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Overview of ongoing Duchenne clinical trials grouped by therapeutic approach. Completed and terminated studies are not listed.
| 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 |
| Company | Drug | Phase | Variant Requirement | Status | Locations | Clinical Trial |
|---|---|---|---|---|---|---|
| Avidity Biosciences | AOC 1044 SAFARI44 | Phase 3 | Acceptable genetic test confirming dystrophin gene mutation amenable to exon 44 skipping. | Not Yet Recruiting | United States | NCT07587242 |
| Dyne Therapeutics | DYNE-251 (Zeleciment Rostudirsen) | Phase 3 | Ambulatory male with confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping. | Actively Recruiting | United States | NCT07608432 |
| Avidity Biosciences | AOC 1044 EXPLORE44 | Phase 2 | Clinical diagnosis of DMD or clear onset of DMD symptoms at or before the age of 6 years. Confirmation of DMD gene mutation amenable to exon 44 skipping. | Active, Not Recruiting | United States | NCT06244082 |
| BioMarin Pharmaceutical | BMN 351 | Phase 2 | Participants must have completed 351-201 without permanent discontinuation of the investigational medicinal product (IMP) or withdrawal from the study. | Enrolling by Invitation | Italy, Netherlands, Spain, Turkey, United Kingdom | NCT07573631 |
| Entrada Therapeutics | ELEVATE-LTE | Phase 2 | Participant completed clinical study ENTR-601-44-201 or ENTR-601-45-201. | Not Yet Recruiting | Belgium, Italy, Netherlands, Spain, United Kingdom | NCT07682129 |
| 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 |
| Wave Life Sciences | WVE-N531 FORWARD-53 | Phase 2 | This is a Phase 2 open-label extension (OLE) study to evaluate the long-term safety, tolerability, efficacy, pharmacokinetics, and the pharmacodynamics (PD) through potential exploratory biomarker(s) of intravenous (IV) WVE-N531 in patients with DMD who participated in another study of WVE-N531. All patients will have rolled over from a previous study of WVE-N531. | Enrolling by Invitation | United Kingdom, Jordan | NCT07209332 |
| BioMarin Pharmaceutical | BMN 351 | Phase 1/2 | Diagnosis of Duchenne muscular dystrophy with a specific genetic change amenable to exon 51 skipping | Active, Not Recruiting | Italy, Netherlands, Spain, Turkey, United Kingdom | NCT06280209 |
| Dyne Therapeutics | DYNE-251 (Zeleciment Rostudirsen) | Phase 1/2 | Male with a confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping. | Active, Not Recruiting | United States, Australia, Belgium, Canada, Ireland, Italy, South Korea, Spain, United Kingdom | NCT05524883 |
| Entrada Therapeutics | ENTR-601-44 (ELEVATE-44) | Phase 1/2 | Genetic diagnosis of Duchenne muscular dystrophy (DMD) and confirmed pathologic variant in the dystrophin gene amenable to exon 44 skipping as reviewed by a central genetic counselor. | Actively Recruiting | Belgium, Italy, Spain, United Kingdom | NCT07037862 |
| Entrada Therapeutics | ENTR-601-45 (ELEVATE-45) | Phase 1/2 | Genetic diagnosis of DMD and confirmed pathologic variant in the dystrophin gene amenable to exon 45 skipping as reviewed by a central genetic counselor. | Actively Recruiting | Belgium, Italy, Netherlands, Spain, United Kingdom | NCT07038824 |
| 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 |
| Wave Life Sciences | WVE-N531 FORWARD-53 | Phase 1/2 | Documented mutation in the DMD gene associated with DMD that is amenable to exon 53 intervention. | Actively Recruiting | United States, Jordan, United Kingdom | NCT04906460 |
| Shanghai Jiao Tong University School of Medicine | LE051 | Early Phase 1 | DMD subjects with a clinical diagnosis of DMD referred to the Duchenne Clinical Practice Guidelines for Progressive Muscular Dystrophy (2020 edition) and whose genetic test results were confirmed to be applicable to exon skipping at No.51. | Actively Recruiting | China | NCT06900049 |
| Company | Drug | Phase | Variant Requirement | Status | Locations | Clinical Trial |
|---|---|---|---|---|---|---|
| Peking Union Medical College Hospital | RAG-18 | Early Phase 1 | Male patient with Duchenne Muscular Dystrophy (DMD), able to provide a written diagnosis from a specialist and a verifiable genetic test report. | Active, Not Recruiting | China | NCT07282652 |
| Company | Drug | Phase | Variant Requirement | Status | Locations | Clinical Trial |
|---|---|---|---|---|---|---|
| Precision BioSciences | PBGENE-DMD | Phase 1/2 | Molecular confirmed DMD diagnosis (DMD mutation fully contained between exons 45 to 55 [inclusive]) | Actively Recruiting | United States | NCT07429240 |
| Company | Drug | Phase | Variant Requirement | Status | Locations | Clinical Trial |
|---|---|---|---|---|---|---|
| Capricor | Deramiocel (CAP-1002) | Phase 3 | Diagnosis of DMD based on clinical and phenotypic manifestations consistent with DMD (e.g., family history of DMD, elevated creatine kinase, dystrophin muscle biopsy, calf pseudohypertrophy, history of Gowers' sign, and gait impairment before 7 years of age) as confirmed by the Investigator. | Active, Not Recruiting | United States | NCT05126758 |
| Capricor | Deramiocel (CAP-1002) | Phase 2 | Documented enrollment in the HOPE-2 trial and completion of trial follow-up through Month 12. | Active, Not Recruiting | United States | NCT04428476 |
| Edgewise Therapeutics | Sevasemten (EDG-5506) | Phase 2 | A documented mutation on the DMD gene and phenotype consistent with Duchenne muscular dystrophy. | Active, Not Recruiting | United States | NCT05540860 |
| Edgewise Therapeutics | Sevasemten (EDG-5506) | Phase 2 | Aged 6 to 17 with a documented mutation on the DMD gene and phenotype consistent with DMD. Prior receipt of an AAV-based gene therapy (≥ 2 years after documented receipt of gene therapy administration or ≥ 3 years after randomization in a randomized study). | Active, Not Recruiting | United States | NCT06100887 |
| Satellos Bioscience | SAT-3247 | Phase 2 | Previously participated in the SAT-3247-CL-101 parent clinical trials. | Enrolling by Invitation | Australia | NCT06867107 |
| Satellos Bioscience | SAT-3247 | Phase 2 | Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing with a confirmed mutation in the DMD gene. Male DMD patients who are ambulatory and aged ≥ 7 to < 10 years at the time of screening. | Actively Recruiting | United States, Australia, Belgium, Canada, Poland, Serbia, Spain, United Kingdom | NCT07287189 |
| Dystrogen Therapeutics | DT-DEC01 | Phase 1/2 | Boys of age 5 to 18 years old (at the time of screening), diagnosed with DMD confirmed by genetic testing. | Actively Recruiting | Poland | 2024-519004-27-00 |
| Myogenica | MyoPAXon | Phase 1 | Duchenne muscular dystrophy, diagnosed by mutations in the DMD (dystrophin) gene and/or absence of immunohistochemical staining for dystrophin on muscle biopsy. | Actively Recruiting | United States | NCT06692426 |
| Company | Drug | Phase | Variant Requirement | Status | Locations | Clinical Trial |
|---|---|---|---|---|---|---|
| Nationwide Children's Hospital | Prednisolone | Phase 4 | Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype. | Actively Recruiting | United States | NCT05412394 |