Landscape → Rare Disease

Duchenne Muscular Dystrophy Clinical Trial Landscape

Data updated: May 20, 2026

160 clinical trials tracked since 2008

Duchenne Muscular Dystrophy is being studied across 160 clinical trials registered since 2008, with 46 programs currently active. The competitive pipeline includes 10 active Phase 3 trials, 29 active Phase 2 trials, and 16 active Phase 1 trials. Top industry sponsors include Sarepta Therapeutics, Inc., NS Pharma, Inc., Italfarmaco.

46 active

160 total (since 2008)

Phase 1 Active

56 total

Phase 2 Active

97 total

Phase 3 Active

35 total

Phase 4 Active

7 total

Top Industry Sponsors

Sorted by active Active Done Failed

Sarepta Therapeutics, Inc. 3 11 5

NS Pharma, Inc. 3 4 0

Italfarmaco 3 2 0

Solid Biosciences Inc. 3 0 0

BioMarin Pharmaceutical 2 1 4

+ 10 more sponsors — see full table below

Featured Phase 3 Trials

NCT07160634 RECRUITING

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

Solid Biosciences Inc. n=80

NCT04281485 ACTIVE NOT RECRUITING

Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

Pfizer n=114

NCT03373968 RECRUITING

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

Italfarmaco n=206

NCT05693142 RECRUITING

AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)

REGENXBIO Inc. n=65

NCT04587908 ACTIVE NOT RECRUITING

A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy（REACH-DMD）

Taiho Pharmaceutical Co., Ltd. n=104

+ 30 more Phase 3 trials — see full database below

Duchenne Muscular Dystrophy — Trials Started by Year (last 10 years)

2016

2017

2018

2019

2020

2021

2022

2023

2024

2025

2026

TheraRadar.com

Full Trial Database

Complete sponsor table, every trial across all phases, plus competitive analytics. Sortable, filterable, exportable for Pro users.

Top Sponsors (all-time)

Sarepta Therapeutics, Inc.

NS Pharma, Inc.

Italfarmaco

Solid Biosciences Inc.

BioMarin Pharmaceutical

Roche

Wave Life Sciences Ltd.

Avidity Biosciences, Inc.

Satellos Bioscience, Inc.

Entrada Therapeutics, Inc.

Active Trials by Phase

Phase 1

Phase 2

Phase 3

Phase 4

Enrollment Size Distribution

1-50

112

51-200

201-500

501-1k

1k+

All Industry Sponsors

Sorted by active Active Done Failed

Sarepta Therapeutics, Inc. 3 11 5

NS Pharma, Inc. 3 4 0

Italfarmaco 3 2 0

Solid Biosciences Inc. 3 0 0

BioMarin Pharmaceutical 2 1 4

Roche 2 1 1

Wave Life Sciences Ltd. 2 1 1

Avidity Biosciences, Inc. 2 1 0

Satellos Bioscience, Inc. 2 0 0

Entrada Therapeutics, Inc. 2 0 0

Edgewise Therapeutics, Inc. 2 0 0

Santhera Pharmaceuticals 1 2 2

Pfizer 1 0 4

Capricor Inc. 1 2 0

Taiho Pharmaceutical Co., Ltd. 1 2 0

NCT07160634 RECRUITING

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

Solid Biosciences Inc. n=80

NCT04281485 ACTIVE NOT RECRUITING

Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

Pfizer n=114

NCT05967351 ENROLLING BY INVITATION

A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study

Sarepta Therapeutics, Inc. n=400

NCT03373968 RECRUITING

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

Italfarmaco n=206

NCT05693142 RECRUITING

AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)

REGENXBIO Inc. n=65

NCT04587908 ACTIVE NOT RECRUITING

A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy（REACH-DMD）

Taiho Pharmaceutical Co., Ltd. n=104

NCT05881408 ACTIVE NOT RECRUITING

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

Sarepta Therapeutics, Inc. n=148

NCT05933057 RECRUITING

Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy

Italfarmaco n=138

NCT05195775 ACTIVE NOT RECRUITING

Tadalafil as Adjuvant Therapy for DMD

University of Florida n=25

NCT07587242 NOT YET RECRUITING

A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping

Avidity Biosciences, Inc. n=70

NCT01247207 COMPLETED

Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

PTC Therapeutics n=270

NCT01648634 COMPLETED

Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy

Assistance Publique - Hôpitaux de Paris n=51

NCT02500381 COMPLETED

Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)

Sarepta Therapeutics, Inc. n=228

NCT05689164 TERMINATED

A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.

Pfizer n=7

NCT05096221 COMPLETED

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)

Sarepta Therapeutics, Inc. n=126

NCT04060199 COMPLETED

Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)

NS Pharma, Inc. n=77

NCT03532542 TERMINATED

An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy

Sarepta Therapeutics, Inc. n=171

NCT04632940 TERMINATED

Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD

Kyntra Bio n=73

NCT04371666 TERMINATED

Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD)

Kyntra Bio n=98

NCT02851797 COMPLETED

Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy

Italfarmaco n=179

NCT03354039 COMPLETED

Tamoxifen in Duchenne Muscular Dystrophy

University Hospital, Basel, Switzerland n=93

NCT01603407 COMPLETED

Finding the Optimum Regimen for Duchenne Muscular Dystrophy

University of Rochester n=196

NCT02814019 TERMINATED

A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids

Santhera Pharmaceuticals n=255

NCT03603288 TERMINATED

Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)

Santhera Pharmaceuticals n=161

NCT01183767 COMPLETED

Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy

Charite University, Berlin, Germany n=33

NCT03907072 TERMINATED

Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy

Wave Life Sciences Ltd. n=6

NCT02255552 COMPLETED

Study of Eteplirsen in DMD Patients

Sarepta Therapeutics, Inc. n=109

NCT03039686 COMPLETED

Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

Hoffmann-La Roche n=166

NCT01557400 COMPLETED

Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada

PTC Therapeutics n=94

NCT03917719 TERMINATED

An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy

Catabasis Pharmaceuticals n=130

NCT02354352 COMPLETED

Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy

Ohio State University n=52

NCT03642145 WITHDRAWN

A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)

PTC Therapeutics

NCT01126697 COMPLETED

Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies

Cooperative International Neuromuscular Research Group n=63

NCT01995032 COMPLETED

L-citrulline and Metformin in Duchenne's Muscular Dystrophy

University Hospital, Basel, Switzerland n=47

NCT00839033 TERMINATED

Evaluation of a Mechanical Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders

Assistance Publique - Hôpitaux de Paris n=14

NCT06128564 ACTIVE NOT RECRUITING

A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)

Hoffmann-La Roche

NCT03368742 ACTIVE NOT RECRUITING

Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

Solid Biosciences Inc.

NCT06138639 RECRUITING

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

Solid Biosciences Inc.

NCT07573631 ENROLLING BY INVITATION

An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy

BioMarin Pharmaceutical

NCT07287189 RECRUITING

Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients

Satellos Bioscience, Inc.

NCT07429240 RECRUITING

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

Precision BioSciences, Inc.

NCT06867107 ENROLLING BY INVITATION

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

Satellos Bioscience, Inc.

NCT06053814 ACTIVE NOT RECRUITING

NS-050/NCNP-03 in Boys With DMD (Meteor50)

NS Pharma, Inc.

NCT06450639 ACTIVE NOT RECRUITING

A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)

Hoffmann-La Roche

NCT06280209 RECRUITING

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy

BioMarin Pharmaceutical

NCT07038824 RECRUITING

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45

Entrada Therapeutics, Inc.

NCT07037862 RECRUITING

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44

Entrada Therapeutics, Inc.

NCT07209332 ENROLLING BY INVITATION

Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy

Wave Life Sciences Ltd.

NCT05996003 RECRUITING

NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)

NS Pharma, Inc.

NCT04906460 RECRUITING

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

Wave Life Sciences Ltd.

NCT07058662 RECRUITING

A Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of BBM-D101 in the Treatment of Duchenne Muscular Dystrophy.

Belief BioMed (Beijing) Co., Ltd

NCT05540860 ACTIVE NOT RECRUITING

A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)

Edgewise Therapeutics, Inc.

NCT06100887 ACTIVE NOT RECRUITING

Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy

Edgewise Therapeutics, Inc.

NCT04428476 ACTIVE NOT RECRUITING

Open-label Extension of the HOPE-2 Trial

Capricor Inc.

NCT04433234 ACTIVE NOT RECRUITING

Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

Daiichi Sankyo Co., Ltd.

NCT06643442 NOT YET RECRUITING

Repurposing Empagliflozin for DMD-associated Cardiomyopathy in Children 6-18 Years of Age

Sebastiano Lava

NCT05524883 ACTIVE NOT RECRUITING

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Dyne Therapeutics

NCT05135663 ACTIVE NOT RECRUITING

Extension Study of NS-089/NCNP-02 in DMD

Nippon Shinyaku Co., Ltd.

NCT06769633 RECRUITING

Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old

Italfarmaco

NCT06290713 RECRUITING

Vasodilator and Exercise Study for DMD (VASO-REx)

University of Florida

NCT06244082 ACTIVE NOT RECRUITING

Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping

Avidity Biosciences, Inc.

NCT03340675 COMPLETED

Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy

Cumberland Pharmaceuticals

NCT04322357 COMPLETED

Twice Weekly Steroids and Exercise as Therapy for DMD

University of Florida

NCT06079736 TERMINATED

A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment

PepGen Inc

NCT05185622 COMPLETED

A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)

Santhera Pharmaceuticals

NCT04240314 COMPLETED

AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.

Megan Waldrop

NCT05670730 COMPLETED

Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping

Avidity Biosciences, Inc.

NCT06833931 WITHDRAWN

A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

PepGen Inc

NCT03333590 COMPLETED

Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy

Kevin Flanigan

NCT04004065 TERMINATED

Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

Sarepta Therapeutics, Inc.

NCT05938023 TERMINATED

A Study of ATL1102 or Placebo in Participants With Non-ambulatory Duchenne Muscular Dystrophy

Percheron Therapeutics

NCT02485938 COMPLETED

HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne)

Capricor Inc.

NCT03375164 COMPLETED

A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)

Sarepta Therapeutics, Inc.

NCT02606136 TERMINATED

Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

Kyntra Bio

NCT04956289 COMPLETED

Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53)

NS Pharma, Inc.

NCT06304064 COMPLETED

Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE)

Capricor Inc.

NCT04336826 COMPLETED

A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

PTC Therapeutics

NCT02667483 COMPLETED

Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

Daiichi Sankyo Co., Ltd.

NCT01761292 COMPLETED

A Study to Assess Safety/Tolerability, pk, Effects on Histology, Clinical Parameters of Givinostat in Children With DMD

Italfarmaco

NCT04179409 COMPLETED

A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.

Kevin Flanigan

NCT02354781 COMPLETED

Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy

Jerry R. Mendell

NCT03985878 TERMINATED

A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)

Sarepta Therapeutics, Inc.

NCT03439670 COMPLETED

A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

ReveraGen BioPharma, Inc.

NCT03558958 TERMINATED

Safety and Efficacy of P-188 NF in DMD Patients

Phrixus Pharmaceuticals, Inc.

NCT03167255 COMPLETED

Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

NS Pharma, Inc.

NCT01099761 TERMINATED

Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy

Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA

NCT04129294 COMPLETED

Exploratory Study of NS-089/NCNP-02 in DMD

National Center of Neurology and Psychiatry, Japan

NCT03648827 COMPLETED

A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

PTC Therapeutics

NCT03796637 COMPLETED

A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren

PTC Therapeutics

NCT02964377 COMPLETED

Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents

Craig McDonald, MD

NCT03218995 COMPLETED

Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping

Sarepta Therapeutics, Inc.

NCT02740972 COMPLETED

Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

NS Pharma, Inc.

NCT03038399 COMPLETED

Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

ReveraGen BioPharma, Inc.

NCT02420379 COMPLETED

Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy

Sarepta Therapeutics, Inc.

NCT01207908 COMPLETED

Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy

Children's Hospital Medical Center, Cincinnati

NCT02310763 TERMINATED

A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy

Pfizer

NCT02907619 TERMINATED

An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy

Pfizer

NCT02515669 TERMINATED

Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD

Hoffmann-La Roche

NCT00759876 TERMINATED

Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)

PTC Therapeutics

NCT02310906 COMPLETED

Phase I/II Study of SRP-4053 in DMD Patients

Sarepta Therapeutics, Inc.

NCT01978366 TERMINATED

Open Label Extension Study of HT-100 in Patients With DMD

Processa Pharmaceuticals

NCT01847573 TERMINATED

Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy

Processa Pharmaceuticals

NCT02819557 COMPLETED

Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy

PTC Therapeutics

NCT01009294 TERMINATED

Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)

PTC Therapeutics

NCT00847379 TERMINATED

Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

PTC Therapeutics

NCT02752048 COMPLETED

A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy

Taiho Pharmaceutical Co., Ltd.

NCT00592553 COMPLETED

Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

PTC Therapeutics

NCT01396239 COMPLETED

Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients

Sarepta Therapeutics, Inc.

NCT01540409 COMPLETED

Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy

Sarepta Therapeutics, Inc.

NCT02858362 TERMINATED

Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrophy (DMD)

Summit Therapeutics

NCT02760277 COMPLETED

An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

ReveraGen BioPharma, Inc.

NCT02525302 TERMINATED

HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02

Akashi Therapeutics

NCT01168908 TERMINATED

Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy

Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

NCT02760264 COMPLETED

A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

ReveraGen BioPharma, Inc.

NCT02167217 COMPLETED

Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy

Washington University School of Medicine

NCT01037309 COMPLETED

Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)

BioMarin Pharmaceutical

NCT02704325 WITHDRAWN

Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2

Kevin Flanigan

NCT02958202 TERMINATED

Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)

BioMarin Pharmaceutical

NCT01826474 TERMINATED

Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy

BioMarin Pharmaceutical

NCT01957059 TERMINATED

A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)

BioMarin Pharmaceutical

NCT02329769 TERMINATED

Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)

BioMarin Pharmaceutical

NCT00844597 COMPLETED

Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients

Sarepta Therapeutics, Inc.

NCT02036463 WITHDRAWN

A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular Dystrophy

Ann & Robert H Lurie Children's Hospital of Chicago

NCT01239758 TERMINATED

Extension Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy

Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA

NCT00758225 COMPLETED

Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)

Santhera Pharmaceuticals

NCT06817382 RECRUITING

A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)

Insmed Gene Therapy LLC

NCT07347548 ACTIVE NOT RECRUITING

A Trial to Investigate the Safety and Pharmacokinetics of GRT6019 in Healthy Male Participants

Grünenthal GmbH

NCT06114056 ACTIVE NOT RECRUITING

A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of Single Intravenous Infusion of JWK007 in Patients With Duchenne Muscular Dystrophy (DMD)

West China Hospital

NCT06692426 RECRUITING

Trial of Cell Based Therapy for DMD

Masonic Cancer Center, University of Minnesota

NCT07172971 NOT YET RECRUITING

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

Larry W. Markham

NCT06224660 RECRUITING

Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy

Sardocor Corp.

NCT07317063 COMPLETED

A Trial to Investigate Safety and Pharmacokinetics of GRT6019, Including Food Effect, in Healthy Male Participants

Grünenthal GmbH

NCT06241950 TERMINATED

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)

Sarepta Therapeutics, Inc.

NCT03362502 TERMINATED

A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy

Pfizer

NCT06597656 TERMINATED

A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74

Sarepta Therapeutics, Inc.

NCT04184882 TERMINATED

A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD)

Astellas Pharma Inc

NCT05338099 COMPLETED

Determine the Safety and Dose of EN001 in Patients With Duchenne Muscular Dystrophy(DMD)

ENCell

NCT02835079 COMPLETED

Treatment Effect of Tamoxifen on Patients With DMD

Hadassah Medical Organization

NCT02246478 COMPLETED

A Study of TAS-205 for Duchenne Muscular Dystrophy

Taiho Pharmaceutical Co., Ltd.

NCT02530905 COMPLETED

Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients

Sarepta Therapeutics, Inc.

NCT02081625 COMPLETED

Exploratory Study of NS-065/NCNP-01 in DMD

National Center of Neurology and Psychiatry, Japan

NCT03508947 COMPLETED

Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy

Wave Life Sciences Ltd.

NCT02241434 WITHDRAWN

Stem Cell Therapy in Duchenne Muscular Dystrophy

Neurogen Brain and Spine Institute

NCT01478022 COMPLETED

To Compare the Pharmacokinetics Profiles of ISO 20, IBU 200 and IBU Plus ISO Combinations 200 + 20

Parent Project, Italy

NCT02295748 COMPLETED

An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort

PTC Therapeutics

NCT02376816 COMPLETED

Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin to Patients With Duchenne Muscular Dystrophy

Jerry R. Mendell

NCT02251600 COMPLETED

A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy

PTC Therapeutics

NCT02516085 COMPLETED

Improved Muscle Function in Duchenne Muscular Dystrophy Through L-Arginine and Metformin

University Hospital, Basel, Switzerland

NCT00873782 COMPLETED

Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy

University of North Carolina, Chapel Hill

NCT02056808 COMPLETED

A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD)

Summit Therapeutics

NCT01335295 COMPLETED

Safety Study of Flavocoxid in Duchenne Muscular Dystrophy

University of Messina

NCT01580501 COMPLETED

PDE Inhibitors in DMD Study (Acute Dosing Study)

Cedars-Sinai Medical Center

NCT01388764 COMPLETED

Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids

Massachusetts General Hospital

Full Duchenne Muscular Dystrophy Pipeline

Every sponsor, every trial across Phase 1–4. Sortable, filterable, exportable.

Unlock with Pro

Data: ClinicalTrials.gov · Trials registered 2008 onwards · Industry sponsors only