Search Results
Viral Infection and Respiratory Illness Universal Study[VIRUS]: COVID-19 Registry (COVID-19)
Researchers are creating a real time COVID-19 registry of current ICU/hospital care patterns to allow evaluations of safety and observational effectiveness of COVID-19 practices and to determine the variations in practice across hospitals.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Farzin.Ahmed@UTSouthwestern.edu
• COVID-19 PCR positive (within 7 days)
• COVID-19 PCR pending
• COVID-19 high clinical suspicion
• Patient without Prior Research Authorization (applicable to Mayo Clinic sites)
• Non COVID-19 related admissions
• Repeated Admission to ICUs/Hospital
Safeguarding the Brain of Our Smallest Infants Phase III (SafeBoosC)
the SafeBoosC-III trial investigates the benefit and harms of treatment based on near-infrared spectroscopy monitoring compared with treatment as usual. The hypothesis is that treatment based on near-infrared spectroscopy monitoring for extremely preterm infants during the first 72 hours of life will result in a reduction in severe brain injury or death at 36 weeks postmenstrual age.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Pollieanna.Sepulveda@UTSouthwestern.edu
• Infants born with postmenstrual age less than 28 weeks
• Signed informed consent, unless the NICU has chosen to use 'opt-out' or deferred consent as consent method.
• Missing written parental informed consent (if the 'opt-out' method is used for consent, lack of a record that the clinical staff have explained the trial and the 'opt-out' consent process to parents and/or a record in the infant's clinical file of parents' decision to opt-out, are exclusion criteria)
• Decision not to conduct full life support
• No possibility to place cerebral NIRS oximeter within six hours after birth
Hydroxychloroquine in Individuals At-risk for Type 1 Diabetes Mellitus (TN-22)
The study is a 2-arm, double blinded, multicenter, 2:1 randomized, placebo controlled clinical trial. Subjects will receive hydroxychloroquine or placebo and close monitoring for progression of T1D.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Michelle.Murphy@UTSouthwestern.edu
• Participant in TrialNet Pathway to Prevention Study (TN01)
• Age 3 years or greater at the time of randomization
• Willing to provide informed consent
• Normal glucose tolerance by OGTT within 7 weeks (no more than 52 days) of baseline
• Two or more diabetes-related autoantibodies present on two separate samples
• Weight of 12 kg or greater at screening
• If a female participant with reproductive potential, willing to avoid pregnancy and undergo pregnancy testing prior to randomization and at each study visit
• Anticipated ability to swallow study medication.
• Abnormal Glucose Tolerance or Diabetes
• History of treatment with insulin or other diabetes therapies
• Ongoing use of medications known to influence glucose tolerance
• Ongoing or anticipated future use of medications known to have untoward interactions with hydroxychloroquine
• Known hypersensitivity to 4-aminoquinoline compounds
• G6PD deficiency
• History of retinopathy
• Have an active infection at time of randomization
• Have serologic evidence of current or past HIV, Hepatitis B (positive for Hepatitis B core antibody or surface antigen), or Hepatitis C infection
• Deemed unlikely or unable to comply with the protocol or have any complicating medical issues, including prolonged QT interval, a disease previously or likely in the future to require immunosuppression, or abnormal clinical laboratory results that interfere with study conduct or cause increased risk.
TruGraf® Long-term Clinical Outcomes Study
This is a prospective, multi-center, observational study. Subjects will have OmniGraf™ (TruGraf® and TRAC™) testing at study enrollment and thereafter every 3 months. In addition subjects will have OmniGraf™ (TruGraf® and TRAC™) testing at any time there is a clinical suspicion of acute rejection. Data collection for the primary objective extends over a 2-year period.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Elaine.Bonilla@UTSouthwestern.edu
• Written informed consent and HIPAA authorization;
• At least 18 years of age;
• Recipient of a primary or subsequent deceased-donor or living-donor kidney transplant;
• At least 3-months post-transplant;
• Stable serum creatinine (per Principal Investigator);
• Treated with any immunosuppressive regimen, and;
• Selected by provider to undergo OmniGraf™ (TruGraf® and TRAC™) testing as part of post-transplant care; and
• Recipient of a combined organ transplant with an extra-renal organ and/or islet cell transplant;
• Recipient of a previous non-renal solid organ and/or islet cell transplant;
• Known to be pregnant;
• Known to be infected with HIV;
• Known to have Active BK nephropathy;
• Known to have nephrotic proteinuria (Per Principal Investigator);
• Participation in other biomarker studies testing clinical utility.
Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia (CAHtalyst)
This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 24 weeks in approximately 165 adult subjects with classic CAH due to 21-hydroxylase deficiency. The study consists of a 6 month randomized, double blind, placebo-controlled period, followed by 1 year of treatment with crinecerfont. Duration of participation is approximately 20 months.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Michelle.Murphy@UTSouthwestern.edu
• Be willing and able to adhere to the study procedures, including all requirements at the study center and return for the follow-up visit.
• Have a medically confirmed diagnosis of classic 21-hydroxylase deficiency CAH.
• Be on a stable regimen of steroidal treatment for CAH.
• Patients of childbearing potential must agree to use hormonal or two forms of nonhormonal contraception (dual contraception) or other highly effective contraception during the study.
• Have a diagnosis of any of the other known forms of classic CAH.
• Have a history of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic glucocorticoid therapy.
• Have a clinically significant unstable medical condition or chronic disease other than CAH.
• Have a history of cancer unless considered cured.
• Are pregnant.
• Have a known history of clinically significant arrhythmia or abnormalities on ECG.
• Have a known hypersensitivity to any corticotropin releasing hormone antagonists.
• Have received any other investigational drug within 30 days before initial screening or plan to use an investigational drug (other than the study drug) during the study.
• Have current substance dependence, or current substance (drug) or alcohol abuse.
• Have had a blood loss ≥550 mL or donated blood or blood products within 8 weeks prior to the study.
Efficacy and Safety of Relacorilant in Patients With Cortisol-Secreting Adrenal Adenomas (GRADIENT)
This is a Phase 3, randomized, double-blind, placebo-controlled study to assess the efficacy, and safety of relacorilant to treat hypercortisolism in patients with cortisol-secreting adrenal adenoma or hyperplasia associated with diabetes mellitus/ impaired glucose tolerance and/or uncontrolled systolic hypertension.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Natalie.Booker@UTSouthwestern.edu
• Shows lack of cortisol suppression
• Suppressed or low early-morning ACTH levels
• A radiologically confirmed adrenal lesion
• Has IGT or DM
• Has uncontrolled hypertension
• Has severe, uncontrolled hypertension
• Has poorly controlled DM
• Has significantly abnormal liver test results or severe renal insufficiency
• Has uncontrolled, clinically significant hypothyroidism or hyperthyroidism
Phase II Randomized Trial of Carboplatin+Pemetrexed+Bevacizumab+/- Atezolizumab in Stage IV NSCLC
While cigarette smoking remains the primary cause of most lung cancer cases, lung carcinoma in never smokers account for nearly 20 percent of cases. Never smokers with lung cancer typically present with different molecular profiles from that of smokers, which results in prognostic and therapeutic implications. Molecular changes in NSCLC that have therapeutic significance include mutations in the epidermal growth factor receptor (EGFR) and rearrangements in the anaplastic lymphoma kinase (ALK) gene. These driver mutations typically are present in lung tumors found in never or light smokers. The addition of bevacizumab to carboplatin and paclitaxel in first-line treatment of non-squamous NSCLC showed improved survival compared to carboplatin and paclitaxel alone, 12.3 vs. 10.3 months respectively. Results from the POINTBREAK trial demonstrated that carboplatin + pemetrexed + bevacizumab is an alternative option to carboplatin + paclitaxel + bevacizumab, with comparable survival but less toxicity. In recent years, immunotherapy has emerged as a form of treatment that can lead to robust responses in a subset of patients. The PD-1 inhibitor nivolumab and the PD-L1 inhibitor atezolizumab have shown prolonged survival in comparison to docetaxel in patients who previously progressed with chemotherapy, irrespective of PD-L1 expression. Thus, this study combines immunotherapeutic agent atezolozumab with an ant-angiogenic agent, bevacizumab, and double platinum therapy (carboplatin and pemetrexed).
Call 833-722-6237
canceranswerline@utsouthwestern.edu
• Patients must have histologically or cytologically confirmed stage IV non-squamous non-small cell lung cancer
• Patients must either have tumors that harbor an EGFR mutation in exon 19 or exon 21, or must be never smoker wild-types. Never smoker wild-types are defined as patients with tumors without an ALK or ROS1 rearrangement, and are not harboring any EGFR mutation (this includes exons 19 or 21, exon 20, and any other rare EGFR mutations). Never smoker wild-type patients must have smoked less than 100 cigarettes in a lifetime. Patients with an EGFR mutation in exon 19 or 21 may be included irrespective of their smoking history. If tissue-based testing for EGFR mutation status is not available, blood-based EGFR testing that confirms presence of a mutation in exon 19 or 21 is acceptable, and these patients may be included in the study
• Patients must have measurable disease by CT or MRI, defined as at least one lesion that can be accurately measured in at least one dimension in accordance with RECIST criteria v 1.1
• Patients with tumors that harbor an EGFR exon 19 or exon 21 mutation must have received prior treatments with one or more TKIs. A washout period of at least 2 weeks is required to begin treatment in this trial. Patients who are never smoker wild-types must be treatment naïve
• All patients must be chemotherapy, VEGF therapy, and immunotherapy naive, with the exception of prior oral TKIs which are required for EGFR mutated patients. The number of prior oral TKIs and duration of use is neither specified nor limited.
• Patients with a history of treated asymptomatic CNS metastases are eligible, provided they meet all of the following criteria:
• Only supratentorial and cerebellar metastases allowed (i.e., no metastases to midbrain, pons, medulla or spinal cord)
• No ongoing requirement for corticosteroids as therapy for CNS disease
• No stereotactic radiation within 7 days or whole-brain radiation within 14 days prior to randomization
• No evidence of interim progression between the completion of CNS-directed therapy and the screening radiographic study Patients with new asymptomatic CNS metastases detected at the screening scan must receive radiation therapy and/or surgery for CNS metastases. Following treatment, these patients may then be eligible without the need for an additional brain scan prior to randomization, if all other criteria are met
• Age > 18 years
• ECOG performance status 0 or 1
• Patients must have normal organ and marrow function as defined below. The use of G-CSF should follow standard recommendations and physician discretion. If blood transfusion is performed for achieving hemoglobin levels, the levels should stay at ≥ 9.0 mg/ml for at least a week after transfusion. Absolute neutrophil count > 1,500/mcL Hemoglobin ≥ 9.0 mg/ml Platelets > 100,000/mcL Total bilirubin ≤1.5 X institutional upper limit of normal (ULN) AST/ALT (SGOT/SGPT) < 3 times institutional normal limits, or up to 5 times institutional normal limits if the patient has liver metastases Creatinine OR Creatinine clearance ≤1.5 X ULN, OR > 40 Ml/min/1.73 m2 for patients with creatinine levels above institutional normal as per Cockcroft-Gault formula International Normalized Ratio (INR) or Prothrombin Time (PT) ≤1.5 X ULN unless subject is receiving anticoagulant therapy as long as PT or PTT is within therapeutic range of intended use of anticoagulants Activated Partial Thromboplastin Time (aPTT) <1.5 X ULN unless subject is receiving anticoagulant therapy as long as PT or PTT is within therapeutic range of intended use of anticoagulants Thyroid stimulating hormone (TSH) Within normal limits a a: If TSH is not within normal limits at baseline, the subject will still be eligible if total T3 or free T4 are within normal limits.
• Patients on full-dose anticoagulation must be on a stable dose (minimum duration 14 days) of oral anticoagulant or low molecular weight heparin (LMWH). If receiving warfarin, the patient must have an INR ≤3.0. For heparin and LMWH there should be no clinically significant active bleeding (with no bleeding within 14 days prior to first dose of protocol therapy) or pathological condition present that carries a high risk of bleeding (for example, tumor involving major vessels or known varices).
• Ability to understand and willingness to sign a written informed consent and HIPAA consent document.
• A core biopsy must be available for the study. The biopsy sample must be adequate for analyses. If the sample is not adequate, the patient must agree to provide a fresh biopsy specimen before the start of treatment. Any available archival tissue will also be collected.
• Urinary protein must be ≤1+ on dipstick or routine urinalysis (UA; if urine dipstick or routine analysis is ≥2+, a 24 hour urine collection for protein must demonstrate <1000 mg of protein in 24 hours to allow participation in the protocol).
• Female subjects of child-bearing potential must be willing to use an effective method of contraception, for the course of the study through at least 6 months after the last dose of study medication.
• Male patients who have WOCBP partners must agree to use effective method of contraception for the course of the study through 8 months after the last dose of study medication.
• Note: Abstinence is acceptable if this is the usual lifestyle and preferred contraception for the subject.
• Patients currently receiving any other investigational agents, immunomodulatory agents, chemotherapy, or TKIs. EGFR mutation-positive patients must have received prior TKI treatment
• The patient has experienced any Grade 3-4 GI bleeding within 3 months prior to first dose of protocol therapy.
• The patient has a history of deep vein thrombosis (DVT), pulmonary embolism (PE), or any other significant thromboembolism (venous port or catheter thrombosis or superficial venous thrombosis are not considered "significant") during the 3 months prior to the first dose of protocol therapy.
• Subjects with untreated CNS metastases are excluded, even if they are asymptomatic. Patients with treated brain metastases will be allowed if brain imaging obtained within 28 days of trial enrollment reveals stable disease.
• Cirrhosis at a level of Child-Pugh B or worse, or cirrhosis of any degree and a history of hepatic encephalopathy, or clinically meaningful ascites resulting from cirrhosis. Clinically meaningful ascites is defined as ascites from cirrhosis requiring diuretics or paracentesis.
• The patient has experienced any arterial thromboembolic events, including but not limited to myocardial infarction, transient ischemic attack, cerebrovascular accident, or unstable angina, within 6 months prior to first dose of protocol therapy.
• The patient has uncontrolled or poorly-controlled hypertension (>150 mmHg systolic or > 100 mmHg diastolic for >4 weeks) despite standard medical management
• Prior history of hypertensive crisis or hypertensive encephalopathy
• Significant vascular disease (e.g., aortic aneurysm requiring surgical repair or recent peripheral arterial thrombosis) within 6 months prior to randomization
• Evidence of bleeding diathesis or coagulopathy (in the absence of therapeutic anticoagulation)
• History of abdominal or tracheosphageal fistula or gastrointestinal perforation within 6 months prior to randomization
• Clinical signs of gastrointestinal obstruction or requirement for routine parenteral hydration, parenteral nutrition, or tube feeding
• Evidence of abdominal free air not explained by paracentesis or recent surgical procedure
• Serious, non-healing wound, active ulcer, or untreated bone fracture within 28 days prior to first dose of protocol therapy
• Subjects with a history of smoking greater than a 100 cigarettes in a lifetime, unless their tumor has an EGFR exon 19 or exon 21 mutation.
• Patients with active, suspected, or known autoimmune disease that has required systemic treatment in the past one year (i.e., with use of disease modifying agents, corticosteroids or immunosuppressive drugs). Hormone replacement therapy (e.g. thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment.
• Patients with a history of hemoptysis (defined as bright red blood or ≥1/2 teaspoons) within 1 month prior to first dose of protocol therapy or with radiographic evidence of major blood vessel invasion or encasement by cancer.
• The patient has undergone major surgery within 28 days prior to first dose of study treatment, or minor surgery/ subcutaneous venous access device placement within 7 days prior to first dose of protocol therapy. The patient has elective or planned major surgery to be performed during the course of the clinical trial.
• The patient is receiving chronic anti-platelet therapy other than aspirin, including non-steroidal anti-inflammatory drugs (NSAIDs, including ibuprofen, naproxen, and others), dipyridamole or clopidogrel, or similar agents. Once-daily aspirin use (maximum dose 325 mg/day) is permitted. Occasional use of NSAIDs is allowed (for example daily use for less than a week; treating physician discretion is permitted to differentiate between occasional vs chronic use)
• Patients who have not recovered from adverse events due to agents administered earlier except neuropathy and alopecia. Physician's discretion is allowed to decide which unresolved adverse events from previous therapy (for NSCLC) prohibit patient participation in this study.
• Patients requiring more than 10 mg prednisolone (or its equivalent) per day are excluded.
• Patients with any evidence of interstitial lung disease (ILD) or pneumonitis or a prior history of ILD or pneumonitis requiring oral or IV glucocorticoids. History of radiation pneumonitis in the radiation field (fibrosis) is permitted
• Patients with active tuberculosis infection are excluded.
• Patients who have received a live vaccine within 30 days prior to cycle 1 Day 1.
• Uncontrolled illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia (significant), cirrhosis, or psychiatric illness/ social situations that would limit compliance with the study requirements.
• Known history of testing positive for immunodeficiency virus (HIV) or known acquired immunodeficiency syndrome (AIDS).
• Known history of chronic hepatitis B virus infection or chronic hepatitis C virus indicating chronic infection that is not cured.
• Subjects with previous malignancies (except non-melanoma skin cancers, and in situ cancers, such as, bladder, gastric, colon, cervical/ dysplasia, melanoma, or breast) are excluded unless a complete remission was achieved at least 2 years prior to study registration and no additional therapy is required or anticipated to be required during the study period.
• Leptomeningeal disease
• Uncontrolled tumor-related pain Patients requiring pain medication must be on a stable regimen at study entry. Symptomatic lesions amenable to palliative radiotherapy (e.g., bone metastases or metastases causing nerve impingement) should be treated prior to randomization. Patients should be recovered from the effects of radiation. There is no required minimum recovery period. Asymptomatic metastatic lesions whose further growth would likely cause functional deficits or intractable pain (e.g., epidural metastasis that is not currently associated with spinal cord compression) should be considered for locoregional therapy, if appropriate, prior to randomization.
• Uncontrolled pleural effusion, pericardial effusion, or ascites requiring recurrent drainage procedures (once monthly or more frequently). Patients with indwelling catheters (e.g., PleurX®) are allowed.
• Ca > 12 mg/dl or corrected serum calcium > ULN Patients who are receiving denosumab prior to randomization must be willing and eligible to receive a bisphosphonate instead while in the study
• Pregnant or breast feeding
• Prior allogeneic bone marrow transplantation or solid organ transplant
• Known hypersensitivity to Chinese hamster ovary cell products or any of the study drugs.
• Clear tumor infiltration into the thoracic great vessels is seen on imaging
• Clear cavitation of pulmonary lesions is seen on imaging
• Subjects with squamous cell carcinoma of the lung.
• Subjects with a lung tumor with a known ALK or ROS1 rearrangement or an EGFR mutation other than in exon 19 or exon 21.
A Study to Evaluate the Efficacy and Safety of Dapirolizumab Pegol in Study Participants With Moderately to Severely Active Systemic Lupus Erythematosus (PHOENYCS GO)
The purpose of this study is to evaluate the ability of dapirolizumab pegol (DZP) as an add-on treatment to standard of care (SOC) medication to achieve clinically relevant long term improvement of moderate to severe disease activity.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Maysa.Ahmed@UTSouthwestern.edu
• Study participant must be ≥16 years of age
• Study participants who have moderate to severe disease activity due to either persisting active SLE or due to an acute worsening of SLE in the scope of frequent flaring/relapsing-remitting systemic lupus erythematosus (SLE) despite stable standard of care (SOC) medication defined as:
• Diagnosed with SLE at least 24 weeks before the Screening Visit (Visit 1) study entry by a qualified physician
• Classified by 2019 SLE European League Against Rheumatism/American College of Rheumatology (EULAR/ACR) classification criteria for SLE
• With serological evidence for SLE at Screening as demonstrated by at least 1 of the following: i) Evidence for anti-dsDNA (in central laboratory at Screening) ii) Either complement C3 < lower limit of normal (LLN) OR complement C4
• Anti-Sjögren's syndrome antibody A (Anti-SSA) (Ro)/Anti-Sjögren's syndrome antibody B (anti-SSB) (La) autoantibodies (central laboratory)
• Historic evidence for anti-dsDNA antibodies d. Moderately to severely active defined as
• British Isles Lupus Assessment Group Disease Activity Index 2004 (BILAG 2004) Grade B in ≥2 organ systems and/or a BILAG 2004 Grade A in ≥1 organ systems at Screening and Baseline Visit AND
• Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) ≥6 at Screening Visit AND
• SLEDAI-2K without labs ≥4 at Baseline Visit e. Receiving the following SOC medication at stable dose:
• Antimalarial treatment in combination with corticosteroids and/or immunosuppressants or as stand-alone treatment if justified OR Treatment with corticosteroids and/or immunosuppressants if anti-malarial treatment is not possible
• Study participant has a history of an anaphylactic reaction to parenteral administration of contrast agents, human or murine proteins, or monoclonal antibodies
• Study participant has a history of malignancy, except the following treated cancers: cervical carcinoma in situ, basal cell carcinoma, or dermatological squamous cell carcinoma
• Study participant has an increased risk for thromboembolic events due to an ongoing heart disease or due to a medical device, including but not limited to vascular graft, valvular heart disease, atrial fibrillation, or a heart rhythm disorder
• Study participant has evidence of human immunodeficiency virus (HIV) infection, agammaglobulinemias, T-cell deficiencies, or human T-cell lymphotropic virus-1 infection
• Study participant had a reactivated latent or opportunistic infection within 12 weeks prior to the first study medication infusion (Visit 2), or is currently receiving suppressive therapy for an opportunistic infection
• Study participants who have received live/live attenuated vaccines within 6 weeks prior to the first study medication infusion
• Study participant has clinically significant active or latent infection
• Study participant has a mixed connective tissue disease, scleroderma, and/or overlap syndrome of these diseases with SLE
• Study participant takes any protocol defined prohibited concomitant medication
• Study participant has previously been randomized within this study or participant has previously been assigned to treatment with dapirolizumab pegol (DZP) in a study evaluating DZP
• Study participant has participated in another study of an IMP within the previous 12 weeks or 5 half-lives of the investigational medicinal product (IMP) whatever is longer or is currently participating in another study of an IMP
• Study participant has chronic kidney failure stage 4, manifested by estimated glomerular filtration rate <30mL/min/1.73m2, or serum creatinine >2.5 mg/dL, or participant has proteinuria >3 g/day, or protein: creatinine ratio >340 mg/mmol at the Screening Visit
Throat and Other HPV-Related Cancers in Men: Identifying Them Early (TRINITY)
This trial studies the screening of human papillomavirus (HPV)-related oropharyngeal and uncommon cancers. Learning the relationship between HPV and cancer risk in men who test positive for HPV antibodies may help doctors to develop early methods of screening for certain types of cancer, and screening for HPV may help doctors to learn which patients may be at a higher risk for developing certain types of cancer.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
• Stage 1
• Stage 1
• Stage 1
• Stage 2 (longitudinal study)
• Eligible for and enrolled in Stage 1
• Test positive to HPV16 E antibodies; be identified as a negative control; test positive for oral HPV16 infection by oral rinse; or test positive for cvDNA
• Sign an approved informed consent document
• Stage 1
• Stage 1
• Stage 1
• Stage 1
• Stage 1
• Stage 1
• Stage 2 (longitudinal study)
LISA in the Delivery Room for Extremely Preterm Infants (DRLISA)
The purpose of this study is to evaluate the effect of LISA used in the delivery room (DR) in decreasing the intubation rates in preterm infants at 23-25 weeks gestational age (GA), during first 72 hours compared to the standard approach of stabilization on nasal CPAP in the DR and administering surfactant in the NICU. Infants in both groups will be resuscitated per NRP algorithm. Infants who maintain a stable HR and respiratory effort on CPAP will qualify for the intervention. Infants in Group 1 (Intervention arm) will receive LISA in DR. CPAP will be titrated between 5-8 cm H20 after LISA. Infants in Group 2 (Control arm) will be transferred to NICU on CPAP. The CPAP level will be increased stepwise every 30 minutes to 7 cm H2O if FiO2 ≥0.3. Infants requiring CPAP 7 at FiO2 ≥0.3 will receive LISA. CPAP will be titrated between 5-8 cm H20 after LISA. Infants in both arms requiring CPAP 7 and FiO2 >0.8 at 20 MOL in the delivery room will be intubated in DR. Any infant with a heart rate not responding with appropriate PPV will be intubated in the DR. CXR will be obtain on admission and umbilical lines will be placed. Infants in both arm who require FiO2 ≥0.6 for ≥1 hour, apnea requiring stimulation 3 times within one hour or ≥6 over 6 hour period, any apnea requiring PPV, or CO2 >0.65 in two consecutive blood gases drawn over two hours will be considered as reasons for intubation after LISA. Primary outcome is the need for MV within 72 hours of life, secondary outcome includes need for MV during first week of life and during hospital stay, bronchopulmonary dysplasia (BPD), intraventricular hemorrhage (IVH), necrotizing enterocolitis (NEC), spontaneous intestinal perforation (SIP), need for treatment of patent ductus arteriosus (PDA), composite death or BPD and mortality. This is a feasibility trial with the intention to enroll 30 infants in each arm of the study over three years.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Kathryn.Mazioniene@UTSouthwestern.edu
• Infants born 23 -25 weeks GA
• Resuscitated without requiring intubation and maintaining HR >100, oxygen saturation per NRP goal saturation limits and regular respiratory effort on CPAP
• Major congenital anomalies
Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children (BEGIN)
This is a two-part, multi-center, prospective longitudinal, exploratory study of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators and their impact on children with cystic fibrosis (CF).
Call 214-648-5005
studyfinder@utsouthwestern.edu, Lindsay.Allen@UTSouthwestern.edu
• Part A:
• Less than 5 years of age at the first study visit.
• Documentation of a CF diagnosis. Part B:
• Participated in Part A OR less than 6 years of age at the first study visit.
• Documentation of a CF diagnosis.
• CFTR mutations consistent with FDA labeled indication of highly effective modulator therapy (ivacaftor or elexacaftor/tezacaftor/ivacaftor).
• Physician intent to prescribe ivacaftor or elexacaftor/tezacaftor/ivacaftor.
• Part A and Part B: Use of an investigational drug within 28 days prior to and including the first study visit. Use of ivacaftor or elexacaftor/tezacaftor/ivacaftor within the 180 days prior to and including the first study visit. Use of chronic oral corticosteroids within the 28 days prior to and including the first study visit.
Natural History Study of and Genetic Modifiers in Spinocerebellar Ataxias
Spinocerebellar ataxias (SCA) are genetic neurological diseases that cause imbalance, poor coordination, and speech difficulties. There are different kinds of SCA and this study will focus on types 1, 2,3, and 6 (SCA 1, SCA 2, SCA 3 , also known as Machado-Joseph disease and SCA 6). The diseases are rare, slowly progressive, cause increasingly severe neurological difficulties and are variable across and within genotypes. The purpose of this research study is to bring together a group of experts in the field of SCA for the purpose of learning more about the disease. The research questions are: 1. How does your disease progress over time? 2. What are the best ways to measure the progression? 3. Do some genes, other than the gene that is abnormal in your disease, have any effect on the way the disease behaves? This is a nationwide study and we expect that 800 patients will participate all over the USA. The participants will be in the study for an indeterminate period of time. Study visits will be done every 6 or 12 months depending on the participating site.
Call 214-648-5005
studyfinder@utsouthwestern.edu, jan.cameronwatts@utsouthwestern.edu
• Presence of symptomatic ataxic disease
• Definite molecular diagnosis of SCA 1, 2,3,or 6 either in the subject or another affected family member
• Willingness to participate in the study and ability to give informed consent.
• Age 6 years and above
• Known recessive, X-linked and mitochondrial ataxias
• Exclusion of SCA 1, 2, 3 and 6 by previous DNA testing,
• A lack of willingness to participate in the study
Non-Invasive Diagnosis of Pediatric Pulmonary Invasive Mold Infections (DOMINIC)
This study will establish a non-invasive diagnostic approach and evaluate clinical outcomes for children at high-risk for pulmonary invasive mold infection (PIMI).
Call 214-648-5005
studyfinder@utsouthwestern.edu, Aruna.Ayalasomayajula@UTSouthwestern.edu
• Males or females age > 120 days and < 22 years at any participating site
• Have at least one of the following conditions associated with a known high incidence of IMI: hematopoietic stem cell transplantation (HSCT), aplastic anemia, or hematologic malignancy
• New (last 96 hours) radiographic evidence of at least one of the following: at least one nodular lesion greater than or equal to 5 mm in size, a cavitary lesion, a lesion with a halo sign, a lesion with a reverse halo sign, or a lesion with an air crescent sign
• Prolonged neutropenia (absolute neutrophil count < 500 cells/µl for a period of ≥ 5 consecutive days) in 30 days prior to qualifying chest MRI or CT scan date OR currently receiving systemic therapy for acute or chronic graft-versus-host disease (GVHD) on the date of the qualifying chest MRI or CT scan
• Subject consent or parental/guardian permission (informed consent) and if appropriate, child assent
• Weight <3 kg, so as to not exceed 3 ml/kg in a single blood draw
• Previous inclusion in this study
Lysosomal Acid Lipase (LAL) Deficiency Registry (ALX-LALD-501)
This is an observational, multi-center, international disease registry designed to collect longitudinal data and create a knowledge base that will be utilized to improve the care and treatment of patients with LAL Deficiency. Participation in the Registry by both physicians and patients is voluntary.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Lisa.Quirk@UTSouthwestern.edu
Study of Ravulizumab in Pediatric Participants With HSCT-TMA
This study will evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of ravulizumab administered by intravenous infusion to pediatric participants, from 1 month to < 18 years of age, with HSCT-TMA. The treatment period is 26 weeks, followed by a 26-week off-treatment follow-up period.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
• 1 month of age up to < 18 years of age at the time of signing the informed consent.
• Received HSCT within the past 6 months.
• Diagnosis of TMA that persists despite initial management of any triggering condition.
• Body weight ≥ 5 kilograms.
• Female participants of childbearing potential and male participants with female partners of childbearing potential must use highly effective contraception starting at Screening and continuing until at least 8 months after the last dose of ravulizumab.
• Participants must be vaccinated against meningococcal infections if clinically feasible, according to institutional guidelines for immune reconstitution after HSCT. Participants must be re-vaccinated against Haemophilus influenzae type b and Streptococcus pneumoniae if clinically feasible, according to institutional guidelines for immune reconstitution after HSCT. All participants should be administered coverage with prophylactic antibiotics according to institutional post-transplant infection prophylaxis guidances, including coverage against Neisseria meningitidis for at least 2 weeks after meningococcal vaccination. Participants who cannot receive meningococcal vaccine should receive antibiotic prophylaxis coverage against Neisseria meningitidis the entire Treatment Period and for 8 months following the final dose of ravulizumab.
• Known familial or acquired 'a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13' deficiency (activity < 5%).
• Known Shiga toxin-related hemolytic uremic syndrome.
• Positive direct Coombs test.
• Diagnosis or suspicion of disseminated intravascular coagulation.
• Known bone marrow/graft failure.
• Diagnosis of veno-occlusive disease.
• Human immunodeficiency virus (HIV) infection (evidenced by HIV-1 or HIV-2 antibody titer).
• Unresolved meningococcal disease.
• Presence or suspicion of sepsis (treated or untreated) within 7 days prior to Screening.
• Pregnancy or breastfeeding.
• Hypersensitivity to murine proteins or to 1 of the excipients of ravulizumab.
• Previously or currently treated with a complement inhibitor.
IV Gallium Study for Patients With Cystic Fibrosis Who Have NTM (ABATE Study) (ABATE)
The purpose of this study is to assess the safety and tolerability of two 5-day infusion cycles of IV gallium in adult patients with CF who are infected with NTM. Funding Source - FDA OOPD
Call 214-648-5005
studyfinder@utsouthwestern.edu, LYNN.FERNANDEZ@UTSouthwestern.edu
• Written informed consent obtained from subject or subject's legal representative
• Be willing and able to adhere to the study visit schedule and other protocol requirements
• Greater than or equal to 18 years of age at Visit 1
• Documentation of a CF diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and one or more of the following criteria:
• Sweat chloride ≥ 60 milliequivalent (mEq)/liter by quantitative pilocarpine iontophoresis test (QPIT)
• Two well-characterized mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene
• Abnormal nasal potential difference (NPD) (change in NPD in response to a low chloride solution and isoproteronol of less than -5 mV)
• Documentation of NTM culture positive defined as follows:
• Two positive NTM culture results from sputum (or BAL) at least 28 days apart (these are the two qualifying positive cultures)
• Both qualifying positive culture results include M. avium complex, M. abscessus complex, or both M. avium and M. abscessus
• Both qualifying positive culture results include the same species or subspecies
• No cultures negative for NTM since the first of the two qualifying positive culture results
• Current NTM species or subspecies has never been treated or previous treatment was associated with clearance of NTM and completed > 2 years prior to Day 1
• Forced expiratory volume in 1 second (FEV1) ≥ 25 % of predicted value at Screening
• Able to expectorate sputum
• Clinically stable with no significant changes in health status within 7 days prior to Day 1
• Enrolled in the CFF Cystic Fibrosis Foundation Patient Registry (CFFPR)
• Willing to discontinue chronic azithromycin use for the duration of the study
• Any of the following abnormal lab values at screening:
• Hemoglobin <10g/dL
• Platelets <100,000/mm3
• White blood cells (WBC) < 4,500/mm3
• Aspartate transaminase (AST), alanine transaminase (ALT), gamma-glutamyl transferase (GGT), or alkaline phosphatase (ALP) ≥3 x upper limit of normal
• Serum creatinine > 2.0 mg/dl and ≥1.5 x upper limit of normal
• Ionized calcium ≤ lower limit of normal (only performed if total calcium is ≤ lower limit of normal)
• History of solid organ or hematological transplantation
• Use of bisphosphonates within 7 days prior to Day 1
• Known sensitivity to gallium
• Use of any investigational drug and/or participated in any interventional clinical trial within 28 days prior to Day 1
• In the opinion of the Investigator, features of active NTM disease are present (e.g., clinical worsening is likely due to NTM disease despite definitive treatment of co-pathogens and/or acute exacerbations)
• Undergoing treatment for NTM disease or anticipate beginning treatment within 3 months
• Current diagnosis of osteoporosis
• For people of childbearing potential:
• Positive pregnancy test at Visit 1 or
• Lactating or
• Unwilling to practice a medically acceptable form of contraception (acceptable forms of contraception: abstinence, hormonal birth control, intrauterine device, or barrier method plus a spermicidal agent), unless surgically sterilized or postmenopausal during the study
• For people able to father a child: unwilling to use adequate contraception (as determined by the investigator) during the study
• Has any other condition that, in the opinion of the Site Investigator/designee, would preclude informed consent or assent, make study participation unsafe, complicate interpretation of study outcome data, or otherwise interfere with achieving the study objectives
• New initiation of chronic therapy (greater than 21 days) within 28 days prior to the Enrollment Visit
Brain Networks and Consciousness
General anesthesia (GA) is a medically induced state of unresponsiveness and unconsciousness, which millions of people experience every year. Despite its ubiquity, a clear and consistent picture of the brain circuits mediating consciousness and responsiveness has not emerged. Studies to date are limited by lack of direct recordings in human brain during medically induced anesthesia. Our overall hypothesis is that the current model of consciousness, originally proposed to model disorders and recovery of consciousness after brain injury, can be generalized to understand mechanisms of consciousness more broadly. This will be studied through three specific aims. The first is to evaluate the difference in anesthesia sensitivity in patients with and without underlying basal ganglia pathology. Second is to correlate changes in brain circuitry with induction and emergence from anesthesia. The third aim is to evaluate the effects of targeted deep brain stimulation on anesthesia induced loss and recovery of consciousness. This study focuses on experimentally studying these related brain circuits by taking advantage of pathological differences in movement disorder patient populations undergoing deep brain stimulation (DBS) surgery. DBS is a neurosurgical procedure that is used as treatment for movement disorders, such as Parkinson's disease and essential tremor, and provides a mechanism to acquire brain activity recordings in subcortical structures. This study will provide important insight by using human data to shed light on the generalizability of the current model of consciousness. The subject's surgery for DBS will be prolonged by up to 40 minutes in order to record the participant's brain activity and their responses to verbal and auditory stimuli.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Sahil.Chilukuri@UTSouthwestern.edu
• Willingness and ability to cooperate during conscious operative procedure for up to 40 minutes
• Clinical diagnosis of Parkinson's disease or essential tremor
• Preoperative MRI without evidence of cortical or subdural adhesions or vascular abnormalities
• Patients with recent use (within one week) of anticoagulant or antiplatelet agent use
• Neurocognitive testing indicating amnestic cognitive deficits
• History of intolerance of propofol or medical indications to use an anesthetic other than propofol
RAndomized Therapy In Status Epilepticus (RAISE)
This study will evaluate the effectiveness and safety of an investigational product, IV ganaxolone, to treat subjects with status epilepticus.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Karla.CastroOchoa@UTSouthwestern.edu
• clinical and/or electrographic seizures
• life expectancy of less than 24 hours
• anoxic brain injury or an uncontrolled metabolic condition as primary cause of SE
• treatment of current SE episode with IV anesthetics
Vagus Nerve Stimulation for Moderate to Severe Rheumatoid Arthritis (RESET-RA)
The RESET-RA study will assess the safety and efficacy of the SetPoint System (study device) for the treatment of adult patients with active, moderate to severe rheumatoid arthritis who have had an inadequate response or intolerance to biologic or targeted synthetic Disease-Modifying Anti-Rheumatic Drugs (DMARDs). The study device contains a miniaturized stimulator (implant) that is surgically placed under general anesthesia on the vagus nerve through a small incision on the left side of the neck (implant procedure). The study will enroll 250 subjects at 40 sites. All eligible subjects will undergo the implant procedure. Half of the subjects will receive active stimulation (treatment) and the other half will receive non-active stimulation (control). After completing primary endpoint assessments at Week 12, there will be a one-way crossover of control subjects to active stimulation and a 180-week open-label follow-up with all subjects (treatment and control) receiving active stimulation to evaluate long-term safety.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Aisha.Qureshi@UTSouthwestern.edu
• 22-75 years of age at screening
• Active moderate or severe RA, defined as at least 4/28 tender and 4/28 swollen joints
• Demonstrated an inadequate response, loss of response, or intolerance to 1 or more approved for rheumatoid arthritis biologic or targeted synthetic Disease-Modifying Anti-Rheumatic Drugs (DMARDs), including Janus kinase inhibitors (JAKi)
• Receiving treatment with at least 1 conventional synthetic DMARD for at least 12 weeks and on a continuous non-changing dose and route of administration for at least 4 weeks prior to Screening and able to continue the same stable dose through Week 12
• Untreated or poorly controlled psychiatric illness or history of substance abuse
• Significant immunodeficiency due to underlying illness
• History of stroke or transient ischemic attack, or diagnosis of cerebrovascular fibromuscular dysplasia
• Clinically significant cardiovascular disease
• Neurological syndromes, including multiple sclerosis, Alzheimer's disease, or Parkinson's disease
• Uncontrolled fibromyalgia
• History of left or right carotid surgery
• History of unilateral or bilateral vagotomy, partial or complete splenectomy
• Recurrent vasovagal syncope episodes
• Current, regular use of tobacco products
• Hypersensitivity/allergy to MRI contrast agents and/or unable to perform MRI
Treatment Combination of Durvalumab, Tremelimumab and Enfortumab Vedotin or Durvalumab and Enfortumab Vedotin in Patients With Muscle Invasive Bladder Cancer Ineligible to Cisplatin (VOLGA)
A Global Study to Determine the Efficacy and Safety of Durvalumab in combination with Tremelimumab and Enfortumab Vedotin or Durvalumab in Combination With Enfortumab Vedotin for Perioperative Treatment in Patients Ineligible for Cisplatin Undergoing Radical Cystectomy for Muscle Invasive Bladder Cancer
Call 833-722-6237
canceranswerline@utsouthwestern.edu
• Histologically or cytologically documented muscle-invasive TCC of the bladder with clinical stage T2-T4aN0/1M0 with transitional and mixed transitional cell histology;
• Medically fit for cystectomy and able to receive neoadjuvant therapy;
• Patients who have not received prior systemic chemotherapy or immunotherapy for treatment of MIBC;
• ECOG performance status of 0,1,2 at enrollment.
• Availability of tumor sample prior to study entry;
• Must have a life expectancy of at least 12 weeks at randomization. Exclusion criteria:
• Evidence of lymph node (N2+) or metastatic TCC/UC disease at the time of screening.
• Active infection
• Uncontrolled intercurrent illness
• Prior exposure to immune-mediated therapy (with exclusion of Bacillus-Calmette Guerin [BCG]), including but not limited to other anti-CTLA-4, anti--PD-1, anti PD-L1, or anti-PD-L2 antibodies.
• Current or prior use of immunosuppressive medication within 14 days before the first dose of IPs.
Effects of Hypoglossal Nerve Stimulation on Cognition and Language in Down Syndrome and Obstructive Sleep Apnea
This study is a prospective, single-arm study conducted under a common implant and follow-up protocol. The objective will be to follow fifty-seven (57) adolescents and young adults (10-21 years of age), with Down syndrome, moderate to severe sleep apnea, and post-adenotonsillectomy, for 12 months after undergoing implant of the Inspire Upper Airway Stimulation (UAS) System. The study is being conducted in order to evaluate objective change in cognition and expressive language after implant and therapy with the Inspire UAS System.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Francesca.Chambers@UTSouthwestern.edu
• Diagnosis of Down syndrome
• Age 10-21 years
• Prior adenotonsillectomy
• Severe OSA (AHI > 10, AHI < 50, no more than 25% AHI attributable to central events) based on prior in-lab PSG performed after adenotonsillectomy and within 18 months of enrollment
• Approval from at least two of the three physician reviewers based upon the results of a routine drug-induced sleep endoscopy (DISE) having occurred within 12 months of enrollment
• Subjects must have either tracheotomy or be ineffectively treated with CPAP due to non-compliance, discomfort, un-desirable side effects, persistent symptoms despite compliance use, or refusal to use the device
• Children and their parents/guardians must be willing to have stimulation hardware permanently implanted, and be willing to participate in follow-up visits, postoperative PSG, and questionnaire completion
• Children's parents/guardians must complete a questionnaire confirming that their child is capable of communicating feelings of pain or discomfort. They must also confirm they are able to assess their child for adverse effects related to device implantation
• Children and their parents/guardians must be proficient in English
• Body mass index (BMI) above the 95th percentile for subject's age
• Circumferential airway collapse at the level of the velopharynx observed during DISE
• Other medical conditions resulting in medical instability (eg. congestive heart failure, recent open heart surgery, immunosuppression, or chronic lung disease or aspiration)
• Presence of another medical condition requiring future magnetic resonance imaging (MRI) of the chest
• Patients with another implantable device which could interact unintentionally with the Inspire system
• Any contraindication for general anesthesia
• History of bleeding or clotting disorders and those on blood thinning or NSAID medications for the week prior to implantation surgery. Subjects will be asked to refrain from the use of NSAIDS for two weeks after implantation or any revision surgeries
• Subject is currently taking muscle relaxant medication
• Life expectancy less than 12 months
• Subject's inability to communicate pain or discomfort to their caretaker/parent, based on parental or investigator assessment
• Nonverbal candidates will be excluded due to an inability to complete testing procedures including expressive language sampling
• Subjects with a co-occurring diagnosis of autism spectrum disorder
• Subjects that have a positive β-HCG
• Subjects deemed unfit for participation by the investigator for any other reason
COMPASSION S3 Post-Approval Study
This study will monitor device performance and outcomes of the SAPIEN 3 Transcatheter Heart Valve (THV) System in subjects with a dysfunctional right ventricular outflow tract (RVOT) conduit or previously implanted surgical valve in the pulmonic position with a clinical indication for intervention.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Kirstie.LeDoux@UTSouthwestern.edu
• Dysfunctional RVOT conduit or previously implanted surgical valve
• RVOT/PV with ≥ moderate regurgitation and/or a mean RVOT/PV gradient of ≥ 35 mmHg
• Inability to tolerate an anticoagulation/antiplatelet regimen
• Active bacterial endocarditis or other active infections
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Participants With Duchenne Muscular Dystrophy (DMD) (EMBARK)
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene transfer therapy at the beginning of the second year.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Kristy.Riddle@UTSouthwestern.edu
• Is ambulatory and from 4 to under 8 years of age at time of randomization.
• Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
• Ability to cooperate with motor assessment testing.
• Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).
• rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
• Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol specified time limits.
• Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
• Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer. Other inclusion or exclusion criteria could apply.
A Study of ISIS 678354 Administered to Participants With Severe Hypertriglyceridemia
The purpose of the study is to evaluate the efficacy of ISIS 678354 as compared to placebo on the percent change in fasting triglycerides (TG) from baseline.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Lakeisha.Cade@UTSouthwestern.edu
• Fasting TG ≥ 500 mg/dL (5.65 mmol/L) at Screening and Qualification
• Patients should be on standard of care lipid-lowering medications per local guidelines unless intolerant. Lipid-lowering medications should be optimized and stabilized for at least 4 weeks prior to Screening to minimize changes in these medications during the study. Key
• Hemoglobin A1c (HbA1c) ≥ 9.5% at Screening
• Platelet count < 100K/cubic millimeters at Screening or Qualification
• Alanine aminotransferase or aspartate aminotransferase > 3.0 × upper limit of normal
• Total bilirubin > upper limit of normal unless due to Gilbert's syndrome
• Estimated GFR < 40 mL/min/1.73 m^2
A Study to Test if Fremanezumab is Effective in Preventing Episodic Migraine in Patients 6 to 17 Years of Age
The primary objective of the study is to evaluate the effectiveness of fremanezumab as compared to placebo for the preventive treatment of episodic migraine (EM). Secondary objectives are to further demonstrate the efficacy of Fremanezumab as compared to placebo for the preventive treatment of EM, to evaluate the safety and tolerability of Fremanezumab in the preventive treatment of EM and to evaluate the immunogenicity of Fremanezumab and the impact of antidrug antibodies (ADAs) on clinical outcomes in participants exposed to Fremanezumab. The total duration of the study is planned to be up to 36 months.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Kiley.Poppino@UTSouthwestern.edu
• The participant has a clinical history of recurrent headache consistent with the diagnosis of migraine for at least 6 months before screening, consistent with ICHD-3 criteria (Headache Classification Committee of the IHS 2013), and a history of ≤=14 headache days per month in each of the 3 months prior to screening (visit 1).
• The participant or parent/caregiver maintain a prospectively collected headache diary
• The participant does not have chronic daily headache. For the purposes of this study, chronic daily headache is operationally defined as <4 headache-free days during the 28-day baseline period. NOTE: Additional criteria apply; please contact the investigator for more information.
• The participant is using medications containing opioids (including codeine) or barbiturates (including Fiorinal®, Fioricet®, or any other combination containing butalbital) for the treatment of migraine during the 3 months prior to the day of the screening visit.
• The participant or parent/caregiver maintain a prospectively collected headache diary
• The participant has used an intervention/device (eg, scheduled nerve block or transcranial magnetic stimulation) for the treatment of migraine or in the head or neck area for any condition during the 2 months prior to the day of the screening visit.
• The participant has a current history of a clinically significant psychiatric condition, any prior history of a suicide attempt, or a history of suicidal ideation with a specific plan within the past 2 years, at the discretion of the investigator.
• The participant has an ongoing infection or a known history of human immunodeficiency virus infection, tuberculosis, Lyme disease, or chronic hepatitis B or C, or a known active infection of coronavirus disease 2019 (COVID-19).
• The participant has a past or current history of cancer.
• The participant is pregnant or nursing.
• The participant has a history of hypersensitivity reactions to injected proteins, including mAbs, or a history of Stevens-Johnson Syndrome or toxic epidermal necrolysis syndrome, or the participant is concomitantly using lamotrigine.
• The participant received a live attenuated vaccine (eg, intranasal flu vaccine, and measles, mumps, and rubella vaccine) within the 12-week period prior to screening. Note: If a medical need arises during the study, the participant may receive a live attenuated vaccine.
• The patient has a current or past medical history of hemiplegic migraine. NOTE: Additional criteria apply; please contact the investigator for more information.
A Study to Test if Fremanezumab is Effective in Preventing Migraine in Children and Adolescents
The primary objective of the study is to evaluate the long-term safety and tolerability of subcutaneous fremanezumab in the preventive treatment of migraine in pediatric participants 6 to 17 years of age (inclusive at enrollment in the pivotal study). Secondary objectives are to evaluate the efficacy of subcutaneous fremanezumab in pediatric participants with migraine and to evaluate the immunogenicity of fremanezumab and the impact of ADAs on clinical outcomes in pediatric participants exposed to fremanezumab. The total duration of the study is planned to be up to 60 months.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Kiley.Poppino@UTSouthwestern.edu
• Participants have completed the pivotal efficacy study and, in the opinion of the Investigator or the Sponsor, are able to complete the study in a safe and compliant way.
• Participants may continue with a stable dose/regimen of the preventive medication they were taking during the pivotal efficacy studies.
• The participant continues to meet appropriate criteria carried forward from the pivotal efficacy study/
• The participant has received all recommended age-appropriate vaccines according to local standard of care and schedule.
• The participant weighs at least 17.0 kg on the day of study enrollment. NOTE: Additional criteria apply; please contact the investigator for more information. Inclusion Criteria for Participants Rolling Over from the Phase 1 Pediatric Pharmacokinetic Study (Study TV48125-CNS-10141):
• The participant/caregiver has demonstrated compliance with the electronic headache diary during the 28-day baseline period by entry of headache data on a minimum of 21 out of 28 days (approximately 75% diary compliance).
• The participant has received all recommended age-appropriate vaccines according to local standard of care and schedule.
• The participant weighs at least 17.0 kg on the day of study enrollment.
• The participant has a body mass index ranging from the 5th to 120% of the 95th percentile, inclusive, on the day of study enrollment.
• Not using preventive medications or using no more than 2 preventive medications for migraine or other medical condition, as long as the dose and regimen have been stable for at least 2 months prior to screening (visit 1). NOTE: Additional criteria apply; please contact the investigator for more information. Inclusion Criteria for Participants Rolling Over from the Pivotal Efficacy Studies (TV48125-CNS-30082 and TV48125-CNS-30083) for Safety and antidrug antibody (ADA) Assessment Only: • Participants may be included in this study if they sign and date the informed consent document or upon consent of a parent or guardian, if the participant is younger than the age of consent, accompanied by assent of the participant.
• In the judgment of the investigator, the participant has a clinically significant abnormal finding on study entry, including hematology, blood chemistry, coagulation tests, or urinalysis values/findings (abnormal tests may be repeated for confirmation).
• The participant has a current history of a clinically significant psychiatric condition, any prior history of a suicide attempt, or a history of suicidal ideation with a specific plan within the past 2 years, at the discretion of the investigator.
• The participant has an ongoing infection or a known history of human immunodeficiency virus infection, tuberculosis, Lyme disease, or chronic hepatitis B or C, or a known active infection of coronavirus disease 2019 (COVID-19).
• The participant has a history of hypersensitivity reactions to injected proteins, including mAbs, or a history of Stevens-Johnson Syndrome or toxic epidermal necrolysis syndrome, or the participant is concomitantly using lamotrigine.
• The participant received a live attenuated vaccine (eg, intranasal flu vaccine, and measles, mumps, and rubella vaccine) within the 12-week period prior to screening. Note: If a medical need arises during the study, the participant may receive a live attenuated vaccine.
• The participant is pregnant or nursing.
• In the judgment of the investigator, the participant has an abnormal finding on the baseline 12-lead ECG considered clinically significant.
• The patient has a current or past medical history of hemiplegic migraine. NOTE: Additional criteria apply; please contact the investigator for more information. Exclusion Criteria for Participants Rolling Over from the Phase 1 Pharmacokinetic Study (TV48125-CNS-10141):
• The participant has any clinically significant cardiovascular (including congenital cardiac anomalies or thromboembolic events), endocrine, gastrointestinal, genitourinary, hematologic, hepatic, immunologic, neurologic, ophthalmic, pulmonary, renal disease, or complications of an infection, at the discretion of the investigator.
• The participant has a current history of a clinically significant psychiatric condition, any prior history of a suicide attempt, or a history of suicidal ideation with a specific plan within the past 2 years, at the discretion of the investigator.
• The participant has an ongoing infection or a known history of human immunodeficiency virus infection, tuberculosis, Lyme disease, or chronic hepatitis B or C, or a known active infection of coronavirus disease 2019 (COVID-19).
• The participant has a history of hypersensitivity reactions to injected proteins, including mAbs, or a history of Stevens-Johnson Syndrome or toxic epidermal necrolysis syndrome, or the participant is concomitantly using lamotrigine.
• The participant received a live attenuated vaccine (eg, intranasal flu vaccine, and measles, mumps, and rubella vaccine) within the 12-week period prior to screening. Note: If a medical need arises during the study, the participant may receive a live attenuated vaccine.
• The participant is pregnant or nursing.
• In the judgment of the investigator, the participant has an abnormal finding on the baseline 12-lead ECG considered clinically significant.
• The patient has a current or past medical history of hemiplegic migraine. NOTE: Additional criteria apply; please contact the investigator for more information. Exclusion Criteria for Participants Rolling Over from the Pivotal Efficacy Studies (TV48125-CNS-30082 and TV48125-CNS-30083) for Safety and antidrug antibody (ADA) Assessment Only: Not Applicable
A Study to Test if Fremanezumab is Effective in Preventing Chronic Migraine in Patients 6 to 17 Years of Age
The primary objective of the study is to evaluate the effectiveness of fremanezumab as compared to placebo for the preventive treatment of chronic migraine (CM). Secondary objectives are to further demonstrate the efficacy of Fremanezumab as compared to placebo for the preventive treatment of CM, to evaluate the safety and tolerability of Fremanezumab in the preventive treatment of CM and to evaluate the immunogenicity of Fremanezumab and the impact of antidrug antibodies (ADAs) on clinical outcomes in participants exposed to Fremanezumab The total duration of the study is planned to be 48 months.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Kiley.Poppino@UTSouthwestern.edu
• The participant has a clinical history of recurrent headache consistent with the diagnosis of migraine for at least 6 months before screening, consistent with ICHD-3 criteria (Headache Classification Committee of the IHS 2013), and a history of ≥15 headache days per month, of which ≥8 headache days were assessed as migraine days per month in each of the 3 months prior to screening (visit 1).
• The participant or parent/caregiver maintain a prospectively collected headache diary
• The participant does not have chronic daily headache. For the purposes of this study, chronic daily headache is operationally defined as <4 headache-free days during the 28-day baseline period. NOTE: Additional criteria apply; please contact the investigator for more information.
• The participant is using medications containing opioids (including codeine) or barbiturates (including Fiorinal®, Fioricet®, or any other combination containing butalbital) for the treatment of migraine during the 3 months prior to the day of the screening visit.
• The participant has used an intervention/device (eg, scheduled nerve block or transcranial magnetic stimulation) for the treatment of migraine or in the head or neck area for any condition during the 2 months prior to the day of the screening visit.
• The participant has a current history of a clinically significant psychiatric condition, any prior history of a suicide attempt, or a history of suicidal ideation with a specific plan within the past 2 years, at the discretion of the investigator.
• The participant has an ongoing infection or a known history of human immunodeficiency virus infection, tuberculosis, Lyme disease, or chronic hepatitis B or C, or a known active infection of coronavirus disease 2019 (COVID-19).
• The participant has a past or current history of cancer.
• The participant is pregnant, nursing.
• The participant has a history of hypersensitivity reactions to injected proteins, including mAbs, or a history of Stevens-Johnson Syndrome or toxic epidermal necrolysis syndrome, or the participant is concomitantly using lamotrigine.
• The participant received a live attenuated vaccine (eg, intranasal flu vaccine, and measles, mumps, and rubella vaccine) within the 12-week period prior to screening. Note: If a medical need arises during the study, the participant may receive a live attenuated vaccine.
• The patient has a current or past medical history of hemiplegic migraine. NOTE: Additional criteria apply; please contact the investigator for more information.
A Study to Compare the Safety and Efficacy of Dysport® and Botox® in Adults With Upper Limb Spasticity. (DIRECTION)
This study is aiming to demonstrate the non-inferiority of AbobotulinumtoxinA (aboBoNT-A) versus OnabotulinumtoxinA (onaBoNT-A) as the primary safety endpoint, and the superiority of aboBoNT-A over onaBoNT-A with respect to duration of response as the key secondary efficacy endpoint when used at optimal doses according to approved prescribing information of each product.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Victoria.Castillo@UTSouthwestern.edu
• Participant must be 18 to 75 years of age inclusive, at the time of signing the informed consent
• 2a. [US/France] Participants with stable Upper Limb Spasticity (ULS) for at least 3 months, in whom treatment of only one upper limb is necessary for the duration of the study;
• 2b. [Canada] Participants with stable post-stroke ULS for at least 3 months, in whom treatment of only one upper limb is necessary for the duration of the study
• Participants who are either naïve to Botulinum toxin type A (BoNT-A) for ULS or who have been previously treated with BoNT-A for ULS;
• Participants with MAS score of at least 2 at elbow, wrist and finger flexors;
• Participants with DAS score of at least 2 on the Principal Target of Treatment (PTT) (one of four functional domains: dressing, hygiene, limb position and pain);
• Participants who require BoNT-A injection in all of the following muscles: flexor carpi radialis, flexor carpi ulnaris, flexor digitorum profundus, flexor digitorum superficialis and biceps brachii;
• Participants for whom injection of a total dose of 900 Units aboBoNT-A or 360 Units onaBoNT-A is considered by the investigator to be clinically appropriate;
• Participants who have been stable for at least 3 months prior to study entry in terms of oral antispasticity, anticoagulant and/or anticholinergic medication if treated, and for at least 1 month prior to study entry in terms of occupational and/or physiotherapy treatment, if treated, and are considered by the investigator likely to remain stable for the duration of the study;
• Major limitations in the passive range of motion in the paretic upper limb;
• Major neurological impairment (other than limb paresis) that could negatively affect functional performance;
• Participants clinically requiring injection into any upper limb muscles other than the five muscles of one arm listed in Section 5.1, or requiring injection into both arms or any lower limb within the timeframe of the study;
• Hypersensitivity to any BoNT product or excipients;
• Hypersensitivity to cow's milk protein (casein);
• Infection at the proposed injection site(s);
• Known peripheral motor neuropathic diseases, amyotrophic lateral sclerosis or neuromuscular junction disorders (e.g. myasthenia gravis or Lambert-Eaton syndrome);
• Any medical condition (including dysphagia or breathing difficulties/compromised respiratory function) that in the opinion of the investigator, might jeopardize the participant's safety;
• Women who are pregnant or lactating
• Participants treated with BoNT of any type for any indication (e.g. bladder injection, headache or cosmetic) within the previous 12 weeks or planned/likely to be treated during the course of the study;
• Prior history of non-responsiveness to BoNT treatment;
• Previous surgery, or administration of alcohol or phenol in the study limb 6 months or earlier from study enrolment or planned/likely to be treated during the course of the study;
• Participants treated with intrathecal baclofen, aminoglycosides or other agents interfering with neuromuscular transmission (e.g. curare-like agents), within the previous 4 weeks or planned/likely to be treated during the course of the study;
• Participants who received a COVID-19 vaccine injection within 7 days before the first planned study intervention injection, or planned/likely to be injected within 7 days after the first planned study intervention injection
• BoNT naïve participants with a history of facial neurogenic disorder (facial paralysis, polyradiculoneuropathy) (only for France).
A Trial to Investigate Long Term Efficacy and Safety of Lonapegsomatropin in Adults With Growth Hormone Deficiency
This is a phase 3 open-label multicenter extension study designed to evaluate the long-term safety and efficacy of Lonapegsomatropin administered once-weekly. The study participants are adults (males and females) with confirmed growth hormone deficiency (GHD) having completed the treatment period in study TCH-306 (foresiGHt).
Call 214-648-5005
studyfinder@utsouthwestern.edu, Natalie.Booker@UTSouthwestern.edu
• Signing of the trial specific informed consent
• Completion of the treatment period and Visit 7 assessments of trial TCH-306, including collection and upload of Visit 7 DXA scan
• Fundoscopy at Visit 7 in trial TCH-306 without signs/symptoms of intracranial hypertension or diabetic retinopathy stage 2 / moderate or above
• Diabetes mellitus if any of the following are met:
• Poorly controlled diabetes, defined as HbA1C higher than 7.5% according to central laboratory at Visit 6 in trial TCH-306
• Use of diabetes mellitus drugs other than metformin and/or dipeptidyl peptidase-4 (DPP-4) inhibitors
• Active malignant disease or history of malignancy. Exceptions are:
• Resection of in situ carcinoma of the cervix uteri
• Complete eradication of squamous cell or basal cell carcinoma of the skin
• Known history of hypersensitivity and/or idiosyncrasy to the investigational product (somatropin or excipients)
• Female who is pregnant, plans to become pregnant, or is breastfeeding
• Female participant of childbearing potential (i.e., fertile, following menarche and until becoming post-menopausal unless permanently sterile) not willing throughout the trial to use contraceptives as required by local law or practice. Details included in Appendix 4/section 10.4 of the protocol
• Male participant not willing throughout the trial to use contraceptives as required by local law or practice. Details included in Appendix 4/ section 10.4 of the protocol
• Any disease or condition that, in the judgement of the investigator, may make the participant unlikely to comply with the requirements of the protocol or any condition that presents undue risk from the investigational product or trial procedures
A Study of HMBD-002, a Monoclonal Antibody Targeting VISTA, as Monotherapy and Combined With Pembrolizumab
This is a phase 1/2, open-label, multi-center, first-in-human, two-stage (Part 1: dose escalation and Part 2: dose expansion) study evaluating multiple doses and schedules of intravenously (IV) administered HMBD-002, with or without pembrolizumab, in patients with advanced solid tumors (i.e., locally advanced and unresectable, or metastatic).
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• Histologic or cytologic evidence of a malignant solid cancer (any histology) with advanced or metastatic disease and no available therapies known to confer clinical benefit.
• Tumor tissue, or paraffin block, ideally from the patient's most recent biopsy. A fresh tumor biopsy will be obtained if archival samples are not available.
• Measurable by Response Evaluation Criteria in Solid Tumors (RECIST), version 1.1.
• At least 18 years old.
• An Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 1.
• Adequate hematopoietic, kidney, and liver functions.
• A left ventricular ejection fraction (LVEF) ≥ 45%.
• Women of childbearing potential (WOCBP) must not be pregnant or breastfeeding. A WOCBP must agree to follow contraceptive guidance during the treatment period and for at least 120 days after the last dose of study treatment.
• Male subjects must agree to follow contraceptive guidance during the study period and for at least 120 days after the last dose of study treatment.
• Patient must give informed written consent for the study. Inclusion Criteria for HMBD-002 Phase 2 Stage Triple Negative Breast Cancer (TNBC)
• Histologic or cytologic evidence of TNBC that is advanced or metastatic.
• Will be requested to undergo a tumor biopsy before treatment and after 6 weeks of treatment.
• Must have received appropriate treatment with at least one prior regimen for TNBC and there are no available therapies known to confer clinical benefit. Non-Small Cell Lung Cancer (Monotherapy and Combination)
• Histologic or cytologic evidence of NSCLC that is advanced or metastatic.
• Will be requested to undergo a tumor biopsy before treatment and after 6 weeks of treatment.
• Absence of an activating mutation of the EGFR or ALK.
• Must have received treatment with an approved therapy if there are other genomic aberrations for which targeted therapies are approved and available.
• Must have had disease progression on at least one approved or comparable standard therapy for NSCLC.
• Must have received appropriate prior treatment with a mAb to PD-1 or PD-L1. Multiple Other Cancers (Combination Therapy Baskets)
• Histologic or cytologic evidence of an advanced or metastatic cancer aside from TNBC and NSCLC with no available therapies known to confer clinical benefit.
• Will be requested to undergo a tumor biopsy before treatment and after 6 weeks of treatment.
• Must have had appropriate treatment for their specific cancer and there is an absence of available therapy with a reasonable likelihood of conferring clinical benefit. Exclusion Criteria
• If the patient received prior therapy with an anti-PD-1 or anti-PD-L1 mAb or with an agent targeting stimulatory or co-inhibitory T-cell receptor and was discontinued from that treatment due to a Grade 3 or higher immune related adverse event.
• Received radiotherapy within 2 weeks of treatment.
• Received radiotherapy exceeding 30 Gray (Gy) to the lung within 6 months of the first dose of study medication.
• Received an allogeneic tissue/solid organ transplant.
• Received a live or live-attenuated vaccine within 30 days prior to the first dose of study medication.
• Received a VISTA targeting agent.
• The patient must have recovered from all AEs due to previous therapies to ≤Grade 1 or baseline.
• The patient has an active autoimmune disease that required systemic treatment in the past.
• Presence of an uncontrolled endocrine disorder.
• Presence of clinically significant cardiovascular disease.
• History of (non-infectious) pneumonitis or interstitial pulmonary disease that required steroids or has current pneumonitis or interstitial pulmonary disease.
• Presence of uncontrolled, clinically significant pulmonary disease.
• A previous a severe hypersensitivity reaction (≥ Grade 3) to pembrolizumab and/or any of its excipients.
• A diagnosis of immunodeficiency or is receiving chronic systemic corticosteroids at a dose that exceeds 10 mg daily of prednisone equivalent or any other form of immunosuppressive therapy within 7 days prior the first dose of study drug. Inhaled or topical steroids are permitted in the absence of active autoimmune disease.
• An uncontrolled intercurrent illness that would limit compliance with the study.
• A positive status for human immunodeficiency virus (HIV).
• A known history of Hepatitis B (defined as HBsAg reactive) or known active Hepatitis C viral (defined as HCV RNA detected) infection.
• Oxygen-dependence.
• A medical condition which, in the opinion of the Investigator, places the patient at an unacceptably high risk for toxicity.
• A positive COVID test within one week of study treatment if not fully vaccinated.
• Another active malignancy that is progressing or has required active treatment within the past 3 years.
• Known active central nervous system metastases and/or carcinomatous meningitis.