Search Results
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
• 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
A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A
This is a prospective, observational, multi-center longitudinal cohort study to describe the real-world effectiveness, safety and treatment usage of efanesoctocog alfa in patients with hemophilia A treated per standard of care in the US and Japan. Patients will be enrolled in the study after the introduction of efanesoctocog alfa in the hemophilia treatment landscape in each study country. Decision to initiate treatment with commercially available efanesoctocog alfa will be made by the treating physician independently from the decision to include patients in the study. No study medication is provided. The data related to efanesoctocog alfa effectiveness, safety and usage will be collected prospectively during routine visits (expected annual/semi-annual visits) for up to 5 years following enrollment /treatment initiation.
Call 214-648-5005
studyfinder@utsouthwestern.edu, lindsey.hartland@childrens.com
Testing Pump Chemotherapy in Addition to Standard of Care Chemotherapy Versus Standard of Care Chemotherapy Alone for Patients With Unresectable Colorectal Liver Metastases: The PUMP Trial
This phase III trial compares hepatic arterial infusion (HAI) (pump chemotherapy) in addition to standard of care chemotherapy versus standard of care chemotherapy alone in treating patients with colorectal cancer that has spread to the liver (liver metastases) and cannot be removed by surgery (unresectable). HAI uses a catheter to carry a tumor-killing chemotherapy drug called floxuridine directly into the liver. HAI is already approved by the Food and Drug Administration (FDA) for use in metastatic colorectal cancer to the liver, but it is only available at a small number of hospitals, and most of the time it is not used until standard chemotherapy stops working. Standard chemotherapy drugs work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Adding HAI to standard chemotherapy may be effective in shrinking or stabilizing unresectable colorectal liver metastases.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Optimization of Saturation Targets And Resuscitation Trial (OptiSTART) (OptiSTART)
This study is designed to answer one of the fundamental gaps in knowledge in the resuscitation of preterm infants at birth: What is the optimal target oxygen saturation (SpO2) range that increases survival without long-term morbidities? Oxygen (O2) is routinely used for the stabilization of preterm infants in the delivery room (DR), but its use is linked with mortality and several morbidities including bronchopulmonary dysplasia (BPD). To balance the need to give sufficient O2 to correct hypoxia and avoid excess O2, the neonatal resuscitation program (NRP) recommends initiating preterm resuscitation with low (≤ 30%) inspired O2 concentration (FiO2) and subsequent titration to achieve a specified target SpO2 range. These SpO2 targets are based on approximated 50th percentile SpO2 (Sat50) observed in healthy term infants. However, the optimal SpO2 targets remain undefined in the preterm infants. Recent data suggest that the current SpO2 targets (Sat50) may be too low. The investigators plan to conduct a multicenter RCT of Sat75 versus Sat50 powered for survival without BPD. The investigators will randomize 700 infants, 23 0/7- 30 6/7 weeks' GA, to 75th percentile SpO2 goals (Sat75, Intervention) or 50th percentile SpO2 goals (Sat50, control). Except for the SpO2 targets, all resuscitations will follow NRP guidelines including an initial FiO2 of 0.3. In Aim 1, the investigators will determine whether targeting Sat75 compared to Sat50 increases survival without lung disease (BPD). In addition, the investigators will compare the rates of other major morbidities such as IVH. In Aim 2, the investigators will determine whether targeting Sat75 compared to Sat50 increases survival without neurodevelopmental impairment at 2 years of age. In Aim 3, the investigators will determine whether targeting Sat75 compared to Sat50 decreases oxidative stress.
Call 214-648-5005
studyfinder@utsouthwestern.edu, shelby.unger@UTSouthwestern.edu
• Prenatally diagnosed cyanotic congenital heart disease
• Prenatally diagnosed congenital diaphragmatic hernia
• Parents request no resuscitation
• If preductal saturations can not be measured by 3 minutes after pulse oximeter sensor is applied to the newborn
Focused Radiation Versus Systemic Therapy for Kidney Cancer Patients With Limited Metastasis, SOAR Study
This phase III trial compares the effect of stero-ablative radiotherapy (SAbR) followed by standard of care systemic therapy, to standard of care systemic therapy alone, in patients with kidney cancer that has spread from where it first started (primary site) to a limited (2-5) number of places in the body (metastatic). Study doctors want to find out if this approach is better or worse than the usual approach for metastatic kidney cancer. The usual approach is defined as the care most people get for metastatic kidney cancer which includes systemic therapy such as immunotherapy (given through the veins) and/or small molecular inhibitor (tablets taken by mouth). Radiotherapy uses high energy x-rays to kill cancer cells and shrink tumors. SAbR uses special equipment to position a patient and deliver radiation to tumors with high precision. Giving SAbR prior to systemic therapy may kill more tumor cells than the usual approach, which is systemic therapy alone.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy (HINALEA 2)
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled are children \<2 years of age genetically diagnosed with SMA.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Tammy.Ramm@UTSouthwestern.edu
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy (HINALEA 1)
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children \< 2 years of age genetically diagnosed with SMA.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Tammy.Ramm@UTSouthwestern.edu
Direct Access Carotid Artery Stenting Using the Neuroguard IEP System (PERFORMANCE III)
The PERFORMANCE III study is a prospective, multicenter single-arm, open label study to evaluate the safety and effectiveness of the Neuroguard IEP® Direct System for the treatment of carotid artery stenosis in subjects at elevated risk for carotid endarterectomy (CEA). Eligible patients greater than or equal to 20 years of age and less than or equal to 80 years of age, are those who have been diagnosed with either de-novo atherosclerotic or post CEA restenotic lesion(s) in the internal carotid arteries (ICA) or at the carotid bifurcation with greater than or equal to 50% stenosis if symptomatic or greater than or equal to 70% stenosis if asymptomatic.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Madison.Baehner@UTSouthwestern.edu
• Male and non-pregnant, non-breastfeeding female subjects whose age is ≥ 20 or ≤ 80 years of age.
• Subject is willing and capable of complying with and understands all study protocol requirements, including the specified follow-up visits, and can be contacted by telephone.
• Subject has signed a written informed consent form that has been approved by the local governing Institutional Review Board (IRB) of the respective clinical site.
• Subject is diagnosed with carotid artery stenosis treatable with carotid artery stenting via direct carotid access and is considered a high operative risk for carotid endarterectomy (CEA).
• Subject is diagnosed with either:
• Symptomatic carotid stenosis ≥ 50% as determined by angiography, CTA, or duplex ultrasound. Symptomatic is defined as having stroke, transient ischemic attack (TIA) in the ipsilateral hemisphere supplied by the target vessel carotid lesion or ipsilateral transient monocular blindness (amaurosis fugax) within 180 days prior to the procedure; or
• Asymptomatic carotid stenosis ≥ 70% as determined by angiography, CTA, or duplex ultrasound.
• Subject has a lesion located in the internal carotid artery (ICA) and/or common carotid artery (CCA).
• Subject has a modified Rankin Scale of ≤ 2 at the time of procedure.
• Females of child-bearing potential have a negative pregnancy test within 24 hours prior to the index procedure.
• Subject is willing and able to take dual anti platelet therapy for a minimum of 30 days following the index procedure.
• Subject meets at least one physiologic or one anatomic high-risk criteria. Anatomic High-Risk Conditions for CEA
• Target lesion at or above C2 (level of jaw). 2. Prior head and neck surgery in the region of the carotid artery. 3. Tracheostomy or tracheostoma. 4. Surgically inaccessible lesion or hostile neck which the investigator deems safe for direct carotid access including but not limited to:
• Prior neck irradiation
• Radial neck dissection
• Cervical spine immobility 5. Prior ipsilateral CEA. 6. Prior cranial nerve injury. 7. Severe tandem lesions. 8. Occlusion of the contralateral CCA or ICA. 9. Severe bilateral ICA stenosis. Physiological High-Risk Conditions for CEA
• Subject is ≥ 70 years of age (maximum 80 years) at the time of enrollment.
• Subject has NYHA Class III or IV congestive heart failure (CHF).
• Subject has chronic obstructive pulmonary disease (COPD) with FEV1 \< 50, on intermittent or chronic oxygen therapy, or a resting PO2 of ≤ 60 mmHg (room air). 4 Subject has left ventricular ejection fraction (LVEF) ≤ 35%. 5. Subject has angina class 3 or 4 or unstable angina. 6. Subject has a history of recent myocardial infarction (between 30 days and 6 weeks prior to index the procedure).
• Subject has coronary artery disease with two or more vessels with ≥ 70% stenosis.
• Subject has planned coronary artery bypass grafting (CABG) or peripheral vascular surgery between 31 and 60 days after index procedure.
• Subject has restenosis following a prior carotid endarterectomy (CEA). Angiographic Inclusion Criteria
• Subject has a lesion located in the internal carotid artery (ICA) and/or common carotid artery (CCA).
• Single de novo or restenotic (post carotid endarterectomy \[CEA\]) target lesion or severe tandem lesions that can be covered by a single Neuroguard stent.
• Target lesion is treatable with a single stent of up to 40 mm in length.
• Index vessel diameter (segment covered by the mid-portion of the stent) is between 4.0 mm and 6.0 mm at the site of the target lesion.
• Distal vessel diameter at the site of Neuroguard filter deployment is between 4.0 mm and 7.0 mm.
• Distal common carotid artery diameter (segment covered by proximal portion of the stent) is between 4.0 mm and 8.0 mm.
• Sufficient landing zone exists in the cervical internal carotid artery distal to the target lesion to allow for the safe and successful deployment of the integrated Neuroguard filter.
• At least 5 cm of atherosclerosis free space in the ipsilateral common carotid artery between the sheath insertion site and the proximal edge of the target lesion.
• Common carotid artery reference diameter is at least 6 mm.
• Target vessel must meet diameter requirements as set forth in the Neuroguard IEP Direct System Instructions for Use (IFU). General Exclusion Criteria
• Life expectancy of less than one year in the opinion of the investigator at the time of enrollment.
• Currently requiring an organ transplantation.
• An evolving acute stroke
• Anticipated or existing potential sources of emboli including left ventricular aneurysm, aortic or mitral mechanical heart valve, severe calcific aortic stenosis (valve area \< 1.0 cm2), endocarditis, moderate to severe mitral stenosis, known previously symptomatic patent foramen ovale (PFO), left atrial thrombus, any intracardiac mass.
• Deep being thrombosis (DVT) or pulmonary embolism (PE) treated within the past 12 months.
• Recently (\< 60 days) implanted heart valve.
• Subject has experienced any episode of paroxysmal atrial fibrillation or atrial flutter within the past 6 months or has a history of paroxysmal atrial fibrillation or atrial flutter requiring chronic anticoagulation.
• History of chronic atrial flutter or chronic atrial fibrillation.
• Anticoagulation with Phenprocoumon (Marcumar®), warfarin, direct thrombin inhibitors, or anti-Xa agents within 14 days of the index procedure.
• Subject with a known hypercoaguable state.
• Acute febrile illness (temperature ≥ 100.4°F or 38°C) or active infection.
• Subject with a SARS-CoV-2/COVID-19 infection within 21 days prior to the index procedure.
• Acute myocardial infarction \< 30 days prior to index procedure.
• Any major surgical procedure (i.e., intraabdominal or intrathoracic surgery or any surgery / interventional procedure involving cardiac or vascular system) 30 days prior to or within 30 days following the index procedure.
• History of disabling stroke with substantial residual disability (modified Rankin score ≥ 3).
• Subject has had a transient ischemic attack (TIA) or amaurosis fugax within 48 hours prior to the index procedure.
• Known severe carotid stenosis contralateral to the target lesion requiring treatment within 30 days of the index procedure.
• Any other neurological deficit not due to stroke that may confound neurological assessments.
• Subject has contralateral laryngeal or vagus nerve injury.
• Subject has severe dementia.
• Subject has intracranial tumor.
• Known hypersensitivity to nitinol or its components (e.g., nickel, titanium).
• History of intracranial hemorrhage within the 12 months prior to the index procedure.
• History of gastrointestinal (GI) bleed within 30 days prior to the index procedure that would interfere with antiplatelet therapy.
• Any condition that precludes proper angiographic assessment or makes direct carotid artery access unsafe (e.g., severe hepatic impairment, malignant hypertension, morbid obesity).
• Subject has less than 5 cm between the direct carotid access site and the proximal edge of the target lesion.
• Known hypersensitivity to contrast media that cannot be adequately premedicated.
• Hemoglobin (Hgb) \< 8 gm/dL, platelet count \< 100,000, international normalized ratio (INR) \> 1.5 (irreversible), or heparin-induced thrombocytopenia.
• Subject has a serum creatinine \> 2.5 mg/dL on the day of the index procedure.
• History or current indication of bleeding diathesis or coagulopathy including thrombocytopenia or an inability to receive heparin in amounts sufficient to maintain an activated clotting time (ACT) at ≥ 250 seconds, or uncorrectable severe anemia.
• Contraindication, intollerance or allergy to standard of care study medications, including antiplatelet therapy or aspirin.
• Previously enrolled in this study or currently enrolled in another interventional device or drug study that has not yet reached the primary endpoint.
• Potential for subject non-compliance with protocol-required follow up or antiplatelet medication in the opinion of the investigator.
• Subject is otherwise unsuitable for intervention or surgery in the opinion of the investigator. Angiographic Exclusion Criteria
• Total occlusion of the target carotid artery.
• Previously placed stent in the target vessel or the planned arteriotomy site.
• Excessive circumferential calcification of the target lesion, defined as \> 3 mm of thickness of calcification seen in orthogonal views on fluoroscopy or on CTA.
• Qualitative characteristics of ipsilateral common carotid artery, ipsilateral external carotid artery, or target lesion that preclude or make difficult the safe introduction of the direct access sheath.
• Angiographic evidence of a mobile filling defect or fresh thrombus in the target carotid artery.
• Presence of "string sign" of the target lesion (a sub-totally occluded, long segment of the true lumen of the artery with markedly reduced contrast flow).
• Non-atherosclerotic carotid stenosis (e.g., dissection, fibromuscular dysplasia).
• Proximal/ostial CCA stenosis ≥ 50% or intracranial stenosis more severe than the target lesion.
• Subject in whom direct carotid access is not possible, including severe tortuosity or stenosis that requires additional endovascular procedures or that prevents safe and expeditious vascular access.
• Subject with intracranial pathology, that in the opinion of the investigator, makes the patient inappropriate for study participation (e.g., arteriovenous malformation, intracranial tumor, microangiopathy or large vessel cerebral vascular disease, etc.) or that would confound the neurological evaluation.
• Angiographic, CT, MR or ultrasound evidence of atherosclerosis of the common carotid artery that would preclude or make difficult safe placement of the sheath and other endovascular devices to the target artery as needed for carotid stenting.
• Angiographic, CT, MR or ultrasound evidence of severe tortuosity of the cervical internal carotid artery. Severe vascular tortuosity is defined as 2 or more bends of 90 degrees or more within 4 cm of the target lesion.
• Angiographic, CT, MR or ultrasound evidence of angulation or tortuosity (≥ 90 degree) of the common carotid artery (CCA) that will transmit a severe loop to the internal carotid after sheath placement.
• Subject with \> 50% stenosis in the common carotid artery (CCA) proximal to the target lesion.
Testing the Effect of M1774 on Hard-to-Treat Refractory SPOP-mutant Prostate Cancer
This phase II trial tests how well M1774 works in treating patients with prostate cancer that does not respond to treatment (refractory) and that has a mutation in the gene responsible for making the speckle type BTB/POZ protein (SPOP). M1774 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving M1774 may be able to shrink or stabilize refractory SPOP-mutant prostate cancer.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
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
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
• 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.
• 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)
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
• Type I hyperlipoproteinemia confirmed by bi-allelic disease-causing variants in any one of the T1HLP genes (LPL, APOC2, APOA5, LMF1, GPIHBP1, or GCKR).
• Fasting serum triglyceride levels of greater than 750 mg/dL.
• Age 8-70 years
• Effective contraception for males and females of childbearing age.
• Off orlistat for a period of 2 months.
• Secondary hypertriglyceridemias due to diabetes, renal disease, hypothyroidism, alcoholism and drug therapy such as estrogens and estrogen analogues, steroids, HIV-1 protease inhibitors, retinoic acid derivatives, interferons, or l-asparaginase.
• On lomitapide or participating in clinical trial of volanesorsen
• 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
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
• 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.
• 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
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
• 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.
• 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).
Safety and Efficacy of Radio Frequency for the Treatment of Mild to Severe Inflammatory Acne
The aim of this trial is to evaluate the safety and efficacy of the InMode RF Pro System with the Morpheus8 face tip (24 pins) applicator for the treatment of mild, moderate and severe, facial acne vulgaris
Call 214-648-5005
studyfinder@utsouthwestern.edu, JENNIFER.BARILLAS@UTSouthwestern.edu
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
Long-Term Safety of Lutetium (177Lu) Vipivotide Tetraxetan in Participants With Prostate Cancer
The purpose of this post-marketing study is to further characterize the long-term outcome of known or potential risks of lutetium (177Lu) vipivotide tetraxetan also known as [177Lu]Lu-PSMA-617 or 177Lu-PSMA-617 and hereinafter referred to as AAA617. The study also seeks to further characterize (as possible) any other serious adverse reaction(s) in the long-term in adults with prostate cancer who received at least one dose of AAA617 from interventional, Phase I-IV Novartis sponsored clinical trials.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
• Signed informed consent must be obtained prior to participation in the study
• Must have received at least one dose of AAA617 within an interventional, Phase I-IV Novartis sponsored clinical trial in prostate cancer and have fulfilled the trial's requirements that allows them to participate in this study.
• Willingness of sexually active participant to use a condom during intercourse for up to 14 weeks from the last dose of AAA617 treatment administered on the parent study.
• Inability to complete the needed investigational examinations due to any reason.
The HistoSonics Edison™ System for Treatment of Primary Solid Renal Tumors Using Histotripsy (#HOPE4KIDNEY) (#HOPE4KIDNEY)
The purpose of this trial is to evaluate the effectiveness and safety of the HistoSonics Edison System for the destruction of kidney tissue by treating primary solid renal tumors.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
• Subject is ≥22 years of age.
• Subject has signed the Institutional Review Board (IRB) approved trial Informed Consent Form (ICF) prior to any trial related tests/procedures and is willing to comply with trial procedures and required follow-up assessments.
• Subject is diagnosed with only one (1) non-metastatic solid renal mass ≤3cm confirmed via CT or MRI ≤30 days prior to the index procedure date.
• Subject has had a biopsy to determine the type of tumor, ≥14 days prior to the index procedure.
• Subject can tolerate general anesthesia.
• Subject has an Eastern Cooperative Oncology Group Performance Status (ECOG PS) grade 0-2 at baseline screening.
• Subject meets all the following functional criteria at ≤14 days prior to the planned index procedure date: * White Blood Count (WBC) ≥3,000/mm3 (≥3 10\*9/L) * Absolute Neutrophil Count (ANC) ≥1,200/mm3 (≥1.2 10\*9/L) * Hemoglobin (Hgb) ≥9 g/dL * Platelet count ≥100,000/mm3 (≥100 10\*9/L) * White Blood Count (WBC) ≤5 hpf via urinalysis * Albumin ≤300 mg/g via urinalysis
• Subject has an eGFR (Glomerular filtration rate) ≥45mL/min, ≤14 days prior to the planned index procedure date.
• The tumor selected for histotripsy treatment must be ≤3cm in longest diameter.
• Subject has an adequate acoustic window to visualize targeted tumor using the HistoSonics Edison System.
• Subject is pregnant or planning to become pregnant or nursing (lactating) during the trial period.
• Subject is being actively treated in another pharmaceutical or device trial ≤30 days prior to planned index procedure date that may interfere with the primary endpoint(s).
• Subjects who have active cancers (not in remission for the last two years) other than non-melanomatous skin cancers.
• In the Investigator's opinion, the subject has co-morbid disease(s) or condition(s) that would cause undue risk and preclude safe use of the HistoSonics Edison System.
• Subject is on dialysis, being considered for dialysis or has acute renal failure.
• Subject has not recovered to Common Terminology Criteria for Adverse Events (CTCAE) grade 2 or better from any adverse effects (except alopecia and neuropathy) related to previous therapy.
• Subject has an International normalized ratio (INR) \>1.5 or uncorrectable coagulopathy, (e.g., known von Willebrand disease, hemophilia, or on anticoagulants), on the planned index procedure date.
• Subject is taking Aspirin (ASA) or NSAIDS ≤14 days prior to the planned index procedure date.
• Subject has a life expectancy less than one (\< 1) year.
• In the investigator's opinion, histotripsy is not a treatment option for the subject.
• Subject has a concurrent condition that could jeopardize the safety of the subject or compliance with the protocol.
• Subject's targeted tumor has had prior locoregional therapy (e.g., ablation, embolization, radiation).
• Subject's targeted tumor is not treatable by the HistoSonics Edison System's working ranges (refer to User Guide).
• In the investigator's opinion, the anticipated risks of intervention outweigh the potential benefits of the intervention.
• Subject has bilateral kidney tumors or has a single functioning kidney.
• Subject has a genetic predisposition to kidney cancer such as: * Von Hippel Lindau (VHL) * Hereditary Papillary Renal Carcinoma (HPRC) * Birt-Hogg-Dubé Syndrome (BHD) * Tuberous Sclerosis Complex (TSC) * Hereditary Leiomyomata's Renal Cell Carcinoma (HLRCC) * Reed's Syndrome * Succinate Dehydrogenase B Deficiency (SDHB) * BRCA 1 associated protein -1 (BAP1) Renal Cell Carcinoma * MITF predisposed Renal Cell Carcinoma
• The targeted tumor is an angiomyolipoma.
• Subject has a known sensitivity to contrast media and cannot be adequately pre-medicated.
• The targeted tumor is not clearly visible with ultrasound, MRI or CT.
• Targeted tumor with adequate margin overlaps the renal pelvis, main renal vessel, ureter, or other vital structure.
• The treatment of the tumor will not allow an adequate margin (as determined by the investigator).
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
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
• 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 ≤4 months after most recent tumor resection
• Ability to consent in English or Spanish
• 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.
A Study to Compare Darolutamide Given With Androgen Deprivation Therapy (ADT) With ADT in Men With Hormone Sensitive Prostate Cancer and Raise of Prostate Specific Antigen (PSA) Levels After Local Therapies (ARASTEP)
Researchers are looking for a better way to treat men at high-risk of biochemical recurrence (BCR) of prostate cancer. BCR means that in men who had prostate cancer and were treated by either surgery and/ or radiation therapy, the blood level of a specific protein called PSA rises. PSA is a marker of prostate cancer cells activity. The PSA increase means that the cancer has come back even though conventional imaging such as computed tomography (CT) scans, magnetic resonance imaging (MRI) and bone scans does not show any lesion of prostate cancer. Recently a more sensitive imaging method called prostate-specific membrane antigen \[PSMA\] positron emission tomography \[PET\]) /computed tomography \[CT\]) scan may identify prostate cancer lesions not detectable by conventional imaging. Men with BCR have a higher risk of their cancer spreading to other parts of the body, particularly when PSA levels raised to a certain limit within a short period of time after local therapies. Once the cancer spreads to other parts of the body, it can become even harder to treat. In men with prostate cancer, male sex hormones (also called androgens) like testosterone can help the cancer grow and spread. To reduce androgens levels in these patients, there are treatments that block androgens production in the body called androgen deprivation therapy (ADT). ADT is often used to stop prostate cancer. Another way to stop prostate cancer growth and spread is to block the action of androgen receptors on prostate cancer cells called androgen receptor inhibitors (ARIs). The new generation ARIs including darolutamide can block the action of androgens receptors and are available for the treatment of prostate cancer in addition to ADT. It is already known that men with prostate cancer benefit from these treatments. The main objective of this study is to learn if the combination of darolutamide and ADT prolongs the time that the participants live without their cancer getting worse, or to death due to any cause, compared to placebo (which is a treatment that looks like a medicine but does not have any medicine in it) and ADT given for a pre-specified duration of 24 months. To do this, the study team will measure the time from the date of treatment allocation to the finding of new cancer spread in the participants by using PSMA PET/CT, or death due to any cause. The PSMA PET/CT scans is performed using a radioactive substance called a "tracer" that specifically binds to the prostate-specific membrane antigen (PSMA) which is a protein often found in large amounts on prostate cancer cells. To avoid bias in treatment, the study participants will be randomly (by chance) allocated to one of two treatment groups. Based on the allocated treatment group, the participants will either take darolutamide plus ADT or placebo plus ADT twice daily as tablets by mouth. The study will consist of a test (screening) phase, a treatment phase and a follow-up phase. The treatment duration is pre-specified to be 24 months unless the cancer gets worse, the participants have medical problems, or they leave the study for any reason. In addition, image guided radiotherapy (IGRT) or surgery is allowed and your doctor will explain the benefits and risks of this type of therapy. During the study, the study team will: * take blood and urine samples. * measure PSA and testosterone levels in the blood samples * do physical examinations * check the participants' overall health * examine heart health using electrocardiogram (ECG) * check vital signs * check cancer status using PSMA PET/CT scans, CT, MRI and bone scans * take tumor samples (if required) * ask the participants if they have medical problems About 30 days after the participants have taken their last treatment, the study doctors and their team will check the participants' health and if their cancer worsened. The study team will continue to check this and regularly ask the participants questions about medical problems and subse
Call 833-722-6237
canceranswerline@utsouthwestern.edu
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
A Study of AC676 for the Treatment of Relapsed/Refractory B-Cell Malignancies
This clinical trial is evaluating a drug called AC676 in participants with Relapsed/Refractory B-cell Malignancies. The main goals of the study are to: * Identify the recommended dose of AC676 that can be given safely to participants * Evaluate the safety profile of AC676 * Evaluate the pharmacokinetics of AC676 * Evaluate the effectiveness of AC676
Call 833-722-6237
canceranswerline@utsouthwestern.edu
• Adult male and female patients, at least 18 years-of-age at the time of signature of the informed consent form (ICF).
• Patients with histologically confirmed relapsed/refractory Chronic Lymphocytic Leukemia (CLL), Small Lymphocytic Lymphoma (SLL), Mantle Cell Lymphoma (MCL), Follicular Lymphoma (FL), non-GCB Diffuse Large B-cell Lymphoma (DLBCL), Marginal Zone Lymphoma (MZL), or Waldenström Macroglobulinemia (WM).
• Must have received at least 2 prior systemic therapies or have no other therapies to provide significant clinical benefit in the opinion of the Investigator or who are not amenable (intolerability, patient choice) to standard therapies.
• Treatment with any of the following: * Small molecule anti-cancer drugs within 5 half-lives or 2 days (whichever is longer, not to exceed 14 days). * Systemic chemotherapy within 14 days. * Radiation therapy within 14 days * Biologics (Antibodies) treatment within 28 days, * Radioimmunoconjugates or toxin conjugates within 12 weeks. * Prior Chimeric antigen receptor (CAR) T cell therapy (and prior use of immunoglobulin replacement therapy to treat associated adverse events) within 3 months. For patients with DLBCL, no prior CAR- T therapy is allowed. * Autologous or allogenic stem cell transplant within 100 days and must not have ongoing graft-versus-host disease (GVHD) and no ongoing therapy to treat GVHD.
• History of central nervous system lymphoma/leukemia in remission for less than 2 years.
• Medical history of active bleeding within 2 months prior to study entry, or susceptible to bleeding by the judgement of investigator.
A Study of Tegavivint (BC2059) in Patients With Advanced Hepatocellular Carcinoma
This study will be conducted in 2 parts. The first part is a phase 1 single-agent dose escalation,optimization, and expansion study of tegavivint in patients with advanced HCC after failure of at least one line of prior systemic therapy. In the second part of the study, the combination of tegavivint plus pembrolizumab will be assessed with a limited dose escalation followed by a randomized dose optimization.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Safety, Tolerability and Efficacy of AntiBKV as Treatment of BKV Infection in Kidney Transplant Recipients (SAFE KIDNEY II)
The purpose of this study is to evaluate the safety, tolerability, and efficacy of AntiBKV in reducing BKV DNAemia and progression to biopsy-confirmed BKVAN in kidney transplant recipients. This study has an operationally seamless phase II/III design. The phase II part will evaluate the safety of AntiBKV in kidney transplant recipients and establish antiviral proof of concept. The phase II part includes a dose-comparison part to generate additional PK and PD data of AntiBKV. The phase III part will assess the efficacy of AntiBKV in kidney transplant recipients. For both the phase II and phase III parts, participants will be randomized to receive either four doses of AntiBKV or four doses of placebo (every four weeks). In phase II, 60 participants will be first randomized (1:1) to receive either four doses of 1,000 mg of AntiBKV or placebo. In an additional dose-comparison extension, another 30 participants will be enrolled and randomized (1:1:1) to receive either four doses of 1,000 mg AntiBKV, four doses of 500 mg AntiBKV, or placebo. Based on a Day 141 analysis after phase II the sample size for the phase III part of the trial will be defined. Both the phase II and phase III parts will follow identical study assessments and schedules for participants. Eligible participants will receive an intravenous infusion of the investigational medicinal product (IMP) that will be administered four times at a four-week interval. For the first ten participants enrolled in the study, the infusion time will be at least 60 minutes. Provided there are no safety concerns observed with the first ten participants the duration of subsequent infusions will be at least 30 minutes. After administration of the final dose, participants will return as out participants for periodic safety, BKV DNAemia, and PK follow-up assessments until the end of the trial visits, 26 weeks post last IMP application. Regular kidney biopsies will be performed at baseline (prior to infusion) and on Day 141 (8 weeks after full dosing). An additional biopsy will be taken on Day 267 (optional) and if clinically indicated.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Elaine.Bonilla@UTSouthwestern.edu
• Male or female aged 18 years or older
• Kidney transplantation within 24 months prior to enrollment
• Kidney transplant recipient with first-time BKV DNAemia (evaluated during routine clinical monitoring by the local laboratory and acknowledged by a physician within four months prior to Day 1). BKV DNAemia is either defined by BKV-DNAemia of one time \>10,000 copies/mL, or \>1,000 copies/mL sustained for at least one week (confirmed by two consecutive measurements. Note: The second, most recent laboratory value must be acknowledged by a physician within four months prior to Day 1)
• Kidney transplant recipients with adequate and/or stable allograft function as indicated by estimated glomerular filtration rate ((e)GFR) ≥ 30 mL/min
• Female subjects (if of childbearing potential) must agree to use adequate and reliable contraceptive measures throughout their participation in the trial. Contraceptive use by women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
• Ability to provide written informed consent
• Patients with previous diagnosis of BKV DNAemia (defined as one time \> 10,000 copies/mL, or \> 1,000 copies/mL sustained for at least one week (confirmed by two consecutive measurements) since last kidney transplantation
• Known hypersensitivity to any component of the IMP
• Transplanted kidney disease with an estimated glomerular filtration rate ((e)GFR) \< 30 mL/minute at screening
• Uncontrolled acute or chronic infection other than BKV infection at screening which might interfere with study participation at the discretion of the investigator
• Recipients who are treated or planned to be treated with a mTOR inhibitor or belatacept as part of their immunosuppression regimen post-transplantation at the time of enrollment and during the study period
• Recipients who are treated or planned to be treated during study participation with leflunomide at the time of enrollment and during the study period
• Recipients who in the opinion of the investigator are likely to require antibody-depletion therapy during trial participation. Antibody-depletion therapies include but are not necessarily limited to plasmapheresis, immunoadsorption, and intravenous immunoglobulins (IVIg)
• Recipients with active kidney transplant rejection or FSGS
• Recipients who have medical conditions or receive concomitant medications that prevent the recipient from undergoing allograft biopsy
• Recipients with known DSA (de novo or pre-transplantation). Kidney transplant recipients with low-level pretransplant DSAs (\< 1000 mean fluorescence intensity (MFI)) can be included if no impact on the study assessments is expected by the discretion of the investigator.
• (exclusion criterium deleted)
• Recipients with extremely high BKV DNAemia (\> 10,000,000 copies/ml) or hemorrhagic cystitis
• Recipients who in the opinion of the investigator are likely to develop recurrent native kidney disease (e.g. IgA nephritis, FSGS, C3 glomerulonephritis)
• Recipients with a functionally significant ureteral stricture
• Pregnant or nursing (lactating) women
• Known current active or latent TB or any history, in the opinion of the investigator, that confers a risk of reactivation of latent TB and precludes the use of conventional immunosuppression
• History of splenectomy or asplenia
• Any condition, that in the opinion of the investigator, would interfere with the evaluation of the investigational product or interpretation of the participant safety data or study results
• History of malignancy within the past five years, except completely excised basal cell or squamous cell carcinoma of the skin, or cervical carcinoma in situ at least two years prior to screening
• Participation in another interventional clinical trial during trial participation or within 30 days prior to the IMP dosing or planned dosing
• History of alcoholism or drug addiction within one year of screening. Substance use disorder will be an exclusion criterion, at investigator's discretion.
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, STEVE.HOPKINS@UTSouthwestern.edu
• 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
• 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
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
• 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)
• 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.
Safety and Efficacy Study of Viaskin Peanut in Peanut-allergic Children 4-7 Years of Age (VITESSE)
The primary purpose of this study is to assess the efficacy and safety of daily DBV712 250 micrograms (mcg) to induce desensitization to peanut in peanut-allergic children 4-7 years of age over a 12-month treatment period.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Priscilla.Arancivia@UTSouthwestern.edu
• Aged 4 through 7 years at Visit 1 (screening).
• Physician-diagnosed peanut allergy or children with a well-documented medical history of IgE-mediated reactions after ingestion of peanut and currently following a strict peanut-free diet.
• Peanut-specific IgE of >0.7 kilo allergy unit per liter (kUA/L) and a positive peanut SPT with the largest wheal diameter of ≥6 millimeter (mm) at Visit 1.
• An ED of ≤100 mg peanut protein at screening DBPCFC. Key
• Severe generalized dermatologic disease involving the application area (interscapular region)
• Uncontrolled persistent asthma.
• Past or current immunotherapy for peanut allergy, including oral immunotherapy (OIT).
• Current immunotherapy for any allergen (including food allergy, allergic rhinitis and/or insect allergy), or treatment with any monoclonal antibody or biologic immunomodulatory therapy within 6 months prior to Visit 1.
Domvanalimab and Zimberelimab in Advanced Liver Cancers
The goal of this clinical trial is to learn about advanced liver and bile duct cancers. The main question it aims to answer is: If the combination of Domvanalimab and Zimberelimab are effective in treating advanced hepatobiliary cancers that have failed prior treatment.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
• Patient must have a histologically confirmed diagnosis consistent with HCC or bile duct cancer (including intrahepatic cholangiocarcinoma, extrahepatic cholangiocarcinoma, and gall bladder cancers); known fibrolamellar HCC, or combined HCC-cholangiocarcinoma will be excluded.
• Locally advanced or metastatic disease
• 2a. Patients with locally advanced or metastatic disease must have disease deemed not amenable to surgical and/or locoregional therapies or patients who have progressed following surgical and/or locoregional therapies.
• 2b. Measurable disease, as defined as lesions that can accurately be measured in at east one dimension according to RECIST version 1.1 at least 1 cm with contrast enhanced dynamic imaging (magnetic resonance imaging or computed tomography).
• Refractory to or relapsed after prior anti-PD-1/L1 antibody therapy. May have received anti-PD-1/L1 monotherapy or combination therapy as any line of therapy including in the neoadjuvant or adjuvant setting. Patients who discontinued prior immune checkpoint inhibitor treatment due to toxicity are not eligible.
• Availability of recent formalin-fixed, paraffin-embedded (FFPE) tumor tissue block or slides in which the biopsy or resection was performed within 3 years. Baseline tissue can be obtained after consent but must be prior to initiation of zimberelimab and domvanalimab. It is strongly recommended that tissue is obtained from biopsies confirming progression of disease on prior therapy so that the patient has not received any intervening systemic anti-cancer treatment from the time that the baseline tissue was obtained.
• Prior locoregional is allowed provided the following are met: 1) at least 2 weeks since prior locoregional therapy including surgical resection, chemoembolization, radiotherapy, or ablation; 2) target lesion has increased in size ≥25% or the target lesion was not treated with locoregional therapy. Patients treated with palliative radiotherapy for symptoms will be eligible as long as the target lesion is not the treated lesion and radiotherapy will be completed at least 2 weeks prior to study drug administration.
• Age ≥ 18 years
• Child-Pugh Score A or B7-8 (only for Cohort A)
• ECOG Performance score of 0-1
• Adequate organ and marrow function (without chronic, ongoing growth factor support or transfusion in the last 2 weeks) as defined below:
• 9a. Platelet count ≥ 50,000/mm^3
• 9b. Hgb ≥ 8.5 g/dl
• 9c. Absolute neutrophil ≥ 1,000 cells/mm^3
• 9d. Total bilirubin ≤ 3.0 mg/ml (This will not apply to subjects with Gilbert's syndrome who have persistent or recurrent hyperbilirubinemia that is predominantly unconjugated in the absence of hemolysis, and such patients may be enrolled based in consultation with the principal investigator).
• 9e. INR ≤ 2
• 9f. AST, ALT ≤5 times ULN
• 9g. Calculated creatinine clearance (CrCl) ≥ 40 mL/min. CrCl can be calculated using the Cockroft-Gault method.
• 9h. Albumin ≥ 2.0 g/dl
• All men, as well as women of child-bearing potential, defined as not surgically sterilized and between menarche and 1-year post menopause, must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) 4 weeks prior to study entry, for the duration of study participation, and for 120 days after the last dose of zimberelimab or domvanalimab. See contraception guidelines in Appendix 1. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately. A female of child-bearing potential is any woman (regardless of sexual orientation, marital status, having undergone a tubal ligation, or remaining celibate by choice) who meets the following criteria:
• Has not undergone a hysterectomy or bilateral oophorectomy; or
• Has not been naturally postmenopausal for at least 12 consecutive months (i.e., has had menses at any time in the preceding 12 consecutive months).
• Women of child-bearing potential must have a negative serum pregnancy test within 72 hours prior to receiving the first dose of study medication
• Subjects are eligible to enroll if they have non-viral-HCC, or if they have HBV-HCC, or HCV-HCC defined as follows:
• 1. HBV-HCC: Hepatitis B subjects will be allowed if they meet the following criteria: On antiviral therapy for HBV or HBV viral load must be less than 100 IU/mL prior to first dose of study drug. Subjects on active HBV therapy with viral loads under 100 IU/ml should stay on the same therapy throughout study treatment. Subjects who are anti-HBc (+), negative for HBsAg, negative for anti-HBs, and have an HBV viral load under 100 IU/mL do not require HBV anti-viral prophylaxis.
• 2. HCV-HCC: Active or resolved HCV infection as evidenced by detectable HCV RNA or antibody. Patients who have failed HCV therapy as evidenced by detectable HCV RNA will be eligible. Subjects with chronic infection by HCV who are treated (successfully or treatment failure) or untreated are allowed on study. In addition, subjects with successful HCV treatment are allowed as long as there are ≥4 weeks between completion of HCV therapy and start of study drug. Successful HCV treatment definition: SVR12.
• Ability to understand and the willingness to sign a written informed consent.
• Willing and able to comply with the requirements and restrictions in this protocol.
• Patients who have received the vector, protein subunit, or nucleic acid COVID-19 vaccines are eligible to enroll.
• Prior liver transplant.
• Known human immunodeficiency virus (HIV) positive (testing not required).
• Use of any live vaccines against infectious diseases within 28 days of first dose of study drug administration.
• History of trauma or major surgery within 28 days prior to the first dose of study drug administration. (Tumor biopsy or placement of central venous access catheter (eg, port or similar) is not considered a major surgical procedure).
• Underlying medical conditions that, in the investigator's opinion, will make the administration of study drugs hazardous, including but not limited to:
• 5a. Interstitial lung disease, including history of interstitial lung disease or non infectious pneumonitis (lymphangitic spread of cancer is not disqualifying),
• 5b. Active viral, bacterial, or fungal infections requiring parenteral treatment within 14 days of the initiation of study drugs,
• 5c. Clinically significant cardiovascular disease,
• 5d. A condition that may obscure the interpretation of toxicity determination or AEs,
• 5e. History of prior solid-organ transplantation.
• Hypersensitivity to IV contrast; not suitable for pre-medication.
• Pre-existing thyroid abnormality with thyroid function that cannot be maintained in the normal range with medication.
• Any active autoimmune disease or a documented history of autoimmune disease or syndrome that required systemic treatment in the past 2 years (ie, with use of disease-modifying agents, corticosteroids, or immunosuppressive drugs), except for vitiligo or resolved childhood asthma/atopy.
• 8a. Replacement therapy (eg, thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency) is not considered a form of systemic treatment.
• 8b. Participants with asthma who require intermittent use of bronchodilators, inhaled corticosteroids, or local corticosteroid injections will not be excluded from this study. Participants on chronic systemic corticosteroids will be excluded from the study.
• Known history of active bacillus tuberculosis.
• Subjects with a condition requiring systemic treatment with either corticosteroids (> 10 mg/day prednisone equivalent) or other immunosuppressive medications within 14 days of study administration. Inhaled or topical steroids and adrenal replacement doses ≤10 mg/day prednisone equivalents are permitted in the absence of autoimmune disease.
• Known severe hypersensitivity reactions to monoclonal antibodies (≥Grade 3).
• Prior malignancy active within the previous 2 years except for locally curable cancers that have been apparently cured, such as basal or squamous cell skin cancer, superficial bladder cancer, or carcinoma in situ of the cervix, breast, or prostate cancer.
• Prisoners or subjects who are involuntarily incarcerated.
• If a participant has symptomatic or clinically active brain metastases including leptomeningeal disease, they must be excluded if:
• Has evidence of progression by neurologic symptoms
• Has metastatic brain lesions that require immediate intervention.
• Has carcinomatous meningitis, regardless of clinical stability
• Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after contraception and until the termination of gestation, confirmed by a positive hCG laboratory test.
• Has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial.
• Has significant dementia or other mental condition that precludes the participant's ability to consent to the study.
• Use of other investigational drugs (drugs not marketed for any indication) within 28 days or 5 half-lives (whichever is longer) of first dose of study drugs.
• Known hypersensitivity to recombinant proteins, or any excipient contained in the study drug formulations.
First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria
The goal of Parts A and B of this Phase 1, first-in-human, randomized study is to assess the safety, tolerability, and pharmacokinetics (PK) of single (SAD) and multiple (MAD) ascending doses of oral JNT-517 in healthy participants. In Part C, the goal is to evaluate the differences in bioavailability between a tablet and suspension formulation of JNT-517 and the food effect in healthy volunteers. All participants in Part C will receive JNT-517. The goal of Part D is to assess the safety, tolerability, PK, and effect on urinary Phe and other amino acids of JNT-517 in participants with phenylketonuria (PKU). Participants in Part D will receive either JNT-517 or placebo and will be blinded to their treatment assignment. The study consists of 4 parts: * Part A: SAD in healthy participants -randomized, double-blind, placebo-controlled * Part B: MAD in healthy participants (14 days)-randomized, double-blind, placebo-controlled * Part C: Relative bioavailability of 2 formulations and food effect in healthy participants-randomized, open-label * Part D: Phase 1b in participants with PKU (4 weeks)-randomized, double-blind, placebo-controlled In each part, participants will complete a Screening Period, a Treatment Period, and a Follow-up Period for safety.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Juana.Luevano@UTSouthwestern.edu
• Males and females 18 to 55 years of age.
• Medically healthy with no clinically significant medical history.
• Body mass index (BMI) of 18-40 kg/m2 and total body weight \>50 kg (110 lbs).
• Non-smoker for at least 2 weeks prior to dosing and willing to abstain during the study. Part D:
• Males and females 18 to 65 years of age, inclusive.
• Diagnosis of PKU with a confirmed genotype.
• At least 2 plasma Phe levels \>600 μM over the past 12 months.
• BMI of 18-40 kg/m2. All Parts:
• Females of childbearing potential must agree to use 2 highly effective contraceptive methods.
• Capable of giving signed informed consent and able to comply with study procedures. Key
• Any acute or chronic medical condition that would prevent the participant from complying with the procedures or place the participant at risk if they participate in the study.
• Positive for hepatitis B or C or human immunodeficiency virus.
• Any history of malignancy in the last 5 years, excluding non-melanoma skin cancer.
• Any history of liver disease.
• Any surgical or medical conditions that may affect study drug absorption, distribution, metabolism, or excretion.
• Participation in another investigational drug trial within 30 days or, if known, 5 half-lives of the investigational drug (whichever is longer).
• History of drug/alcohol abuse in the last year.
• Current, recent, or suspected infection within 4 weeks of Screening of SARS-CoV-2/COVID-19.
• Received a vaccine for SARS-CoV-2/COVID-19 within 14 days of Screening.
• Unable to tolerate oral medication.
• Allergy to JNT-517 or any component of the investigational product.
• Received \>50 mL of blood or plasma within 30 days of Screening or \>500 mL of blood or plasma within 60 days of Screening.