Active DMD Clinical Studies Overview

Last updated:July 2, 2026

Gene Therapy

Gene Therapy active clinical studies
Company	Drug	Phase	Variant Requirement	Status	Locations	Clinical Trial
Sarepta Therapeutics	SRP-9001-402 (Delandistrogene Moxeparvovec) ELEVIDYS	Phase 4	Has an established clinical diagnosis of DMD based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test.	Not Yet Recruiting	United States	NCT07542314
Sarepta Therapeutics	SRP-9001-303 (Delandistrogene Moxeparvovec) ELEVIDYS	Phase 3	Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.	Active, Not Recruiting	United States, Australia, Belgium, Canada, Germany, Hong Kong, Israel, Italy, Japan, South Korea, Spain, Sweden, Taiwan, United Kingdom	NCT05881408
Solid Biosciences	SGT-003	Phase 3	Established clinical diagnosis of DMD and documented DMD gene mutation predictive of DMD phenotype.	Actively Recruiting	United States, Canada, Australia	NCT07160634
REGENXBIO	RGX-202	Phase 2/3	DMD gene mutation in exons 18 and above, and a clinical picture consistent with typical DMD with the exception of a participant (Cohort 1b) with DMD gene mutation in exons 12-17.	Actively Recruiting	United States, Canada	NCT05693142
GENERIUM	GNR-097	Phase 2	Ambulatory boys aged 4-9 years with a documented diagnosis of DMD and clinical manifestations of the disease.	Actively Recruiting	Russia, Belarus	NCT07673809
Hoffmann-La Roche	ENVOL (Delandistrogene Moxeparvovec) ELEVIDYS	Phase 2	Has a definitive diagnosis of DMD prior to screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test	Active, Not Recruiting	Belgium, France, Germany, Italy, Spain, United Kingdom	NCT06128564
Solid Biosciences	SGT-003	Phase 1/2	Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype confirmed by Sponsor genetic testing. In cases where a genotype may be predictive of residual dystrophin production and/or a clear clinical diagnosis of DMD cannot be made (e.g., due to age), evaluation of dystrophin levels in baseline muscle biopsies may be required to determine eligibility under this criterion.	Actively Recruiting	United States, Canada, Italy, United Kingdom	NCT06138639
Solid Biosciences	SGT-001	Phase 1/2	Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype.	Active, Not Recruiting	United States	NCT03368742
Insmed Gene Therapy LLC	INS1201	Phase 1	Has a definitive diagnosis of DMD prior to Screening or as part of Screening based on genetic testing. Note that participants who rescreen do not have to repeat genetic testing for the diagnosis of DMD if one is already on file. Genetic reports must describe a frameshift deletion, frameshift duplication, premature stop ("nonsense"), canonical splice site mutation, or other pathogenic variant in the DMD gene fully contained between exons 18 to 58 (inclusive) that is expected to lead to absence of a functional dystrophin protein (mutations in exons 1-17 or 59-71 are therefore not permitted).	Actively Recruiting	United States	NCT06817382
Sarepta Therapeutics	SRP-9001-103 (Delandistrogene Moxeparvovec) ELEVIDYS	Phase 1	For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.	Actively Recruiting	United States	NCT04626674
Shanghai Jiao Tong University School of Medicine	BBM-D101	Early Phase 1	Genetically confirmed diagnosis of DMD.	Actively Recruiting	China	NCT06641895
Shanghai Siponuoyin Biotechnology Co Ltd	SPOT-mRNA03 (SPOT-03)	Early Phase 1	Boys aged ≥ 2 years to < 8 years and capable of walking independently for at least 10 meters.	Actively Recruiting	China	NCT07188012
Sarepta Therapeutics	SRP-9001-305 (Delandistrogene Moxeparvovec) ELEVIDYS	Interventional	The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received delandistrogene moxeparvovec in a previous clinical study. No study drug will be administered as part of this study.	Enrolling by Invitation	United States, Belgium, Germany, Hong Kong, Italy, Japan, Spain, Taiwan, United Kingdom	NCT05967351
REGENXBIO	RGX-202	Observational	Has an established clinical diagnosis of DMD based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test.	Actively Recruiting	United States	NCT05683379
Sarepta Therapeutics	SRP-9001-401 (Delandistrogene Moxeparvovec) ELEVIDYS	Observational	Has an established clinical diagnosis of DMD based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test.	Enrolling by Invitation	United States	NCT06270719

Sarepta Therapeutics

Not Yet Recruiting

Drug: SRP-9001-402 (Delandistrogene Moxeparvovec) ELEVIDYS
Phase: Phase 4
Variant Requirement: Has an established clinical diagnosis of DMD based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test.
Locations: United States
Clinical Trial: NCT07542314

Sarepta Therapeutics

Active, Not Recruiting

Drug: SRP-9001-303 (Delandistrogene Moxeparvovec) ELEVIDYS
Phase: Phase 3
Variant Requirement: Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
Locations: United States, Australia, Belgium, Canada, Germany, Hong Kong, Israel, Italy, Japan, South Korea, Spain, Sweden, Taiwan, United Kingdom
Clinical Trial: NCT05881408

Solid Biosciences

Actively Recruiting

Drug: SGT-003
Phase: Phase 3
Variant Requirement: Established clinical diagnosis of DMD and documented DMD gene mutation predictive of DMD phenotype.
Locations: United States, Canada, Australia
Clinical Trial: NCT07160634

REGENXBIO

Actively Recruiting

Drug: RGX-202
Phase: Phase 2/3
Variant Requirement: DMD gene mutation in exons 18 and above, and a clinical picture consistent with typical DMD with the exception of a participant (Cohort 1b) with DMD gene mutation in exons 12-17.
Locations: United States, Canada
Clinical Trial: NCT05693142

GENERIUM

Actively Recruiting

Drug: GNR-097
Phase: Phase 2
Variant Requirement: Ambulatory boys aged 4-9 years with a documented diagnosis of DMD and clinical manifestations of the disease.
Locations: Russia, Belarus
Clinical Trial: NCT07673809

Hoffmann-La Roche

Active, Not Recruiting

Drug: ENVOL (Delandistrogene Moxeparvovec) ELEVIDYS
Phase: Phase 2
Variant Requirement: Has a definitive diagnosis of DMD prior to screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test
Locations: Belgium, France, Germany, Italy, Spain, United Kingdom
Clinical Trial: NCT06128564

Solid Biosciences

Actively Recruiting

Drug: SGT-003
Phase: Phase 1/2
Variant Requirement: Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype confirmed by Sponsor genetic testing. In cases where a genotype may be predictive of residual dystrophin production and/or a clear clinical diagnosis of DMD cannot be made (e.g., due to age), evaluation of dystrophin levels in baseline muscle biopsies may be required to determine eligibility under this criterion.
Locations: United States, Canada, Italy, United Kingdom
Clinical Trial: NCT06138639

Solid Biosciences

Active, Not Recruiting

Drug: SGT-001
Phase: Phase 1/2
Variant Requirement: Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype.
Locations: United States
Clinical Trial: NCT03368742

Insmed Gene Therapy LLC

Actively Recruiting

Drug: INS1201
Phase: Phase 1
Variant Requirement: Has a definitive diagnosis of DMD prior to Screening or as part of Screening based on genetic testing. Note that participants who rescreen do not have to repeat genetic testing for the diagnosis of DMD if one is already on file. Genetic reports must describe a frameshift deletion, frameshift duplication, premature stop ("nonsense"), canonical splice site mutation, or other pathogenic variant in the DMD gene fully contained between exons 18 to 58 (inclusive) that is expected to lead to absence of a functional dystrophin protein (mutations in exons 1-17 or 59-71 are therefore not permitted).
Locations: United States
Clinical Trial: NCT06817382

Sarepta Therapeutics

Actively Recruiting

Drug: SRP-9001-103 (Delandistrogene Moxeparvovec) ELEVIDYS
Phase: Phase 1
Variant Requirement: For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
Locations: United States
Clinical Trial: NCT04626674

Shanghai Jiao Tong University School of Medicine

Actively Recruiting

Drug: BBM-D101
Phase: Early Phase 1
Variant Requirement: Genetically confirmed diagnosis of DMD.
Locations: China
Clinical Trial: NCT06641895

Shanghai Siponuoyin Biotechnology Co Ltd

Actively Recruiting

Drug: SPOT-mRNA03 (SPOT-03)
Phase: Early Phase 1
Variant Requirement: Boys aged ≥ 2 years to < 8 years and capable of walking independently for at least 10 meters.
Locations: China
Clinical Trial: NCT07188012

Sarepta Therapeutics

Enrolling by Invitation

Drug: SRP-9001-305 (Delandistrogene Moxeparvovec) ELEVIDYS
Phase: Interventional
Variant Requirement: The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received delandistrogene moxeparvovec in a previous clinical study. No study drug will be administered as part of this study.
Locations: United States, Belgium, Germany, Hong Kong, Italy, Japan, Spain, Taiwan, United Kingdom
Clinical Trial: NCT05967351

REGENXBIO

Actively Recruiting

Drug: RGX-202
Phase: Observational
Variant Requirement: Has an established clinical diagnosis of DMD based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test.
Locations: United States
Clinical Trial: NCT05683379

Sarepta Therapeutics

Enrolling by Invitation

Drug: SRP-9001-401 (Delandistrogene Moxeparvovec) ELEVIDYS
Phase: Observational
Variant Requirement: Has an established clinical diagnosis of DMD based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test.
Locations: United States
Clinical Trial: NCT06270719

Exon Skipping

Exon Skipping active clinical studies
Company	Drug	Phase	Variant Requirement	Status	Locations	Clinical Trial
NS Pharma	Brogidirsen NS-089/NCNP-02-201	Phase 2	Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 44 to restore the dystrophin mRNA reading frame.	Actively Recruiting	United States, Australia, Canada, Japan, New Zealand, South Korea, Turkey	NCT05996003
NS Pharma	NS-050/NCNP-03 Meteor50	Phase 1/2	Confirmed DMD exon deletion in the dystrophin gene that is amenable to skipping of exon 50 to restore the dystrophin mRNA reading frame.	Active, Not Recruiting	United States, Canada, Japan, South Korea, Turkey	NCT06053814

NS Pharma

Actively Recruiting

Drug: Brogidirsen NS-089/NCNP-02-201
Phase: Phase 2
Variant Requirement: Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 44 to restore the dystrophin mRNA reading frame.
Locations: United States, Australia, Canada, Japan, New Zealand, South Korea, Turkey
Clinical Trial: NCT05996003

NS Pharma

Active, Not Recruiting

Drug: NS-050/NCNP-03 Meteor50
Phase: Phase 1/2
Variant Requirement: Confirmed DMD exon deletion in the dystrophin gene that is amenable to skipping of exon 50 to restore the dystrophin mRNA reading frame.
Locations: United States, Canada, Japan, South Korea, Turkey
Clinical Trial: NCT06053814