Duchenne Muscular Dystrophy Clinical Trials

17 studies are recruiting for duchenne muscular dystrophy in the United States 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. Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients

   Phase 2a trial of SAT-3247 in ambulatory DMD patients aged ≥ 7 and < 10 years. The trial has two parts. In Part 1, the trial will study two doses of SAT-3247 in a randomized, double-blind, placebo-controlled weekday regimen for 12 weeks to determine the optimal dose, safety, tolerability, and preli…

   Stage

   Phase 2

   Ages

   7 to 9

   Site

   University of California Los Angeles, Los Angeles, California

   Locations

   21 in total

   Run by

   Satellos Bioscience, Inc.

   See if you can join NCT07287189 on ClinicalTrials.gov

2. 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

3. Evaluating VM100 Nutritional Supplement for Improving Quality of Life in Duchenne Muscular Dystrophy Patients

   This pilot study will investigate the potential efficacy of VM100, a nutritional supplement specifically formulated for patients with DMD, on quality of life and physical symptoms. Twenty patients (aged 8 an over) will be enrolled to undergo a 10-week placebo-controlled intervention with VM100. Out…

   Stage

   Not phased

   Ages

   6 and older

   Site

   University of Florida, Gainesville, Florida

   Run by

   University of Florida

   See if you can join NCT07766980 on ClinicalTrials.gov

4. Once Weekly Infant Corticosteroid Trial for DMD

   The hypothesis tested here is that a lower dose of intermittent oral corticosteroids (5mg/kg/week) will be equally effective to the 10mg/kg/week dose.

   Stage

   Phase 4

   Ages

   0 to 2

   Site

   Lurie Children's Hospital of Chicago, Chicago, Illinois

   Locations

   4 in total

   Run by

   Anne M. Connolly

   See if you can join NCT05412394 on ClinicalTrials.gov

5. 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

   The Children's Hospital of Westmead, Sydney, New South Wales

   Locations

   7 in total

   Run by

   Solid Biosciences Inc.

   See if you can join NCT07160634 on ClinicalTrials.gov

6. 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

   Phoenix Children's Hospital, Phoenix, Arizona

   Locations

   33 in total

   Run by

   Catalyst Pharmaceuticals, Inc.

   See if you can join NCT06564974 on ClinicalTrials.gov

7. 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

8. Wearable Technology to Evaluate Hyperglycemia and HRV in DMD

   Duchenne muscular dystrophy (DMD) is an X-linked disorder that causes muscle wasting, cardiopulmonary failure, and premature death. Heart failure is a leading cause of death in DMD, but substantial knowledge gaps exist regarding predisposing risk factors. In the general population, hyperglycemia, i…

   Stage

   Not phased

   Ages

   10 and older

   Site

   Vanderbilt University Medical Center, Nashville, Tennessee

   Run by

   Vanderbilt University Medical Center

   See if you can join NCT06124196 on ClinicalTrials.gov

9. Vasodilator and Exercise Study for DMD (VASO-REx)

   Examining two strategies as potential adjuvant therapies for Duchenne muscular dystrophy (DMD); aerobic exercise training (to induce adaptations in skeletal muscle and improve cardiovascular health) and tadalafil, an FDA-approved vasodilator (to optimize blood flow and muscle perfusion which is imp…

   Stage

   Phase 2

   Ages

   6 and older

   Site

   University of Florida Clinical and Translational Research Building, Gainesville, Florida

   Run by

   University of Florida

   See if you can join NCT06290713 on ClinicalTrials.gov

10. The Duchenne Registry

    The Duchenne Registry is an online, patient-report registry for individuals with Duchenne and Becker muscular dystrophy and carrier females. The purpose of the Registry is to connect Duchenne and Becker patients with actively recruiting clinical trials and research studies, and to educate patients …

    Stage

    Not phased

    Ages

    Any age

    Site

    The Duchenne Registry / PPMD, Washington D.C., District of Columbia

    Run by

    The Duchenne Registry

    See if you can join NCT02069756 on ClinicalTrials.gov

11. Trial of Cell Based Therapy for DMD

    This is a single-center, single-arm, interventional phase 1 trial to evaluate the safety and tolerability of local injection of induced pluripotent stem cell (iPSC)- derived CD54+ allogeneic muscle progenitor cells in individuals with Duchenne muscular dystrophy (DMD)

    Stage

    Phase 1

    Ages

    18 and older

    Site

    Masonic Cancer Center, Minneapolis, Minnesota

    Run by

    Masonic Cancer Center, University of Minnesota

    See if you can join NCT06692426 on ClinicalTrials.gov

12. Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study

    This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne's muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies.

    Stage

    Phase 2

    Ages

    7 and older

    Site

    U.O.S.D. Centro Traslazionale di Miologia e Patologie Neurodegenerative, Building 16 - ground floor IRCCS Istituto Giannina Gaslini,, Genova,

    Locations

    39 in total

    Run by

    Italfarmaco

    See if you can join NCT03373968 on ClinicalTrials.gov

13. 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

14. Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy

    Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies. The investigators are interested in finding less invasive methods that reduce the need for muscle biopsies. The purpose of this research is …

    Stage

    Not phased

    Ages

    5 and older

    Site

    Massachusetts General Hospital, Boston, Massachusetts

    Locations

    2 in total

    Run by

    Massachusetts General Hospital

    See if you can join NCT05016908 on ClinicalTrials.gov

15. Biomarker Development for Muscular Dystrophies

    Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies. The investigators are interested in finding less invasive methods that reduce the need for muscle biopsies. The purpose of this research is …

    Stage

    Not phased

    Ages

    5 and older

    Site

    Brigham and Women's Hospital, Boston, Massachusetts

    Locations

    5 in total

    Run by

    Massachusetts General Hospital

    See if you can join NCT05019625 on ClinicalTrials.gov

16. Wearable Technology to Evaluate Hyperglycemia and HRV in DMD - Longitudinal Aim

    Duchenne Muscular Dystrophy (DMD) is an X-linked disorder that causes muscle wasting, cardiopulmonary failure, and premature death. Heart failure is a leading cause of death in DMD, but substantial knowledge gaps exist regarding predisposing risk factors. In the general population, hyperglycemia, i…

    Stage

    Not phased

    Ages

    10 and older

    Site

    Vanderbilt University Medical Center, Nashville, Tennessee

    Run by

    Vanderbilt University Medical Center

    See if you can join NCT06093100 on ClinicalTrials.gov

17. Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy

    The purpose of this research study is to determine the potential of magnetic resonance imaging, spectroscopy, and whole body imaging to monitor disease progression and to serve as an objective outcome measure for clinical trials in Muscular Dystrophy (MD). The investigators will compare the muscles…

    Stage

    Not phased

    Ages

    5 to 62

    Site

    University of Florida, Gainesville, Florida

    Locations

    3 in total

    Run by

    University of Florida

    See if you can join NCT01484678 on ClinicalTrials.gov