Duchenne Muscular Dystrophy Clinical Trials in Little Rock, Arkansas

5 studies are recruiting for duchenne muscular dystrophy in Little Rock, Arkansas right now. Each one is listed on ClinicalTrials.gov, the public registry the research teams themselves file with. TrialBoost is not a doctor, a research site, or a study sponsor: the research team decides who takes part.

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Studies now recruiting

1. A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort

   Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed. This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with Duchenne Muscular D…

   Stage

   Phase 1

   Ages

   2 and older

   Site

   Arkansas Children's Hospital, Little Rock, Arkansas

   Locations

   12 in total

   Run by

   Sarepta Therapeutics, Inc.

   See if you can join NCT04626674 on ClinicalTrials.gov

2. A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)

   This is a Phase 3, double-blind, placebo-controlled study with the primary objective of evaluating the efficacy of a single IV infusion of SGT-003 in pediatric ambulant male participants with DMD. The secondary objectives include the evaluation of additional efficacy and safety outcomes. The study …

   Stage

   Phase 3

   Ages

   7 to 11

   Site

   Arkansas Children's Hospital, Little Rock, Arkansas

   Locations

   7 in total

   Run by

   Solid Biosciences Inc.

   See if you can join NCT07160634 on ClinicalTrials.gov

3. Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT

   The goal of this study is to collect additional information on the safety of long-term treatment with AGAMREE® and to explore long-term clinical impact of AGAMREE® on quality of life, as assessed by standardized patient-reported outcome measures (QoL questionnaires) in male patients aged 2 years an…

   Stage

   Not phased

   Ages

   2 and older

   Site

   Arkansas Childrens Hospital, Little Rock, Arkansas

   Locations

   33 in total

   Run by

   Catalyst Pharmaceuticals, Inc.

   See if you can join NCT06564974 on ClinicalTrials.gov

4. A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

   This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 5 cohorts in this study. Cohort 1 will include participants 4 to < 7 years o…

   Stage

   Phase 1

   Ages

   0 to 17

   Site

   Arkansas Children's Hospital, Little Rock, Arkansas

   Locations

   15 in total

   Run by

   Solid Biosciences Inc.

   See if you can join NCT06138639 on ClinicalTrials.gov

5. Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)

   This is a Phase 1b/2 open-label study to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and clinical effects of intravenous (IV) WVE-N531 in patients with Duchenne muscular dystrophy (DMD). To participate in the study, patients must have a documented mutation of the …

   Stage

   Phase 1

   Ages

   4 to 18

   Site

   Arkansas Children's Hospital, Little Rock, Arkansas

   Locations

   5 in total

   Run by

   Wave Life Sciences USA, Inc.

   See if you can join NCT04906460 on ClinicalTrials.gov