which therapy is regarded as futile by the PI.
* Advanced malignancy which is actively being treated or may be treated with palliative chemotherapy or radiation.
* ICU hospitalization \> 14 days during this hospital admission (to include days spent at ICU of an outside hospital) at the time of screening.
* Active COVID-19 infection with a primary admission diagnosis of COVID-19.
* Chronic use of ventricular assist devices.
* ESRD requiring chronic kidney replacement therapy.
* History of CKD (greater than Stage 3).
* AKI stage 0 or stage 1 at the time of CKRT initiation.
* Non-ATN AKI diagnosis. We intend on relying on local nephrology subspecialty expertise to reasonably exclude non-ATN diagnoses based on clinical suspicions combined with prespecified objective criteria. If there is a reasonable suspicion that the subject has non-ATN AKI based on this, they will be excluded from the trial.
* Acute coronary syndromes, acute stroke, or acute major vascular compromise requiring medical or surgical interventions within 48 hours of randomization.
* Active hemorrhage requiring blood transfusions at the time of screening.
* Acute on Chronic Liver Failure.
* Suspicion of hepato-renal syndrome.
* Presence of any solid organ transplant at any time prior to admission.
* Severe burns with a modified Baux score \> 100 (%TBSA+Age+17 for Inhalation Injury).
* Bone marrow transplant within the last year.
* Chronic immunosuppression with an average of \>20 mg/day of prednisone or other steroid sparing immunosuppressants for the past 30 days prior to hospital admission.
* Individuals who have a history of primary or secondary immune disorders including, but not limited to, HIV or AIDS.
* Dry weight of \>150kg.
* Platelet count \<15,000/mm3.
* Patient is a prisoner or member of a vulnerable population.
* Patient is pregnant or breast feeding.
* Concurrent enrollment in another interventional clinical trial for an investigational drug or device.
* Need for plasmapheresis. DEVICE: Selective Cytopheretic Device, OTHER: Standard of Care
Acute Kidney Injury
continuous kidney replacement therapy, continuous renal replacement therapy, acute kidney injury, organ failure, inflammation, dialysis, acute tubular necrosis
UT Southwestern
A Study to Investigate the Safety and Tolerability of Ziftomenib in Combination With Venetoclax/Azacitidine, Venetoclax, 7+3, or 7+3+Quizartinib in Patients With AML
Ziftomenib is an investigational drug in development for the treatment of patients with acute myeloid leukemia (AML) with certain genetic alterations. This protocol has 3 separate arms that will investigate the benefits and risks of adding ziftomenib to standard-of-care (SOC) drug treatments in patients who have AML with certain genetic mutations. Both newly diagnosed and relapsed refractory patients with AML will be assigned to different cohorts based on specific study criteria and physician discretion. The purpose of this study is to assess the safety, tolerability, and early signs of efficacy of ziftomenib in combination with SOC drugs to treat AML.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Yazan Madanat
ALL
18 Years and over
PHASE1
NCT05735184
STU-2023-0858
Key
Inclusion Criteria:
* Patients must have a documented NPM1 mutation or KMT2A rearrangement and have either newly diagnosed or relapsed/refractory AML
* Those intending treatment with intensive chemotherapy in Arm C should be NPM1-m and FLT3-ITD+ with an allelic ratio ≥0.05 and eligible for FLT3-targeted treatment
* Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2
* Adequate liver, renal, and cardiac function according to protocol defined criteria
* A female of childbearing potential must agree to use adequate contraception as well as a double barrier method from the time of screening through 180 days following the last dose of study intervention. A male of childbearing potential must agree to use abstinence or use a double barrier method of contraception from the time of screening through 180 days following the last dose of study intervention
* Female patients of childbearing potential who receive quizartinib in Arm C should use a highly effective method of contraception during quizartinib treatment and for 7 months after the last dose
Key Exclusion Criteria:
* Diagnosis of either acute promyelocytic leukemia or blast phase chronic myeloid leukemia
* Known history of BCR-ABL alteration
* Advanced malignant hepatic tumor
* Administration of live attenuated vaccines within 14 days prior to, during, or after treatment until B-cell recovery
* Active central nervous system (CNS) involvement by AML.
* Clinical signs/symptoms of leukostasis or WBC \> 25,000 / microliter. Hydroxyurea and/or leukapheresis and/or up to 2 doses of cytarabine if used per institutional SOC for control of leukocytosis are permitted to meet this criterion
* Not recovered to Grade ≤1 (NCI-CTCAE v5.0) from all nonhematological toxicities except for alopecia
* Known clinically active human immunodeficiency virus, active hepatitis B or active hepatitis C infection
* For newly diagnosed cohorts: received prior chemotherapy for leukemia, except hydroxyurea and/or leukapheresis and/or up to 2 doses of cytarabine per institutional standards to control leukocytosis, or prior treatment with all-transretinoic acid for initially suspected acute promyelocytic leukemia
* For relapsed/refractory cohorts: received chemotherapy, immunotherapy, radiotherapy, or any ancillary therapy that is considered to be investigational \< 14 days prior to the first dose of ziftomenib or within 5 drug half-lives prior to the first dose of study drug
* Uncontrolled intercurrent illness including, but not limited to, cardiac illness as defined in the protocol
* Mean QT interval corrected for heart rate by Fredericia's formula (QTcF)
* Arm A and Arm B: \>480 ms on triplicate ECGs
* Arm C: \>450 ms on triplicate ECGs
* Uncontrolled infection
* Women who are pregnant or lactating
* An active malignancy and currently receiving chemotherapy for that malignancy or disease that is uncontrolled/progressing
* Patients who have active GVHD requiring \>0.5 mg/kg prednisone or any new or increase in immunosuppressants in the prior 2 weeks for GVHD treatment DRUG: Ziftomenib, DRUG: Venetoclax, DRUG: Azacitidine, DRUG: Daunorubicin, DRUG: Cytarabine, DRUG: Quizartinib
Acute Myeloid Leukemia, Mixed Lineage Leukemia Gene Mutation, Refractory AML, AML With Mutated NPM1, Acute Myeloid Leukemia Recurrent, Acute Myeloid Leukemia, in Relapse, NPM1 Mutation, KMT2Ar, Myeloid Sarcoma, Nucleophosmin 1-mutated Acute Myeloid Leukemia, Myeloid and Monocytic Leukemia
Leukemia, Myeloid, AML, Hematological malignancy, KMT2A, NPM1, Menin, Acute Leukemia, Newly diagnosed AML, Untreated AML, venetoclax, cytarabine, daunorubicin, KMT2A-r, NPM1 mutation, Refractory AML, Acute Myeloid Leukemia, in Relapse, quizartinib
UT Southwestern
Efficacy, Safety, and Tolerability of Two Administrations of COMP360 in Participants With TRD
Efficacy, Safety, and Tolerability of two administrations of COMP360 in participants with treatment-resistant depression (TRD)
Call 214-648-5005
studyfinder@utsouthwestern.edu, Ann.House@UTSouthwestern.edu
Madhukar Trivedi
All
18 Years and over
Phase 3
NCT05711940
STU-2023-0052
Key
• Aged ≥18 years at Screening
• Major depression without psychotic features (single or recurrent episode as informed by the Diagnostic and Statistical Manual of Mental Disorders, 5th edition [DSM-5])
• If the current major depressive episode is the participant's first lifetime episode of depression, the length of the current episode must be ≥3 months and ≤2 years at Screening
• MADRS total score ≥20 at Screening and Baseline to ensure at least moderate severity of depression
• TRD, defined as failure to respond to an adequate dose and duration of two, three, or four different pharmacological treatments for the current episode as determined through the Massachusetts General Hospital Antidepressant Treatment Response Questionnaire (MGH-ATRQ) and using the supplementary advice on additional antidepressants not included in MGH-ATRQ
• At Screening, agreement to discontinue all prohibited medications Key
• Prior or ongoing bipolar disorder, any psychotic disorder, including schizophrenia, schizophreniform disorder, schizoaffective disorder, brief psychotic disorder (unless substance induced or due to a medical condition), antisocial personality disorder as assessed by a structured clinical interview (MINI 7.0.2)
• Lifetime paranoid, schizoid, schizotypal, histrionic, narcissistic personality disorder, or any ongoing serious psychiatric comorbidity based on medical history and clinical judgement
• Borderline personality disorder as demonstrated by medical history or the Mini International Neuropsychiatric Interview Plus (MINI plus) - borderline personality disorder module
• Ongoing post-traumatic stress disorder, obsessive-compulsive disorder, or anorexia nervosa as assessed by medical history and a structured clinical interview (MINI
• 0.2)
• Psychiatric inpatient within the past 12 months prior to Screening
• Use of electroconvulsive therapy, deep brain stimulation, or vagus nerve stimulation during the current depressive episode
• Transcranial magnetic stimulation within the past six months prior to Screening
• Current enrolment in a psychological therapy programme that will not remain stable for the duration of the study. Psychological therapies cannot have been initiated within 30 days prior to Screening
• Exposure to COMP360 psilocybin therapy prior to Screening
Inclusion Criteria:
• Aged ≥18 years at Screening
• Major depression without psychotic features (single or recurrent episode as informed by the Diagnostic and Statistical Manual of Mental Disorders, 5th edition [DSM-5])
• If the current major depressive episode is the participant's first lifetime episode of depression, the length of the current episode must be ≥3 months and ≤2 years at Screening
• MADRS total score ≥20 at Screening and Baseline to ensure at least moderate severity of depression
• TRD, defined as failure to respond to an adequate dose and duration of two, three, or four different pharmacological treatments for the current episode as determined through the Massachusetts General Hospital Antidepressant Treatment Response Questionnaire (MGH-ATRQ) and using the supplementary advice on additional antidepressants not included in MGH-ATRQ
• At Screening, agreement to discontinue all prohibited medications Key
Exclusion Criteria:
• Prior or ongoing bipolar disorder, any psychotic disorder, including schizophrenia, schizophreniform disorder, schizoaffective disorder, brief psychotic disorder (unless substance induced or due to a medical condition), antisocial personality disorder as assessed by a structured clinical interview (MINI 7.0.2)
• Lifetime paranoid, schizoid, schizotypal, histrionic, narcissistic personality disorder, or any ongoing serious psychiatric comorbidity based on medical history and clinical judgement
• Borderline personality disorder as demonstrated by medical history or the Mini International Neuropsychiatric Interview Plus (MINI plus) - borderline personality disorder module
• Ongoing post-traumatic stress disorder, obsessive-compulsive disorder, or anorexia nervosa as assessed by medical history and a structured clinical interview (MINI
• 0.2)
• Psychiatric inpatient within the past 12 months prior to Screening
• Use of electroconvulsive therapy, deep brain stimulation, or vagus nerve stimulation during the current depressive episode
• Transcranial magnetic stimulation within the past six months prior to Screening
• Current enrolment in a psychological therapy programme that will not remain stable for the duration of the study. Psychological therapies cannot have been initiated within 30 days prior to Screening
• Exposure to COMP360 psilocybin therapy prior to Screening
Drug: Psilocybin
Treatment Resistant Depression
psilocybin
UT Southwestern
Janus Kinase (JAK) Inhibitors to Preserve C-Peptide Production in New Onset Type 1 Diabetes (T1D)
A multi-center, placebo-controlled, double blind, 1:1:1 randomized control clinical trial testing two different JAK Inhibitors abrocitnib, ritlecitinib, and placebo in subjects with recent onset Stage 3 Type 1 Diabetes within 100 days of diagnosis.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Michelle.Murphy@UTSouthwestern.edu
Perrin White
ALL
12 Years to 35 Years old
PHASE2
NCT05743244
STU-2023-1068
Inclusion Criteria:
• Provide informed consent or assent as appropriate and, if \< 18 years of age have a parent or legal guardian provide informed consent
• Age 12-35 years (both inclusive) at the time of signing informed consent and assent
• Diagnosis of T1D within 100 days of the baseline visit (V0).
• Positive for at least one islet cell autoantibody; Glutamate decarboxylase (GAD)65A, mIAA (if obtained within 10 days of the onset of insulin therapy), IA-2A, ICA, or ZnT8A
• Stimulated C-peptide of ≥0.2 pmol/mL measured during mixed-meal tolerance test (MMTT) conducted at least 21 days from diagnosis of diabetes
• HbA1c ≤ 10 %
• Body weight ≥ 35kg at screening
• Willing to comply with intensive diabetes management and wear a Continuous Glucose Monitoring Device (CGM)
• Participants who are Cytomegalovirus (CMV) and/or Epstein-Barr virus (EBV) seronegative at screening must be CMV and/or EBV Polymerase chain reaction (PCR) negative within 30 days of randomization and may not have had signs or symptoms of a CMV and/or EBV-compatible illness lasting longer than 7 days within 30 days of the baseline visit (V0).
• Participants who are CMV and/or EBV seropositive at screening must be CMV PCR negative and/or EBV PCR \<2,000 IU/mL and must have no signs or symptoms of acute infection at the time of the baseline visit (V0).
• Be up to date on recommended vaccinations based on age of participants\*
• Participants are required to receive killed influenza vaccination at least 2 weeks prior to the baseline visit (V0) when vaccine for the current or upcoming flu season is available. Enrollment must be delayed at least 4 weeks from administration of a killed vaccine other than influenza and COVID-19 and 6 weeks from a live vaccination. Live vaccinations and non-live vaccinations (other than influzena and COVID-19) should not be given while on study drug and be postponed at least 3 months after the last dose of study drug.
• If participant is female with reproductive potential, she must have a negative pregnancy test at screening and be willing to avoid pregnancy using a highly-effective contraceptive method for the duration of the study
• Males of reproductive age must use a highly-effective contraceptive method during the treatment phase and for 3 months following last dose of study drug * For COVID-19 vaccination, all participants will be strongly encouraged to be up-to-date with COVID-19 vaccine (s) as indicated by country-specific guidelines at least 2 weeks prior to the baseline visit (V0).
Exclusion Criteria:
• Current or ongoing use of non-insulin pharmaceuticals or medication that affect glycemic control or glucose homeostasis within 7 days prior to screening or any prohibited concomitant medication listed in section 4.8
• Untreated hypothyroidism or active Graves' disease
• Concurrent treatment with other immunosuppressive agents (including biologics or steroids), other than inhaled or topical glucocorticoids
• Active acute or chronic infection requiring treatment with oral antibiotics, antivirals, antiparasitics, antiprotozoals, or antifungals within 1 month prior to Day 0 or superficial skin infection within 1 week prior to Day 0
• Active acute or chronic infection requiring treatment with intravenous therapy (IV) within a minimum 1 month prior to Day 0 a. Specific cases should be reviewed by Infectious Disease Committee prior to enrollment
• Have active signs or symptoms of acute infection at the time of the baseline visit (V0).
• Significant trauma or major surgery within 1 month of signing informed consent.
• Considered in imminent need for surgery or with elective surgery scheduled to occur during the study
• History of disseminated herpes zoster or disseminated herpes simplex or a recurrent (more than one episode of) localized, dermatomal herpes zoster
• Have evidence of prior or current tuberculosis infection as assessed by Purified Protein Derivative (PPD), interferon gamma release assay (IGRA) or by history
• Have evidence of current or past HIV or Hepatitis B infection
• Have evidence of active Hepatitis C infection
• Have current, confirmed COVID-19 infection
• Current or history of Deep vein thrombosis (DVT), Pulmonary embolism (PE), or other thromboembolic events or history of inherited coagulopathies
• First degree relative with a history of unprovoked venous thromboembolism (i.e. without known underlying cause such as trauma, surgery, immobilization, prolonged travel, pregnancy, hormone use, or plaster cast), which suggests that a participant may be at increased risk of inherited coagulation disorder
• Any present malignancies or history of malignancy, other than a successfully treated nonmelanoma skin cancer
• History of any lymphoproliferative disorder such as EBV-related lymphoproliferative disorder, history of lymphoma, history of leukemia, or signs and symptoms suggestive of current lymphatic or lymphoid disease
• Known or suspected polymorphism in the Cytochrome P450 2C19 (CYP2C19 gene, resulting in classification as a poor CYP2C19 metabolizer).
• Have renal impairment (eGFR\< 60 mL/min)
• Currently on anti-platelet therapies, excluding low dose aspirin
• One or more screening laboratory values as stated
• Neutrophils \< 1,500 /μL
• Lymphocytes \< 800 /μL
• Platelets \< 150,000 / μL
• Hemoglobin \< 6.2 mmol/L (10.0 g/dL)
• Potassium \> 5.5 mmol/L or \<3.0 mmol/L
• Sodium \> 150mmol/L or \< 130mmol/L
• AST or ALT ≥ 2.5 times the upper limit of normal
• Bilirubin ≥ 1.5 times upper limit of normal unless diagnosed with Gilbert's syndrome
• LDL \>160 mg/dL
• Vaccination with a live virus within the last 6 weeks and killed vaccine within 4 weeks (except 2 weeks for flu vaccine and COVID vaccine)
• Be currently pregnant or lactating or anticipate becoming pregnant during the study
• Male participants able to father children and female participants of childbearing potential who are unwilling or unable to use 2 effective methods (at least 1 highly effective method) of contraception, including abstinence, as outlined in this protocol for the duration of the study and for at least 3 months after the last dose of investigational product
• Be currently participating in another T1D treatment study
• Have hearing loss with progression over the previous 5 years, or sudden hearing loss, or middle or inner ear disease such as otitis media, cholesteatoma, Meniere's disease, labyrinthitis, or other auditory condition that is considered acute, fluctuating, or progressive
• Acute coronary syndrome (e.g., myocardial infarction, unstable angina pectoris) and any history of cerebrovascular disease within 24 weeks before screening; Heart failure NYHA (New York Heart Association) III, NYHA IV
• ANY of the following conditions at screening: a. Screening 12-lead electrocardiogram (ECG) that demonstrates: i. Clinically significant abnormalities requiring treatment (eg, acute myocardial infarction, serious tachy- or brady-arrhythmias) or indicating serious underlying heart disease (eg, cardiomyopathy, Wolff-Parkinson- White syndrome); ii. Confirmed QT corrected using Fridericia's correction factor (QTcF) prolongation (\>450 milliseconds). b. Long QT Syndrome, a family history of Long QT Syndrome, or a history of Torsades de Pointes (TdP).
• History of chronic alcohol abuse or intravenous drug abuse or other illicit drug abuse within 2 years prior to screening
• Current or past use of tobacco or nicotine containing products more than the equivalent of 5 cigarettes per day
• Participant is the investigator or any sub-investigator, research assistant, pharmacist, study coordinator, other staff or relative thereof directly involved in the conduct of the trial
• Have any complicating medical issues or abnormal clinical laboratory results that may interfere with study conduct, or cause increased risk
• Any condition that in the investigator's opinion may adversely affect study participation or may compromise the study results
DRUG: Abrocitinib 200 MG Oral Tablet, DRUG: Ritlecitinib, DRUG: Placebo
Diabetes Mellitus, Type 1, Pancreas
TrialNet, T1D
UT Southwestern; Children’s Health
A Study of Enzalutamide Plus the Glucocorticoid Receptor Antagonist Relacorilant Versus Placebo for Patients With High-risk Localized Prostate Cancer
Researchers conducting this study hope to learn about the safety and effectiveness of combining two study drugs, relacorilant and enzalutamide, plus androgen deprivation therapy (ADT), also known as hormone therapy. This study is for individuals who have been diagnosed with advanced, high-risk prostate cancer and standard therapies available to treat your disease have not been effective. Participation in this research will last about 3 years and 9 months.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Kevin Courtney
MALE
18 Years to old
PHASE2
NCT05726292
STU-2024-0040
Inclusion Criteria:
• Histologically or cytologically confirmed prostatic adenocarcinoma without primary small cell histology
• Localized disease: * Surgical resectability must be documented prior to enrollment * No evidence of distant metastatic disease on abdominopelvic imaging, bone imaging * Enlarged lymph nodes below the iliac bifurcation (clinical stage N1) is allowed * Either cross-sectional abdominopelvic imaging + technetium bone scan or PSMA PET imaging will be acceptable to rule out distant metastatic disease
• High or very high-risk disease (https://www.nccn.org/professionals/physician_gls/pdf/prostate.pdf) as defined by having one or more of the following: * Clinical T3a or higher * Histologic Grade Group 4 or 5 * PSA \>20
• Eastern Cooperative Oncology Group performance status ≤ 1 (Appendix A)
• Total serum testosterone 100 ng/dL
• Patients must have normal hepatic function as defined below: * Total bilirubin \<1.5 X the upper limit of normal (note that in subjects with Gilbert's syndrome, if total bilirubin is \>1.5 X ULN, measure direct and indirect bilirubin. If direct bilirubin is ≤1.5 X ULN, the subject may be eligible) * AST(SGOT)/ALT(SGPT) \<2.5 X institutional upper limit of normal * Albumin 3.0 g/dL
• Patients must have normal bone marrow function as defined below: * Platelet count (plt) 100,000 /L * Hemoglobin (Hgb) 10 g/dL * Absolute neutrophil count (ANC) 1500
• Patients must have adequate renal function as defined below: • glomerular filtration rate (GFR) 30 mL/min
• Ability to understand and the willingness to sign a written informed consent document.
• Patients with active diabetes mellitus on glucose lowering medications are eligible provided they agree to and are able to self-monitor daily blood glucose levels due to potential risk of lowering glucose levels on relacorilant.
• Male patient and his female partner who is of childbearing potential must use 2 acceptable methods of birth control (one of which must include a condom as a barrier method of contraception) starting at screening and continuing throughout the study period and for 3 months after final study drug administration. Two acceptable methods of birth control thus include the following: * Condom (barrier method of contraception) AND * One of the following is required:
• Established use of oral, injected or implanted hormonal method of contraception by the female partner;
• Placement of an intrauterine device (IUD) or intrauterine system (IUS) by the female partner;
• Additional barrier method: Occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/suppository by the female partner;
• Tubal ligation in the female partner;
• Vasectomy or other procedure resulting in infertility (e.g., bilateral orchiectomy), for more than 6 months.
• 2 Exclusion Criteria
• Therapy with ANY hormonal therapy for prostate cancer (prior 5-alpha-reductase inhibitors for benign prostate disease is allowed but must be discontinued prior to study initiation).
• Inability to swallow capsules or known gastrointestinal malabsorption.
• History of other malignancies, with the exception of: adequately treated non-melanoma skin cancer, adequately treated superficial bladder cancer, stage 1 or 2 malignancies that are without evidence of disease, or other cancers curatively treated with no evidence of disease for \> 5 years from enrollment.
• Blood pressure that is not controlled despite \> 2 oral agents (SBP \>160 and DBP \>90 documented during the screening period with no subsequent blood pressure readings \>160/100).
• History of seizure disorder or active use of anticonvulsants. Medications used to treat neuropathic pain such as gabapentin or pregabalin are allowed.
• Serious inter-current infections or non-malignant medical illnesses that are uncontrolled.
• Active psychiatric illness/social situations that would limit compliance with protocol requirements.
• New York Heart Association (NYHA) class II, class III, or IV congestive heart failure (any symptomatic heart failure).
• Concurrent therapy with strong inhibitors of Cytochrome P450 3A4 or CYP2C8 due to concerning possible drug-drug interactions.
• Concurrent therapy with strong inducers of Cytochrome P450 3A4 due to concerning possible drug-drug interactions.
• Presence of concurrent medical conditions requiring systemic glucocorticoids for immunosuppression (e.g. autoimmune diseases, organ transplantation) that is active and has required glucocorticoids in the last 6 months.
DRUG: Relacorilant, DRUG: Enzalutamide, OTHER: Placebo (Sugar Pill), OTHER: Androgen Deprivation Therapy, PROCEDURE: Radical Prostatectomy
Prostate Cancer, Prostate Adenocarcinoma, Prostate
prostate cancer
UT Southwestern
Study of Lurbinectedin Monotherapy in Pediatric and Young Adult Participants With Relapsed/Refractory Ewing Sarcoma (EMERGE 101)
This study is conducted in two phases. The phase 1 portion of the study evaluates the safety, tolerability, pharmacokinetics (PK), recommended phase 2 dose (RP2D), and effectiveness of lurbinectedin monotherapy in pediatric participants with previously treated solid tumors. This is followed by the phase 2 portion, to further assess the effectiveness and safety in pediatric and young adult participants with recurrent/refractory Ewing sarcoma.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Avanthi Shah
ALL
2 Years to 30 Years old
PHASE1
NCT05734066
STU-2023-0156
Key
Inclusion Criteria:
Age
* Participant must meet the following age requirements at the time the informed consent form (ICF) (and assent form, if applicable) is signed:
* Phase 1 Part 1: participants must be ≥ 2 to \< 18 years of age.
* Phase 1 Part 2: participants must be ≥ 2 to ≤ 30 years of age.
* Phase 2: participants must be ≥ 2 to ≤ 30 years of age.
Type of Participant and Disease Characteristics
* Participant has a confirmed solid tumor
* The participant has a Lansky/Karnofsky performance status score of ≥ 50%.
* The participant has adequate liver function, evidenced by the following laboratory values:
* Aspartate aminotransferase (AST), alanine aminotransferase (ALT) ≤ 2.5 × upper limit of normal (ULN).
* Total bilirubin ≤ 1.5 × institutional ULN (with the exception of participants with Gilbert's syndrome who must have bilirubin \< 3 × institutional ULN).
* The participant has adequate bone marrow function, evidenced by the following:
* Absolute neutrophil count (ANC) ≥ 1.0 × 109/L (independent of growth factor support within 1 week of screening laboratories).
* Platelets ≥ 100 × 109/L (without platelet transfusion within previous 7 days of screening laboratories).
* Hemoglobin ≥ 8 g/dL (note: may have been transfused).
* The participant has an adequate renal function:
* Calculated creatinine clearance (use Cockcroft-Gault formula for participants ≥ 18 years; Schwartz equation for participants \< 18 years) ≥ 60 mL/min.
* The participant has an adequate cardiac function:
* Left ventricular ejection fraction or shortening fraction per institutional norm ≥ institutional lower level of normal.
* The participant has creatine phosphokinase ≤ 2.5 × institutional ULN.
Weight
* The participant has body weight ≥ 15 kg.
Sex and Contraceptive/Barrier Requirements
Male participants:
Male participants are eligible to participate if they agree to the following during the study intervention period and for at least 4 months after the last dose of study intervention:
* Refrain from donating sperm.
PLUS, either:
* Be abstinent from heterosexual intercourse as their preferred and usual lifestyle (abstinent on a long-term and persistent basis) and agree to remain abstinent.
OR
* Must agree to use contraception/barrier as detailed below:
* Agree to use a male condom with female partner and use of an additional highly effective contraceptive method with a failure rate of \< 1% per year when having sexual intercourse with a Woman of childbearing potential (WOCBP) who is not currently pregnant.
* Note: male participants who are azoospermic (vasectomized or due to a medical cause) are still required to follow the protocol-specified contraception/barrier criteria.
Female participants:
A female participant is eligible to participate if she is not pregnant or breastfeeding, and one of the following conditions applies:
* Is a Woman of nonchildbearing potential (WONCBP). OR
* Is a WOCBP and using an acceptable contraceptive method during the study intervention period (at least 7 months after the last dose of study intervention). The investigator should evaluate the potential for contraceptive method failure (eg, noncompliance, recently initiated) in relationship to the first dose of study intervention.
* A WOCBP must have a negative highly sensitive pregnancy test (urine or serum as required by local regulations) within 7 days before the first dose of study intervention.
* If a urine test cannot be confirmed as negative (eg, an ambiguous result), a serum pregnancy test is required. In such cases, the participant must be excluded from participation if the serum pregnancy result is positive.
* Additional requirements for pregnancy testing during and after study intervention.
* The investigator is responsible for review of medical history, menstrual history, and recent sexual activity to decrease the risk for inclusion of a woman with an early undetected pregnancy.
Informed Consent
* Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.
Key Exclusion Criteria:
Medical Conditions
* corrected QT interval (QTc) prolongation defined as a QTc ≥ 470 ms using the Bazett formula.
* Known symptomatic Central nervous system (CNS) metastases requiring steroids. Participants with previously diagnosed CNS metastases are eligible if they have completed their treatment and have recovered from the acute effects of radiation therapy or surgery prior to enrollment, have discontinued high dose steroid treatment for these metastases for at least 2 weeks, and are neurologically stable (physiologic doses of steroids and short courses of steroids for other indications are acceptable).
* Persisting toxicity related to prior therapy; however, alopecia, sensory neuropathy, hypothyroidism, and rash Grade ≤ 2 are acceptable, and other Grade ≤ 2 adverse events (AEs) not constituting a safety risk based on the investigator's judgement are acceptable.
* An uncontrolled intercurrent illness including but not limited to ongoing or active infection requiring antibiotic, antifungal, or antiviral therapy, symptomatic heart failure, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
* Any other major illness that, in the investigator's judgment, could substantially increase the risk associated with participation in this study.
* Any other diseases, metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates the use of an investigational drug or that may affect the interpretation of the results or render the participant at high-risk for treatment complications.
Prior/Concomitant Therapy
* Received prior treatment with lurbinectedin or trabectedin.
* Received prior treatment with any investigational product within 4 weeks of first infusion of study intervention. Observational studies are permitted.
* Received live or live attenuated vaccines within 4 weeks of the first dose of study treatment or plans to receive live vaccines during study participation. Administration of inactive vaccines or messenger ribonucleic acid (mRNA) vaccines (for example, inactivated influenza vaccines or COVID-19 vaccines) are allowed.
* Had major surgery ≤ 4 weeks or radiation therapy ≤ 2 weeks prior to enrollment unless fully recovered. Prior palliative radiotherapy is permitted, provided it was completed at least 2 weeks prior to participant enrollment.
* Received prior allogeneic bone marrow transplantation or solid organ transplant.
* Received chemotherapy ≤ 3 weeks prior to start of study intervention.
Diagnostic Assessments
* Hepatitis B virus (HBV) or Hepatitis C virus (HCV) infection at screening (positive HBV surface antigen or Polymerase chain reaction (PCR) test for HCV RNA if HCV antibody test is positive).
* Human immunodeficiency infection at screening (positive anti-HIV antibody).
Other Exclusions
* Has a known or suspected hypersensitivity to any of the components of the study intervention.
* The participant or parent(s)/guardian(s) is/are unable to comply with the study visit schedule and other protocol requirements, in the opinion of the investigator DRUG: Lurbinectedin
Refractory Ewing Sarcoma, Relapsed Ewing Sarcoma, Ewing Sarcoma, Sarcoma
Solid Tumors, lurbinectedin, ewing's sarcoma, soft tissue sarcoma, sarcomas
Children’s Health
DEFIANCE: RCT of ClotTriever System Versus Anticoagulation In Deep Vein Thrombosis (DEFIANCE)
This study is a prospective, multicenter, randomized controlled trial of an interventional strategy using the ClotTriever System to achieve and maintain vessel patency (ClotTriever Intervention Arm) versus conservative medical management using anticoagulation therapy alone (Conservative Medical Management Arm) in the treatment of subjects with symptomatic unilateral iliofemoral DVT. The study will collect data on demographics, comorbidities, details from the DVT diagnosis and treatment, and clinical outcomes through the 6-month follow up visit.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Charlton.Starcke@UTSouthwestern.edu
Moaz Choudhary
All
18 Years and over
N/A
NCT05701917
STU-2023-0117
Inclusion Criteria
• Age ≥ 18 years
• Proximal lower extremity unilateral DVT involving at least the common femoral, external iliac, or common iliac veins, alone or in combination
• Symptom onset within 12 weeks of enrollment in the study
• Significant symptoms, as defined by a Villalta score > 9
• Willing and able to provide informed consent Exclusion Criteria
• Bilateral iliofemoral DVT
• Prior venous stent in the target venous segment
• IVC aplasia/hypoplasia or other congenital anatomic anomalies of the IVC or iliac veins
• IVC filter in place at the time of enrollment
• Limb-threatening circulatory compromise (e.g., phlegmasia)
• Clot in transit including IVC thrombus presenting as extension of >2cm into the IVC from the CIV
• Symptomatic PE with right heart strain where the physician judges that a DVT intervention is inappropriate at this time.
• Inability to be a candidate for intervention due to medical or technical reasons based on physician judgement
• Severe allergy, hypersensitivity to, or thrombocytopenia from heparin
• Severe allergy to iodinated contrast agents that cannot be mitigated
• Hemoglobin < 8.0 g/dL, INR > 1.7 before warfarin was started, or platelets < 50,000/µl which cannot be corrected prior to enrollment
• Severe renal impairment (estimated GFR < 30 ml/min) in patients who are not yet on dialysis
• Inability to provide therapeutic anticoagulation per Investigator discretion
• Uncontrolled severe hypertension on repeated readings (systolic > 180mmHg or diastolic > 105mmHg)
• Recently (< 30 days) had DVT interventional procedure
• Subject is participating in another study that may interfere with this study
• Life expectancy < 6 months or chronic non-ambulatory status
• Known hypercoagulable states that, in the opinion of the Investigator, cannot be medically managed throughout the study period
• Subject has any condition for which, in the opinion of the Investigator, participation would not be in the best interest of the subject (e.g., contraindication to use of ClotTriever per local approved labeling, compromise the well-being or that could prevent, limit, or confound the protocol-specified assessments)
• Subject has previously completed or withdrawn from this study
• Patient unwilling or unable to conduct the follow up visits per protocol
• Age ≥ 18 years
• Proximal lower extremity unilateral DVT involving at least the common femoral, external iliac, or common iliac veins, alone or in combination
• Symptom onset within 12 weeks of enrollment in the study
• Significant symptoms, as defined by a Villalta score > 9
• Willing and able to provide informed consent Exclusion Criteria
• Bilateral iliofemoral DVT
• Prior venous stent in the target venous segment
• IVC aplasia/hypoplasia or other congenital anatomic anomalies of the IVC or iliac veins
• IVC filter in place at the time of enrollment
• Limb-threatening circulatory compromise (e.g., phlegmasia)
• Clot in transit including IVC thrombus presenting as extension of >2cm into the IVC from the CIV
• Symptomatic PE with right heart strain where the physician judges that a DVT intervention is inappropriate at this time.
• Inability to be a candidate for intervention due to medical or technical reasons based on physician judgement
• Severe allergy, hypersensitivity to, or thrombocytopenia from heparin
• Severe allergy to iodinated contrast agents that cannot be mitigated
• Hemoglobin < 8.0 g/dL, INR > 1.7 before warfarin was started, or platelets < 50,000/µl which cannot be corrected prior to enrollment
• Severe renal impairment (estimated GFR < 30 ml/min) in patients who are not yet on dialysis
• Inability to provide therapeutic anticoagulation per Investigator discretion
• Uncontrolled severe hypertension on repeated readings (systolic > 180mmHg or diastolic > 105mmHg)
• Recently (< 30 days) had DVT interventional procedure
• Subject is participating in another study that may interfere with this study
• Life expectancy < 6 months or chronic non-ambulatory status
• Known hypercoagulable states that, in the opinion of the Investigator, cannot be medically managed throughout the study period
• Subject has any condition for which, in the opinion of the Investigator, participation would not be in the best interest of the subject (e.g., contraindication to use of ClotTriever per local approved labeling, compromise the well-being or that could prevent, limit, or confound the protocol-specified assessments)
• Subject has previously completed or withdrawn from this study
• Patient unwilling or unable to conduct the follow up visits per protocol
Device: ClotTriever System, Drug: Commercially available/market approved anticoagulation medication including but not limited to: Heparin Sodium, Coumadin, Rivaroxaban, Apixaban, etc.
Venous Thromboembolism, Deep Venous Thrombosis, Post-Thrombotic Syndrome
Venous Thromboembolism, Deep Venous Thrombosis, Post-Thrombotic Syndrome, Anticoagulation, Percutaneous Mechanical Thrombectomy
UT Southwestern