Duchenne muscular dystrophy: open trials in United States

• NCT06606340

  A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice

  Research · Sarepta Therapeutics, Inc.

  Kennedy Krieger Institute, Baltimore · UVA Children's Hospital, Charlottesville · Children's Hospital Medical Center, Cincinnati · Nationwide Children's Hospital, Columbus · Duke Lenox Baker Children's Hospital, Durham · Cook Children's Hospital, Fort Worth · University of Florida, Gainesville · Penn State Health Milton S. Hershey Medical Center, Hershey · Texas Children's Hospital, Houston · University of Iowa Hospitals and Clinics, Iowa City · University of Kansas Medical Center, Kansas City · Arkansas Children's Hospital Research Institute, Little Rock · Children's Hospital Los Angeles, Los Angeles · Columbia University Medical Center - PIN, New York · Children's Hospital of Orange County - Main Campus - PIN, Orange · Children's Hospital of Philadelphia, Philadelphia · UPMC Children's Hospital of Pittsburgh, Pittsburgh · University of California, Davis Medical Center, Sacramento · Seattle Children's Hospital, Seattle · Washington University, St Louis · Montefiore Medical Center, The Bronx · Children's National Hospital, Washington D.C. · Atrium Health Wake Forest University Health Sciences, Winston-Salem

  Registry updated 2026-11-02

• NCT07609394

  Duchenne Electronic Health Record Study

  Research · The Duchenne Registry

  Children's Hospital Colorado, Aurora · UT Southwestern Medical Center, Dallas · Duke University Medical Center, Durham · University of Iowa Health Care, Iowa City · Arkansas Children's Hospital, Little Rock · Yale Children's Hospital, New Haven · UC Davis Health, Sacramento · Primary Children's Hospital, Salt Lake City · University Of Utah Health, Salt Lake City · Seattle Children's Hospital, Seattle · Children's National Medical Center, Washington D.C.

  Registry updated 2026-11-02

• NCT05019625

  Biomarker Development for Muscular Dystrophies

  Research · Massachusetts General Hospital

  Boston Children's Hospital, Boston · Massachusetts General Hospital, Boston · Brigham and Women's Hospital, Boston · University of Pittsburgh, Pittsburgh · Wake Forest University, Winston-Salem

  Registry updated 2026-10-29

• NCT06270719

  An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy

  Research · Sarepta Therapeutics, Inc.

  Childrens Hospital Medical Center of Akron, Akron · UNM Clinical and Translational Science Center, Albuquerque · University of Michigan, Ann Arbor · University of Colorado - PPDS, Aurora · Children's of Alabama, Birmingham · Boston Children's Hospital, Boston · University of Virginia, Charlottesville · Ann and Robert H Lurie Childrens Hospital of Chicago, Chicago · Duke Lenox Baker Children's Hospital, Durham · Cook Children's Hospital, Fort Worth · Connecticut Children's Medical Center - Hartford, Hartford · Penn State Health Milton S. Hershey Medical Center, Hershey · Indiana Clinical and Translational Science Institute, Indianapolis · University of Kansas Medical Center, Kansas City · Arkansas Children's Hospital, Little Rock · Children's Hospital Los Angeles, Los Angeles · Norton Children's Neuroscience Institute - Louisville, Louisville · The Board of Regents of the University of Wisconsin, Madison · Nicklaus Children's Hospital, Miami · The Medical College of Wisconsin, Milwaukee · Vanderbilt University Medical Center, Nashville · Children's Hospital of The King's Daughters, Norfolk · Nemours Children's Hospital - Orlando, Orlando · Children's Hospital of Philadelphia, Philadelphia · OHSU Healthcare (Oregon Health and Science University), Portland · Seattle Children's Hospital, Seattle · Stony Brook Medicine Advanced Specialty Care, Stony Brook · All Children's Research Institute, Inc., St. Petersburg · Children's National, Washington D.C.

  Registry updated 2026-10-23

• NCT07429240

  PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)

  Trial Treatment · Phase 1/2 · Precision BioSciences, Inc.

  Arkansas Children's Hospital, Little Rock · Washington University School of Medicine, St Louis

  Registry updated 2026-10-21

• NCT06867107

  An Open-label Long-term Follow-up Study of SAT-3247 for Participants With Duchenne Muscular Dystrophy Including Those Who Participated in SAT-3247-CL-101

  Trial Treatment · Phase 2 · Satellos Bioscience, Inc.

  Nationwide Children's Hospital, Columbus · University of California, Los Angeles, Los Angeles · St. Jude Children's Research Hospital, Memphis · Washington University School of Medicine, St Louis

  Registry updated 2026-10-13

• NCT07766980

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

  Trial Treatment · Phase 2 · University of Florida

  University of Florida, Gainesville

  Registry updated 2026-10-08

• NCT07542314

  Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting

  Trial Treatment · Phase 4 · Sarepta Therapeutics, Inc.

  Baylor College of Medicine, Houston

  Registry updated 2026-10-06

• NCT07160634

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

  Trial Treatment · Phase 3 · Solid Biosciences Inc.

  Neurology Rare Disease Center, Flower Mound · Arkansas Children's Hospital, Little Rock · Children's Hospital of The King's Daughters, Norfolk

  Registry updated 2026-09-28

• NCT06564974

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

  Research · Catalyst Pharmaceuticals, Inc.

  Atrium Health Neurosciences Institute, Charlotte · University of Virginia Health System (UVAHS) - Pediatric Neuromuscular Center, Charlottesville · Lurie Children's Hospital of Chicago, Chicago · Cincinnati Children's Hospital Medical Center, Cincinnati · University of Texas Southwestern Medical Center, Dallas · Neurology Rare Disease Center - Neurology & Neuromuscular Care Center, Denton · Duke University Medical Center and Childrens Health Center, Durham · University of Florida Clinical and Translational Science Institue, Gainesville · Helen DeVos Children's Hospital, Grand Rapids · Penn State Milton S. Hershey Medical Center- Penn State Hershey Neuroscience Institute, Hershey · Indiana University Health Riley Hospital for Children, Indianapolis · University of Kansas Medical Center, Kansas City · Arkansas Childrens Hospital, Little Rock · Loma Linda University Pediatric Subspecialty Clinics, Loma Linda · Children's Hospital Los Angeles, Los Angeles · Valley Children's Hospital, Madera · University of Miami, Miami · Nicklaus Children's Hospital, Miami · Children's Wisconsin, Milwaukee · Masonic Children's Hospital - Discovery Clinic, Minneapolis · University of Massachusetts Memorial Medical Center, North Worcester · Nemours Children's Hospital, Orlando · Stanford University, Palo Alto · Children's Hospital of Philadelphia (CHOP), Philadelphia · Phoenix Children s Hospital, Phoenix · University of California - Davis, Sacramento · The University of Texas Health Science Center at San Antonio, San Antonio · Seattle Children's Hospital, Seattle · Johns Hopkins All Children's Hospital, St. Petersburg · The Children's Hospital at Montefiore, The Bronx · Children's National Health System, Washington D.C.

  Registry updated 2026-09-24

• NCT06066580

  Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy

  Trial Treatment · Phase 2 · Edgewise Therapeutics, Inc.

  Rare Disease Research, Atlanta · UC Denver, Aurora · National Neuromuscular Research Institute, Austin · Kennedy Krieger Institute, Baltimore · Northwestern University, Chicago · University of Cincinnati Gardner Neuroscience Institute, Cincinnati · Nationwide Children's Hospital, Columbus · Neurology Rare Disease Center, Denton · University of Florida, Gainesville · Rare Disease Research, LLC NC, Hillsborough · Indiana University School of Medicine, Indianapolis · University of Iowa, Iowa City · University of Kansas Medical Center, Kansas City · UC San Diego, La Jolla · Arkansas Children's Hospital, Little Rock · UCLA Medical Center, Los Angeles · UC Irvine Medical Center, Orange · Stanford Neuroscience Health Center, Palo Alto · University of Pennsylvania, Philadelphia · Virginia Commonwealth University Health, Richmond · University of Rochester Medical Center, Rochester · UC Davis Medical Center, Sacramento · Washington University School of Medicine, St Louis · University of Massachusetts Memorial Medical Center, Worcester

  Registry updated 2026-09-17

• NCT04626674

  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

  Trial Treatment · Phase 1 · Sarepta Therapeutics, Inc.

  Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago · Nationwide Children's Hospital, Columbus · Duke University Medical Center, Durham · Neurology Rare Disease Center, Flower Mound · University of Florida, Gainesville · University of California San Diego, La Jolla · Arkansas Children's Hospital, Little Rock · University of California, Los Angeles, Los Angeles · Children's Hospital of The King's Daughters, Norfolk · Stanford University, Palo Alto · University of California - Davis, Sacramento · Washington University in St. Louis, St Louis

  Registry updated 2026-09-11

• NCT07172971

  Sodium/Glucose Cotransporter-2 Inhibitors (SGLT2i) Therapy in Duchenne Cardiomyopathy

  Trial Treatment · Phase 1 · Vanderbilt University Medical Center

  Vanderbilt University Medical Center, Nashville

  Registry updated 2026-08-24

• NCT07092540

  The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy

  Research · University of Rochester

  Cincinnati Children's Hospital Medical Center, Cincinnati · Nationwide Children's Hospital, Columbus · University of Rochester, Rochester · Stony Brook Medicine, Stony Brook

  Registry updated 2026-08-19

• NCT05412394

  Once Weekly Infant Corticosteroid Trial for DMD

  Trial Treatment · Phase 4 · Anne M. Connolly

  Lurie Children's Hospital of Chicago, Chicago · University of Cincinnati, Cincinnati · Nationwide Children's Hospital, Columbus · University of Texas, Southwestern, Dallas

  Registry updated 2026-08-14

• NCT07674758

  Modeling Mortality in Duchenne Muscular Dystrophy Cardiomyopathy: Identification of Surrogate Outcome Measures for DMD Drug Trials

  Research · Vanderbilt University Medical Center

  Lurie Children's, Chicago · Nationwide Children's, Columbus · Duke Children's Hospital, Durham · Riley Children's Hospital, Indianapolis · Vanderbilt University Medical Center, Nashville · Children's Hospital of Richmond at VCU, Richmond · UC Davis, Sacramento · Seattle Children's, Seattle · Children's National, Washington D.C.

  Registry updated 2026-07-28

• NCT03836300

  Parent and Infant Inter(X)Action Intervention (PIXI)

  Trial Treatment · RTI International

  RTI International, Research Triangle Park

  Registry updated 2026-07-13

• NCT06138639

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

  Trial Treatment · Phase 1/2 · Solid Biosciences Inc.

  Rare Disease Research, Atlanta · Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago · Nationwide Children's Hospital, Columbus · Arkansas Children's Hospital, Little Rock · University of California Los Angeles Medical Center, Los Angeles · Children's Hospital of The King's Daughters, Norfolk · Children's Hospital of Philadelphia, Philadelphia · Oregon Health and Sciences University, Portland · University of California - Davis, Sacramento · University of California, San Diego · Seattle Children's Hospital, Seattle · Washington University in St. Louis, St Louis

  Registry updated 2026-07-08

• NCT07127978

  A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy

  Research · ITF Therapeutics LLC

  Boston Children's Hospital, Boston · Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago · Duke University / Lenox Baker Children's Hospital, Durham · Arkansas Children's Research Institute, Little Rock · Columbia University Medical Center, New York · Oregon Health and Science University, Portland · Seattle Children's Hospital, Seattle · Children's National Hospital, Washington D.C. · University of Massachusetts Chan Medical School, Worcester

  Registry updated 2026-07-02

• NCT07642635

  Glucagon-Like Peptide-1 Receptor Agonists to Attenuate Metabolic Risk in Individuals With Duchenne Muscular Dystrophy

  Trial Treatment · Phase 1/2 · Vanderbilt University Medical Center

  Vanderbilt University Medical Center, Nashville

  Registry updated 2026-06-11

• NCT07608432

  Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)

  Trial Treatment · Phase 3 · Dyne Therapeutics

  Rare Disease Research, LLC, Hillsborough

  Registry updated 2026-05-27

• NCT06124196

  Wearable Technology to Evaluate Hyperglycemia and HRV in DMD

  Research · Vanderbilt University Medical Center

  Vanderbilt University Medical Center, Nashville

  Registry updated 2026-05-22

• NCT06290713

  Vasodilator and Exercise Study for DMD (VASO-REx)

  Trial Treatment · Phase 2 · University of Florida

  University of Florida Clinical and Translational Research Building, Gainesville

  Registry updated 2026-05-15

• NCT02069756

  The Duchenne Registry

  Research · The Duchenne Registry

  The Duchenne Registry / PPMD, Washington D.C.

  Registry updated 2026-05-08

• NCT06839469

  Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders

  Research · Columbia University

  Boston Children's Hospital, Boston · Columbia University Irving Medical Center, New York · Stanford University, Palo Alto

  Registry updated 2026-04-09

All Duchenne muscular dystrophy trials and the map

This page is not medical advice. Talk to your doctor before contacting a trial.

Report an error