This is a Phase 3, randomized, double-blind, ELX/TEZ/IVA-controlled, parallel-group, multicenter study to evaluate the efficacy of VX-21/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in cystic fibrosis (CF) subjects who are heterozygous for F508del and a minimal function mutation (F/MF subjects).

The objective of this study is to determine the safety and efficacy of transcatheter aortic valve replacement (TAVR) via a transfemoral approach in HF patients with moderate AS as compared with OHFT. International, multi-center, randomized, open-label, clinical trial comparing the safety and efficacy of TAVR with the SAPIEN 3 or SAPIEN 3 Ultra THV and OHFT versus OHFT in HF patients, with moderate AS. All patients are followed from randomization until at least 1 year after randomization of the last patient. Patients are followed from randomization until 1 year after randomization for the last patient (efficacy assessment time interval [EATI]).

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare neurological disorder in which there is inflammation of nerve roots and peripheral nerves and destruction of the fatty protective covering (myelin sheath) over the nerves. There is an unmet medical need for an efficacious treatment with a favorable safety and tolerability profile and more convenient administration than that provided by current treatments. A weekly subcutaneous (SC) treatment option consisting of efgartigimod PH20 SC administered within a few minutes could offer clinically significant benefits to CIDP patients. Stage A (open-label, efgartigimod PH20 SC; 4-12 weeks) Primary objective: to assess the activity of efgartigimod PH20 SC based on the percentage of patients classified as treatment responders. Stage B (double-blind, randomized-withdrawal, efgartigimod PH20 SC or placebo; up to 48 weeks) Primary objective: to determine the efficacy of efgartigimod PH20 SC compared to placebo based on the time needed for the occurrence of the first evidence of clinical deterioration.

A multicenter, randomized, adaptive allocation clinical trial to determine if increasing durations of induced hypothermia are associated with an increasing rate of good neurological outcomes and to identify the optimal duration of induced hypothermia for neuroprotection in comatose survivors of cardiac arrest.

The purpose of this study is to compare how well axatilimab helps cGVHD at different dose levels and to find out whether there are side effects from its use.

The primary objective for this observational study is to collect general and medical data from children, adolescents, and young adults who had pediatric onset rheumatic disease. This data will be used to evaluate the long-term safety and efficacy of therapeutic agents used to treat these diseases. This information will allow investigators to accurately report and follow changes in current medication use patterns and compare these to proposed standards and current treatment recommendations. The use of a single registry will allow for more analysis of the different therapeutic agents by allowing them to be compared to each other.

This study is evaluating the efficacy of MultiStem (drug) on functional outcome in participants with ischemic stroke.

This is a randomized, double-blind, placebo-controlled, parallel-group study with an optional open-label extension testing the safety and efficacy of inebilizumab in participants with Myasthenia Gravis.

This study is designed for the following purpose: - To assess the risk of delayed adverse events (AEs) following exposure to genemodified(GM) T cells - To monitor for long-term persistence of GM T cells, including analysis of vector integration sites, as appropriate. - To monitor for generation of replication competent retroviruses (RCR) - To assess long-term efficacy following treatment with GM T cells - Describe growth, developmental outcome, and sexual maturity status for subjects who were aged < 18 years at time of GM T cell treatment - To assess long term health-related quality of life following treatment with GM T cells

TRACK-FA is a multi-site, international, prospective, observational neuroimaging study in Friedreich’s Ataxia. The objective is to obtain natural history MRI and MRS data over time in the brain and spinal cord in a large cohort of FRDA patients and healthy control. This will permit validation of neuroimaging biomarkers in FRDA for future clinical trials. In addition to neuroimaging data, the study will collect data from clinical, cognitive and mood assessments as well as blood samples to measure blood markers (frataxin and neurofilament light chain). The present IRB protocol covers only subjects enrolled at the University of Minnesota.

The Sponsor of this study, ModernaTX, is studying the mRNA-1273 vaccine for the prevention of COVID-19 in children. This study is being conducted to learn about the safety, any side effects, and how your child’s body responds to the study vaccine (the “immune response”).

This study is intended to determine the clinical benefit of tabelecleucel (EBV-specific cytotoxic T-lymphocytes) in subjects with EBV-associated diseases.

This is a prospective randomized clinical trial, to determine whether dose-intensive tandem Consolidation, in a randomized comparison with single cycle Consolidation, provides an event-free survival (EFS) and overall survival (OS). The study population (children < 120 months) will be high-risk patients (non-Wnt and non-Shh sub-groups) with medulloblastoma, and for all patients with central nervous system (CNS) embryonal tumors completing "Head Start 4" Induction. This study will further determine whether the additional labor intensity (duration of hospitalizations and short-term and long-term morbidities) associated with the tandem treatment is justified by the improvement in outcome. It is expected that the tandem (3 cycles) Consolidation regimen will produce a superior outcome compared to the single cycle Consolidation, given the substantially higher dose intensity of the tandem regimen, without significant addition of either short-term or long-term morbidities.

The primary objective of this research project is development and validation of a new, non-contrast gated aortic (NCGA) computer tomography scan algorithm for screening of aortic aneurysm in the chest and abdomen in at risk patients. This study would initially be performed in patients with a known aneurysm and done in addition to their indicated surveillance CT scan.

This is a phase II study of FDA-approved CAR-T products for patients with hematologic malignancies. The study provides criteria for consistent treatment and management according to FDA labelling of CAR-T products and does not contain experimental components. Patients will be assigned to Arms A B and C based on age, CAR-T product and diagnosis. Overall remission rate, safety events and other endpoints will be calculated for Arms A B and C separately.

This study will investigate the efficacy and safety of REC-2282 in patients with progressive NF2 mutated meningiomas who have either NF2 disease-related meningioma or recurrent sporadic meningiomas that have NF2 mutations. This study is a parallel-group, two-staged, Phase 2/3, randomized, multi-center study with two cohorts: Cohort A followed by Cohort B. The purpose of Cohort A is to provide early data on efficacy and safety of REC-2282 in participants with progressive NF2 mutated meningiomas, and provide guidance for the correct dose, population, sample size, and endpoint for the confirmatory part of the study (Cohort B). Additional goals for Cohort A are to assess effects of food on drug absorption. The purpose of Cohort B of the study is to assess the efficacy and safety of REC-2282 compared with placebo in participants with progressive NF2 mutated meningiomas.

In this sequential, multiple assignment, randomized trial (SMART) current smokers who are eligible for lung cancer screening will be identified using the electronic medical record at the University of Minnesota, Minneapolis VA, and Allina Health (N=1000). All participants will receive 8 weeks of evidence-based first-line smoking cessation treatment. Participants will be eligible for three potential randomizations during one year of smoking intervention: 1) to timing of identifying early response to treatment at 4 vs. 8 weeks (all participants), 2) to telephone-based tobacco longitudinal care (TLC) vs. TLC plus pharmacist-administered Medication Therapy Management (incomplete responders to first-line treatment, Primary Aim), and 3) to monthly TLC contact vs. quarterly TLC contact (complete responders to first-line treatment, Secondary Aim). The primary outcome will be 6 months of prolonged abstinence measured 18 months after the beginning of treatment.

The purpose of this study is to collect information on how patients with PE recover after treatment with the Indigo Aspiration System. The Indigo Aspiration System is a medical device that has been cleared by the U.S. Food and Drug Administration (FDA) for removing blood clots from the blood vessels throughout the body, excluding the head. The device is commercially available globally. Participants will be in this research study for about one year. Participants will be asked to complete a screening and baseline visit, device procedure in-patient visit as part of routine treatment for their PE, one post-procedure visit in the hospital and two follow-up visits. The study team will collect information on tests and procedures done during these visits from their medical records. They will also be asked to complete a quality of life questionnaire.

This is a two stage phase II trial of HLA-haploidentical related hematopoietic cells transplant (Haplo-HCT) using a reduced intensity conditioning (cyclophosphamide, fludarabine, melphalan, total body irradiation) with modifications based on factors including age and comorbidities. Bone marrow is the donor graft source. This study is designed to estimate disease-free survival (DFS) at 1 year posttransplant. Since the goal is to estimate the DFS at a long-term time-point (1 year post-transplant), the design is a generalization of the Simon design. Rather than suspension of the trial for evaluation after stage 1, this design uses an optimal interim analysis for futility without suspension of accrual.

The National Institutes of Health (NIH)-sponsored collaborative APOL1 Long-term Kidney Transplantation Outcomes Network (APOLLO) is charged with prospectively assessing the effects of renal-risk variants (RRVs) in the apolipoprotein L1 gene (APOL1) on outcomes for kidneys from donors with recent African ancestry and the recipients of their kidneys, after deceased- and living-donor renal transplantation. For the purposes of APOLLO, recent African ancestry is defined as individuals with similar genetic make-up to those currently residing in Africa. APOLLO will also study the impact of APOL1 RRVs on the health of living kidney donors with recent African ancestry.

A Partially-Blind, Randomized, Controlled, Parallel-Group Dose Ranging Study to Determine the Efficacy, Safety and Tolerability of AeroFactTM (SF-RI 1 surfactant for inhalation combined with a dedicated drug delivery system) in Preterm Infants at Risk of Worsening Respiratory Distress Syndrome. To determine an optimal dose of AeroFact™ administered to preterm infants on nCPAP or nIMV vs. nCPAP or nIMV alone in reducing the incidence of intubation/cannulation and bolus surfactant instillation in the first 7 days after birth. To evaluate pulmonary outcomes and respiratory resource utilization at 3, 6, 9, and 12 months PMA

This phase II trial studies OST31-164 as a single agent every 3 weeks for 48 weeks, with 4 doses constituting 1 treatment cycle (12 weeks per cycle). Each patient will receive treatment at a dose of 1x109 CFU until Week 48 or until disease progression, unacceptable toxicity, or the patient meets any other treatment discontinuation criteria. Following treatment discontinuation, all patients will enter a 3-year survival follow-up period. The primary endpoints are disease control during the first 12 months after enrollment and safety assessments (adverse events [AEs], physical examinations, clinical laboratory tests, vital sign measurements, performance status, and tests to monitor for the persistence of Lm).

A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Pediatric Subjects with Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment Participants will be randomized to placebo or Crinecerfont for 28 weeks followed by an open-label period where all participants will receive Crinecerfont for 24 weeks. The purpose of this research study in children (ages 2 to 17 years) with CAH are: • To study whether crinecerfont can lower high levels of adrenal androgens (male hormones) and high doses of glucocorticoid medication • To study whether improving high androgen levels and high glucocorticoid doses can lead to improvements in CAH • To learn about what happens to crinecerfont in the body by measuring levels in your child’s body after starting the study drug. • To study the safety and tolerability of crinecerfont The study will last for about 60 weeks with 14 study visits. The main activities in this study are: physical exams, height and weight measurements, vital signs, study drug dosing, glucocorticoid dose adjustments, blood draws, saliva and urine tests, electrocardiogram (ECG) testing, x-ray for bone age, ultrasound of testes (boys), and questionnaires.