Duchenne Muscular Dystrophy trial readouts
registry pull 2026-08-19Primary-completion dates that listed drug and biotech companies filed with ClinicalTrials.gov for studies in Duchenne Muscular Dystrophy. They are the sponsor's own estimated windows, revised as a study runs — not announcement dates. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Upcoming readout windows
next 180 days · by month6 studies across 6 companies, in the next 180 days.
August 2026 1
| Ticker | Name | Study | Phase | Status | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|
| MSLE | Satellos Bioscience, Inc. | 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-101NCT06867107Treatment emergent adverse events | Phase 2 | Enrolling by invitation | 2026-08-30 | in 10 d | 2026-04-23 |
September 2026 1
| Ticker | Name | Study | Phase | Status | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|
| RGNX | REGENXBIO Inc. | AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)NCT05693142Part 1 Safety measured by incidence of Adverse Events and Serious Adverse Events | Phase 2/3 | Active, not recruiting | Sep 2026 | ≤ 41 d | 2026-07-21 |
October 2026 2
| Ticker | Name | Study | Phase | Status | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|
| SLDB | Solid Biosciences Inc. | Microdystrophin Gene Transfer Study in Adolescents and Children With DMDNCT03368742Number of Participants with Treatment Emergent Adverse Events (TEAEs) | Phase 1/2 | Active, not recruiting | 2026-10-15 | in 56 d | 2026-08-10 |
| SRPT | Sarepta Therapeutics, Inc. | A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)NCT03992430Part 1: Incidence of Adverse Events (AEs) | Phase 3 | Active, not recruiting | 2026-10-31 | in 72 d | 2026-02-27 |
January 2027 2
| Ticker | Name | Study | Phase | Status | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|
| 4568.T | Daiichi Sankyo Co., Ltd. | Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular DystrophyNCT04433234Incidence of Adverse Events (AEs) | Phase 2 | Active, not recruiting | 2027-01-31 | in 164 d | 2025-09-19 |
| EWTX | Edgewise Therapeutics, Inc. | A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)NCT05540860Number of adverse events during treatment with sevasemten or placebo | Phase 2 | Active, not recruiting | Jan 2027 | ≤ 164 d | 2025-11-12 |
Primary completion is the date the sponsor filed with the registry — their own projection, revised whenever they revise it, unless the row is marked actual.
Every registered study in Duchenne Muscular Dystrophy
most recently updated first96 studies match.
| Ticker | Name | Market cap | Study | Phase | Status | Interventions | Conditions | Enrollment | Start | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| MSLE | Satellos Bioscience, Inc. | $154M | Phase 2 Study of SAT-3247 in Pediatric Ambulatory PatientsNCT07287189Safety of SAT-3247 | Phase 2 | Recruiting | Drug: SAT-3247, Placebo | Duchenne Muscular Dystrophy, Duchenne, DMD, Neuromuscular Diseases, Muscular Dystrophies | 51 | 2025-12-08 | 2027-03-31 | in 223 d | 2026-08-14 |
| TRDA | Entrada Therapeutics, Inc. | $283M | A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44NCT07037862Number of participants with Treatment Emergent Adverse Events (TEAEs) according to study protocol (Part A and Open Label (OL) Period) | Phase 1/2 | Recruiting | Drug: ENTR-601-44, ENTR-601-44 - matching placebo | Duchenne Muscular Dystrophy (DMD) | 24 | 2025-06-30 | 2029-03-28 | in 951 d | 2026-08-13 |
| TRDA | Entrada Therapeutics, Inc. | $283M | A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45NCT07038824Number of participants with Treatment Emergent Adverse Events (TEAEs) according to study protocol (Part A and OL Period). | Phase 1/2 | Recruiting | Drug: ENTR-601-45, ENTR-601-45 - matching placebo | Duchenne Muscular Dystrophy (DMD) | 24 | 2025-08-30 | 2029-03-01 | in 924 d | 2026-08-13 |
| SLDB | Solid Biosciences Inc. | $974M | A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)NCT07160634Change From Baseline in Time to Rise (TTR) from Supine Velocity (rise/s) at Day 540 | Phase 3 | Recruiting | Drug: SGT-003, Placebo | Duchenne Muscular Dystrophy | 80 | 2025-10-22 | Jan 2029 | ≤ 895 d | 2026-08-13 |
| DYN | Dyne Therapeutics | $4.9B | Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 SkippingNCT05524883Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Phase 1/2 | Active, not recruiting | Drug: DYNE-251, Placebo | Duchenne Muscular Dystrophy (DMD) | 86 | 2022-08-12 | Sep 2031 | ≤ 1,867 d | 2026-08-11 |
| SLDB | Solid Biosciences Inc. | $974M | Microdystrophin Gene Transfer Study in Adolescents and Children With DMDNCT03368742Number of Participants with Treatment Emergent Adverse Events (TEAEs) | Phase 1/2 | Active, not recruiting | Genetic: SGT-001 | Duchenne Muscular Dystrophy | 12 | 2017-12-06 | 2026-10-15 | in 56 d | 2026-08-10 |
| KROS | Keros Therapeutics, Inc. | $402M | Safety and Efficacy of KER-065 in Participants With Duchenne Muscular DystrophyNCT07704099Number of participants with treatment-emergent adverse events (TEAEs and serious adverse events (SAEs) | Phase 2 | Not yet recruiting | Drug: KER-065 | Duchenne Muscular Dystrophy | 36 | 2026-09-14 | 2029-07-17 | in 1,062 d | 2026-08-10 |
| BMRN | BioMarin Pharmaceutical | $13.0B | An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular DystrophyNCT07573631To assess the long-term safety and tolerability of BMN 351 in participants with DMD | Phase 2 | Enrolling by invitation | Drug: BMN 351 | Duchenne Muscular Dystrophy (DMD) | 18 | 2026-04-20 | Dec 2031 | ≤ 1,959 d | 2026-07-24 |
| PFE | Pfizer | $155.3B | Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular DystrophyNCT04281485results2025-06-08Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52 | Phase 3 | Active, not recruiting | Genetic: PF-06939926, PF-06939926 · Other: Placebo, Placebo | Duchenne Muscular Dystrophy | 114 | 2020-11-05 | 2024-05-15actual | — | 2026-07-22 |
| RGNX | REGENXBIO Inc. | $731M | AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)NCT05693142Part 1 Safety measured by incidence of Adverse Events and Serious Adverse Events | Phase 2/3 | Active, not recruiting | Genetic: RGX-202 | Duchenne Muscular Dystrophy | 65 | 2023-01-04 | Sep 2026 | ≤ 41 d | 2026-07-21 |
| CPRX | Catalyst Pharmaceuticals, Inc. | $3.9B | Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMITNCT06564974observationalChange in Height z-score | — | Recruiting | Drug: Vamorolone | Duchenne Muscular Dystrophy | 250 | 2024-09-25 | Feb 2032 | ≤ 2,019 d | 2026-07-20 |
| SLDB | Solid Biosciences Inc. | $974M | A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)NCT06138639Incidence of treatment-emergent adverse events (AEs) | Phase 1/2 | Recruiting | Genetic: SGT-003 | Duchenne Muscular Dystrophy | 60 | 2024-05-06 | 2027-05-06 | in 259 d | 2026-07-08 |
| RGNX | REGENXBIO Inc. | $731M | AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMDNCT05683379observationalPrevalence of anti-AAV8 antibodies in patients with DMD | — | Active, not recruiting | Diagnostic test: AAV8 DetectCDx | Duchenne Muscular Dystrophy | 200 | 2022-12-20 | 2026-06-30actual | — | 2026-07-07 |
| TRDA | Entrada Therapeutics, Inc. | $283M | Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping to Evaluate the Safety and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Products (ELEVATE-LTE)NCT07682129Number of participants with Treatment Emergent Adverse Events (TEAEs) according to study protocol (Part A and OL Period) | Phase 2 | Not yet recruiting | Drug: ENTR-601-44, ENTR-601-45 | Duchenne Muscular Dystrophy (DMD) | 80 | Aug 2026 | Mar 2032 | ≤ 2,050 d | 2026-07-02 |
| BMRN | BioMarin Pharmaceutical | $13.0B | A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular DystrophyNCT06280209To evaluate and safety and tolerability of single and multiple doses of BMN 351 (incidence, severity, and dose-relationship of adverse effects and changes in laboratory parameters). | Phase 1/2 | Active, not recruiting | Drug: BMN 351 | Duchenne Muscular Dystrophy | 18 | 2024-01-03 | 2027-04-14 | in 237 d | 2026-06-25 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | 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 CohortNCT04626674Part 1 (Cohorts 1 to 5): Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12, as Measured by Western Blot | Phase 1 | Recruiting | Genetic: delandistrogene moxeparvovec | Duchenne Muscular Dystrophy | 83 | 2020-11-23 | 2027-12-31 | in 498 d | 2026-06-24 |
| DYN | Dyne Therapeutics | $4.9B | Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)NCT07608432Rise From Floor (RFF) velocity | Phase 3 | Recruiting | Drug: Zeleciment Rostudirsen (DYNE-251), Placebo | Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne, Muscular Dystrophy (DMD), DMD, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Duchenne Type, Muscular Dystrophy, Duchenne and Becker Types, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Genetic Disease, Inborn, Genetic Disease, X-Linked, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neuromuscular Diseases (NMD) | 90 | Jun 2026 | Dec 2030 | ≤ 1,594 d | 2026-05-27 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing SettingNCT07542314Cohort 1: Number of Participants with ALI | Phase 4 | Not yet recruiting | Drug: ELEVIDYS, Sirolimus, Glucocorticoids, Antibiotics | Duchenne Muscular Dystrophy | 20 | 2026-07-31 | 2027-03-31 | in 223 d | 2026-05-26 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | 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)NCT05881408Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72 | Phase 3 | Active, not recruiting | Genetic: delandistrogene moxeparvovec, placebo | Duchenne Muscular Dystrophy | 148 | 2023-05-31 | 2027-05-31 | in 284 d | 2026-05-22 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular DystrophyNCT06270719observationalMean Change From Baseline in Time to Walk/Run 10 Meters (10MWR) (Calculated Velocity) at Month 12 | — | Enrolling by invitation | Genetic: Delandistrogene Moxeparvovec · Drug: Standard of Care | Duchenne Muscular Dystrophy | 500 | 2024-02-07 | 2029-12-31 | in 1,229 d | 2026-05-04 |
| MSLE | Satellos Bioscience, Inc. | $154M | 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-101NCT06867107Treatment emergent adverse events | Phase 2 | Enrolling by invitation | Drug: SAT-3247 | Duchenne Muscular Dystrophy (DMD) | 10 | 2025-08-20 | 2026-08-30 | in 10 d | 2026-04-23 |
| RGNX | REGENXBIO Inc. | $731M | Long Term Follow-up for RGX-202NCT06491927observationalEvaluation of long-term safety of RGX-202 | — | Enrolling by invitation | Genetic: No Intervention | Duchenne Muscular Dystrophy | 66 | 2024-05-08 | 2029-12-01 | in 1,199 d | 2026-03-24 |
| PTCT | PTC Therapeutics | $6.1B | Long-Term Outcomes of Ataluren in Duchenne Muscular DystrophyNCT03179631results2026-03-10DB Period: Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 72 - Modified Intention-to-treat (mITT) Population | Phase 3 | Completed | Drug: Ataluren, PLACEBO | Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn | 360 | 2017-07-06 | 2022-03-05actual | — | 2026-03-10 |
| PTCT | PTC Therapeutics | $6.1B | Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)NCT01247207Number of Participants With Adverse Events | Phase 3 | Completed | Drug: Ataluren | Duchenne Muscular Dystrophy | 270 | 2010-11-30 | 2026-02-10actual | — | 2026-03-09 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)NCT03992430Part 1: Incidence of Adverse Events (AEs) | Phase 3 | Active, not recruiting | Drug: Eteplirsen | Muscular Dystrophy, Duchenne | 160 | 2020-07-13 | 2026-10-31 | in 72 d | 2026-02-27 |
| PTCT | PTC Therapeutics | $6.1B | Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)NCT02369731observationalPercentage of Participants With Adverse Events | — | Completed | — | Muscular Dystrophy, Duchenne | 316 | 2015-04-30 | 2025-03-31actual | — | 2026-02-24 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical StudyNCT05967351Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI) | Phase 3 | Enrolling by invitation | Genetic: delandistrogene moxeparvovec | Duchenne Muscular Dystrophy | 400 | 2023-09-27 | 2033-10-31 | in 2,629 d | 2026-02-06 |
| PEPG | PepGen Inc | $202M | A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping TreatmentNCT06079736Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 in MAD period) | Phase 2 | Terminatedsponsor's stated reason: PGN-EDO51 development terminated by Sponsor | Drug: PGN-EDO51 | Duchenne Muscular Dystrophy | 7 | 2024-01-03 | 2025-08-28actual | — | 2025-12-02 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | 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)NCT06241950Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content | Phase 1 | Terminatedsponsor's stated reason: Study is being terminated due to a business decision. | Genetic: delandistrogene moxeparvovec · Biological: imlifidase | Duchenne Muscular Dystrophy | 5 | 2024-01-29 | 2025-10-10actual | — | 2025-11-20 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)NCT02500381Change From Baseline in the 4-Step Ascend Velocity at Week 96 | Phase 3 | Completed | Drug: SRP-4045, SRP-4053, Placebo | Duchenne Muscular Dystrophy | 228 | 2016-09-28 | 2024-11-12actual | — | 2025-11-18 |
| EWTX | Edgewise Therapeutics, Inc. | $4.8B | A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)NCT05540860Number of adverse events during treatment with sevasemten or placebo | Phase 2 | Active, not recruiting | Drug: Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3, Sevasemten Dose 4, Sevasemten Dose 5, Placebo | Duchenne Muscular Dystrophy | 76 | 2022-10-24 | Jan 2027 | ≤ 164 d | 2025-11-12 |
| EWTX | Edgewise Therapeutics, Inc. | $4.8B | Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene TherapyNCT06100887Number of adverse events during treatment with sevasemten or placebo | Phase 2 | Active, not recruiting | Drug: Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3, Placebo | Duchenne Muscular Dystrophy | 43 | 2024-03-22 | Mar 2027 | ≤ 223 d | 2025-11-06 |
| PFE | Pfizer | $155.3B | Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular DystrophyNCT05429372Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events | Phase 2 | Terminatedsponsor's stated reason: All participants who have received fordadistrogene movaparvovec in any Pfizer study will now be assessed for long-term safety in one combined study: C3391003 | Genetic: PF-06939926 | Muscular Dystrophy, Duchenne | 10 | 2022-08-08 | 2025-10-03actual | — | 2025-10-21 |
| PFE | Pfizer | $155.3B | A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.NCT05689164Number of participants with serious adverse events | Phase 3 | Terminatedsponsor's stated reason: All participants who have received fordadistrogene movaparvovec in any Pfizer study will now be assessed for long-term safety in 1 combined study: C3391003 | Biological: fordadistrogene movaparvovec | Duchenne Muscular Dystrophy | 7 | 2023-03-13 | 2025-09-24actual | — | 2025-10-21 |
| 4568.T | Daiichi Sankyo Co., Ltd. | $31.7B | Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular DystrophyNCT04433234Incidence of Adverse Events (AEs) | Phase 2 | Active, not recruiting | Drug: DS-5141b | Duchenne Muscular Dystrophy | 8 | 2020-06-30 | 2027-01-31 | in 164 d | 2025-09-19 |
| PFE | Pfizer | $155.3B | A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular DystrophyNCT03362502results2024-09-20Number of Participants With All-Causality Treatment-Emergent Adverse Events (TEAEs) for 1-Year Follow-Up | Phase 1 | Terminatedsponsor's stated reason: All participants who have received fordadistrogene movaparvovec in any Pfizer study will now be assessed for long-term safety in 1 combined study: C3391003 | Genetic: PF-06939926 | Duchenne Muscular Dystrophy | 23 | 2018-01-23 | 2022-03-28actual | — | 2025-09-15 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical PracticeNCT06606340observationalLoss of Ambulation (LOA) | — | Enrolling by invitation | Drug: Eteplirsen, Golodirsen, Casimersen | Duchenne Muscular Dystrophy | 300 | 2019-01-07 | 2033-12-31 | in 2,690 d | 2025-09-10 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | 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 AAVrh74NCT06597656Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression Adjusted by Muscle Content Biopsied Muscle as Measured by Western Blot | Phase 1 | Terminatedsponsor's stated reason: Study is being terminated due to a business decision. | Genetic: delandistrogene moxeparvovec · Procedure: Plasmapheresis | Duchenne Muscular Dystrophy | 3 | 2024-09-18 | 2025-08-05actual | — | 2025-09-04 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)NCT05096221results2024-12-10Part 1: Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52 | Phase 3 | Completed | Genetic: delandistrogene moxeparvovec, placebo | Duchenne Muscular Dystrophy | 126 | 2021-10-27 | 2023-10-04actual | — | 2025-07-08 |
| PEPG | PepGen Inc | $202M | A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping TreatmentNCT06833931Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 during the MAD period) | Phase 2 | Withdrawnsponsor's stated reason: Global PGN-EDO51 development voluntarily discontinued by Sponsor | Drug: IV infusion · Other: Placebo | Duchenne Muscular Dystrophy (DMD) | 0 | 2024-12-17 | 2025-05-28actual | — | 2025-06-24 |
| MSLE | Satellos Bioscience, Inc. | $154M | First in Human SAD/MAD Safety and PK Study With Adult DMD Safety and PK CohortNCT06565208Incidence and severity of treatment emergent adverse events | Early Phase 1 | Completed | Drug: SAT-3247, matched placebo | Duchenne Muscular Dystrophy | 77 | 2024-08-21 | 2025-04-28actual | — | 2025-05-15 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | 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 TreatmentNCT04004065Part A: Incidence of Adverse Events (AEs) | Phase 2 | Terminatedsponsor's stated reason: The study was terminated following review of safety data. | Drug: Vesleteplirsen | Duchenne Muscular Dystrophy | 62 | 2019-06-26 | 2023-10-30actual | — | 2025-03-10 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)NCT03375164results2024-05-23Number of Participants With Adverse Events (AEs) | Phase 1/2 | Completed | Genetic: delandistrogene moxeparvovec | Duchenne Muscular Dystrophy | 4 | 2018-01-04 | 2023-04-25actual | — | 2024-11-14 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)NCT03769116results2023-09-15Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content | Phase 1/2 | Completed | Genetic: delandistrogene moxeparvovec, placebo | Muscular Dystrophy, Duchenne | 41 | 2018-12-05 | 2020-12-08actual | — | 2024-11-14 |
| 4503.T | Astellas Pharma Inc | $26.0B | A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD)NCT04184882Number of participants with Treatment Emergent Adverse Events (TEAEs) | Phase 1 | Terminatedsponsor's stated reason: Operational Reasons | Drug: Bocidelpar, Placebo | Duchenne Muscular Dystrophy (DMD) | 8 | 2021-02-24 | 2022-09-04actual | — | 2024-10-22 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular DystrophyNCT03532542results2024-09-19Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs) | Phase 3 | Terminatedsponsor's stated reason: The trial was stopped to reduce the clinical trial burden on participants while ensuring continued treatment via a post-trial access program with commercially available casimersen and golodirsen. Study was not terminated due to safety concerns. | Drug: Casimersen, Golodirsen | Duchenne Muscular Dystrophy | 171 | 2018-08-02 | 2023-07-26actual | — | 2024-09-19 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen)NCT03675126results2024-09-19Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs) | Phase 1/2 | Terminatedsponsor's stated reason: The sponsor has decided to integrate 5051-102 into 5051-201. Participants from 5051-102 will be eligible to enroll in 5051-201 Part B. | Drug: SRP-5051 | Muscular Dystrophy, Duchenne | 15 | 2018-12-19 | 2021-08-25actual | — | 2024-09-19 |
| PFE | Pfizer | $155.3B | A Natural History Study In Chinese Male Patients With Duchenne Muscular DystrophyNCT03760029results2024-09-19Age of Participants When They Failed to Walk | Not applicable | Completed | Other: Visit frequency | Duchenne Muscular Dystrophy | 312 | 2019-07-24 | 2023-03-21actual | — | 2024-09-19 |
| KYNB | Kyntra Bio | $28M | Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)NCT02606136results2021-12-30Annual Change From Baseline in Percent Predicted Forced Vital Capacity (ppFVC) at Week 104 | Phase 2 | Terminatedsponsor's stated reason: Sponsor Decision | Drug: Pamrevlumab | Duchenne Muscular Dystrophy | 21 | 2016-01-04 | 2020-05-07actual | — | 2024-08-27 |
| KYNB | Kyntra Bio | $28M | Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMDNCT04632940results2024-08-26Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52 | Phase 3 | Terminatedsponsor's stated reason: Study did not meet its primary endpoint. | Drug: Pamrevlumab, Placebo, Corticosteroids | Duchenne Muscular Dystrophy | 73 | 2021-03-03 | 2023-06-12actual | — | 2024-08-26 |
| KYNB | Kyntra Bio | $28M | Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD)NCT04371666results2024-03-12Change From Baseline in the Total Score of Performance of Upper Limb (PUL) 2.0 Version at Week 52 | Phase 3 | Terminatedsponsor's stated reason: Study did not meet its primary endpoint. | Drug: Pamrevlumab, Placebo, Corticosteroids | Duchenne Muscular Dystrophy | 98 | 2020-08-10 | 2023-02-13actual | — | 2024-03-12 |
| 4568.T | Daiichi Sankyo Co., Ltd. | $31.7B | Study of DS-5141b in Patients With Duchenne Muscular DystrophyNCT02667483results2024-03-07Number of Participants Reporting at Least One Treatment-emergent Adverse Event (TEAE) In Participants With Duchenne Muscular Dystrophy | Phase 1/2 | Completed | Drug: DS-5141b | Duchenne Muscular Dystrophy | 8 | Oct 2015 | 2020-10-20actual | — | 2024-03-07 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)NCT03985878results2023-08-18Number of Participants Experiencing Adverse Events (AEs) | Phase 2 | Terminatedsponsor's stated reason: Participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment. There are no safety concerns with Eteplirsen. | Drug: Eteplirsen | Duchenne Muscular Dystrophy | 15 | 2019-06-26 | 2022-08-31actual | — | 2023-08-18 |
| SLDB | Solid Biosciences Inc. | $974M | Evaluation of Home Based Assessments on Participants With DMDNCT05657938observationalWearable sensor device compliance | — | Completed | Device: Wearable Device · Other: Duchenne Video Assessment (DVA) | Duchenne Muscular Dystrophy | 17 | 2022-10-13 | 2023-04-04actual | — | 2023-08-14 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)NCT03375255Number of Participants with Adverse Events (AEs) | Phase 1 | Completed | Drug: SRP-5051 | Muscular Dystrophy, Duchenne | 15 | 2018-02-05 | 2019-08-19actual | — | 2022-07-06 |
| PTCT | PTC Therapeutics | $6.1B | A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)NCT03648827results2022-04-05Percent Change From Baseline in Dystrophin Level at Week 40, as Measured by ECL | Phase 2 | Completed | Drug: Ataluren | Duchenne Muscular Dystrophy | 20 | 2018-12-21 | 2020-10-23actual | — | 2022-04-05 |
| PTCT | PTC Therapeutics | $6.1B | A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With AtalurenNCT03796637results2022-04-05Mean Dystrophin Levels as Measured by Electrochemiluminescence (ECL) | Phase 2 | Completed | Drug: Ataluren | Duchenne Muscular Dystrophy | 6 | 2019-04-11 | 2019-06-03actual | — | 2022-04-05 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 SkippingNCT03218995results2021-12-09Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and TEAEs Leading to Discontinuation From Study Drug | Phase 2 | Completed | Drug: Eteplirsen | Duchenne Muscular Dystrophy | 15 | 2017-08-16 | 2021-03-10actual | — | 2021-12-09 |
| PFE | Pfizer | $155.3B | A Low Interventional Study to Monitor Activity Using Wearable Sensors in Duchenne Muscular DystrophyNCT04254172observationalMean change from baseline and variability of activity measures | — | Terminatedsponsor's stated reason: This low-interventional study (involving no study drug) has been terminated early due to the COVID-19 pandemic, and not for any safety or efficacy reasons. | Device: Activity Monitor | Duchenne Muscular Dystrophy (DMD) | 2 | 2020-02-19 | 2020-08-19actual | — | 2021-10-15 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) PatientsNCT02530905results2021-05-17Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Phase 1 | Completed | Drug: SRP-4045, Placebo | Duchenne Muscular Dystrophy | 12 | 2015-10-08 | 2018-10-03actual | — | 2021-05-17 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | Study of Eteplirsen in DMD PatientsNCT02255552results2020-07-01Change From Baseline in the 6 Minute Walk Test (6MWT) Distance at Week 96 | Phase 3 | Completed | Drug: eteplirsen | Duchenne Muscular Dystrophy (DMD) | 109 | 2014-11-17 | 2019-06-14actual | — | 2021-01-25 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular DystrophyNCT02420379results2020-07-22Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Phase 2 | Completed | Drug: eteplirsen | Duchenne Muscular Dystrophy (DMD) | 33 | 2015-06-30 | 2018-12-17actual | — | 2021-01-25 |
| PFE | Pfizer | $155.3B | A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular DystrophyNCT02310763results2019-07-23Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Week 49 | Phase 2 | Terminatedsponsor's stated reason: Termination Date 30AUG2018: Reason for termination: Lack of Efficacy | Biological: PF-06252616 · Drug: Placebo | Duchenne Muscular Dystrophy | 121 | 2014-11-24 | 2018-04-30actual | — | 2020-12-07 |
| PTCT | PTC Therapeutics | $6.1B | Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and CanadaNCT01557400results2020-11-25Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Phase 3 | Completed | Drug: Ataluren | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Dystrophinopathy | 94 | 2012-05-20 | 2018-01-19actual | — | 2020-11-25 |
| PFE | Pfizer | $155.3B | An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular DystrophyNCT02907619results2019-11-25Number of Participants With Dose Reduced or Temporary Discontinuation Due to AEs | Phase 2 | Terminatedsponsor's stated reason: Termination Date: 30Aug2018; Reason for termination: Lack of Efficacy | Biological: PF-06252616 | Duchenne Muscular Dystrophy | 59 | 2016-10-13 | 2018-11-22actual | — | 2020-11-23 |
| PTCT | PTC Therapeutics | $6.1B | Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)NCT00759876results2020-10-29Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Phase 2 | Terminatedsponsor's stated reason: Terminated early because similar study with Ataluren (PTC124-GD-007-DMD; NCT00592553) exhibited lack of efficacy at the high dose (not due to safety concerns). | Drug: Ataluren | Duchenne Muscular Dystrophy | 36 | 2008-08-13 | 2010-05-17actual | — | 2020-10-29 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | Phase I/II Study of SRP-4053 in DMD PatientsNCT02310906results2020-10-19Part 1: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and TEAEs Leading to Discontinuation | Phase 1/2 | Completed | Drug: Placebo, SRP-4053 | Duchenne Muscular Dystrophy | 39 | 2015-01-13 | 2019-03-25actual | — | 2020-10-19 |
| PCSA | Processa Pharmaceuticals | $6M | Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular DystrophyNCT01847573Safety and tolerability of administering single and multiple ascending doses of HT-100 in DMD boys | Phase 1/2 | Terminatedsponsor's stated reason: Dosing stopped | Drug: HT-100 | Duchenne Muscular Dystrophy | 17 | May 2013 | 2016-03-30actual | — | 2020-09-03 |
| PCSA | Processa Pharmaceuticals | $6M | Open Label Extension Study of HT-100 in Patients With DMDNCT01978366Safety and tolerability of administration of 6 months of chronic, oral, multiple doses of HT-100 to boys with DMD. | Phase 2 | Terminatedsponsor's stated reason: Dosing stopped | Drug: HT-100 | Duchenne Muscular Dystrophy | 17 | Oct 2013 | 2016-04-30actual | — | 2020-09-03 |
| PTCT | PTC Therapeutics | $6.1B | Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular DystrophyNCT02819557results2020-08-28Number of Participants With Treatment Emergent Adverse Events (TEAEs), TEAEs Leading to Discontinuation, and Serious Adverse Events (SAEs) | Phase 2 | Completed | Drug: Ataluren | Duchenne Muscular Dystrophy | 14 | 2016-06-09 | 2018-02-09actual | — | 2020-08-28 |
| PTCT | PTC Therapeutics | $6.1B | An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation DystrophinopathyNCT02090959results2020-08-11Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Phase 3 | Terminatedsponsor's stated reason: Sponsor decision due to commercial availability of ataluren. | Drug: Ataluren | Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn | 219 | 2014-03-20 | 2018-06-12actual | — | 2020-08-11 |
| PTCT | PTC Therapeutics | $6.1B | Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)NCT01826487results2020-08-04Change From Baseline in 6MWD at Week 48 | Phase 3 | Completed | Drug: Ataluren, Placebo | Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn | 230 | 2013-03-26 | 2015-08-20actual | — | 2020-08-04 |
| PTCT | PTC Therapeutics | $6.1B | Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)NCT01009294results2020-07-29Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Phase 2 | Terminatedsponsor's stated reason: Terminated early because a similar study with Ataluren exhibited lack of efficacy at the high dose (not due to safety concerns). | Drug: Ataluren, Chronic Corticosteroid Therapy | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy | 6 | 2010-01-13 | 2010-03-23actual | — | 2020-07-29 |
| PTCT | PTC Therapeutics | $6.1B | Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)NCT00847379results2020-07-15Number of Participants With Treatment-Emergent Adverse Events (AEs) | Phase 2 | Terminated | Drug: Ataluren | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy | 173 | 2009-01-31 | 2010-05-24actual | — | 2020-07-15 |
| RMD | ResMed | $31.7B | European Home Mechanical Ventilation RegistryNCT02315339observationalDetermine the mortality rate and the number of hospital readmissions | — | Terminated | — | Pulmonary Disease, Chronic Obstructive, Amyotrophic Lateral Sclerosis, Spinal Cord Injury, Muscular Dystrophies, Obesity Hypoventilation Syndrome, Kyphoscoliosis, Congenital Central Hypoventilation Syndrome, Duchenne Muscular Dystrophy, Myopathies, Myotonic Dystrophy | 37 | Aug 2014 | Jan 2019actual | — | 2020-04-09 |
| PTCT | PTC Therapeutics | $6.1B | Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)NCT00592553results2020-04-07Change From Baseline in 6MWD at Week 48 | Phase 2 | Completed | Drug: Ataluren, Placebo | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy | 174 | 2008-02-29 | 2009-12-31actual | — | 2020-04-07 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy PatientsNCT01396239results2015-11-09Change in the Number (%) of Dystrophin Positive Fibers | Phase 2 | Completed | Drug: AVI-4658 (Eteplirsen) · Other: Placebo | Duchenne Muscular Dystrophy | 12 | Jul 2011 | Feb 2012actual | — | 2020-03-30 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular DystrophyNCT01540409results2019-07-10Change From Baseline in the 6 Minute Walk Test (6MWT) at Week 240 | Phase 2 | Completed | Drug: AVI-4658 (Eteplirsen) | Duchenne Muscular Dystrophy (DMD) | 12 | 2012-02-27 | 2016-04-15actual | — | 2020-03-30 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular DystrophyNCT02286947results2019-02-20Number of Participants With Treatment Emergent Adverse Events | Phase 2 | Completed | Drug: Eteplirsen | Muscular Dystrophy, Duchenne | 24 | Nov 2014 | 2017-04-21actual | — | 2020-03-30 |
| SMMT | Summit Therapeutics | $10.2B | Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrophy (DMD)NCT02858362results2020-01-02Change From Baseline in Magnetic Resonance Spectroscopy (MRS) Fat Fraction (FF) for Leg Muscles | Phase 2 | Terminatedsponsor's stated reason: The study was terminated due to lack of efficacy in Cohorts 1 and 2. | Drug: Ezutromid | Duchenne Muscular Dystrophy | 43 | Jun 2016 | 2018-04-11actual | — | 2020-01-02 |
| LLY | Eli Lilly and Company | $1.15T | A Study of Tadalafil for Duchenne Muscular DystrophyNCT01865084results2017-03-01Change From Baseline in Six Minute Walk Distance (6MWD) in Meters | Phase 3 | Terminatedsponsor's stated reason: The study is being terminated for lack of efficacy | Drug: Tadalafil, Placebo | Muscular Dystrophy, Duchenne | 331 | Sep 2013 | Dec 2015actual | — | 2019-10-09 |
| PTCT | PTC Therapeutics | $6.1B | A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)NCT03642145Period 1 and 2: Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Phase 3 | Withdrawnsponsor's stated reason: Study is no longer necessary given the safety and efficacy of emflaza in this age range has already been established after review of already available data. | Drug: Deflazacort | Duchenne Muscular Dystrophy | 0 | 2018-10-31 | 2021-07-31 | — | 2019-06-21 |
| BMRN | BioMarin Pharmaceutical | $13.0B | Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)NCT01037309results2015-04-09Increase in Dystrophin Expression in the Muscle Biopsies by Immunofluorescence Analyses of Cross-sections and by Western Blot Analyses of Total Protein Extracts | Phase 1/2 | Completed | Drug: PRO044 SC, PRO044 IV | Duchenne Muscular Dystrophy | 18 | Dec 2009 | May 2013actual | — | 2018-10-16 |
| PTCT | PTC Therapeutics | $6.1B | Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular DystrophyNCT02592941expanded access | — | Approved for marketing | Drug: Deflazacort | Duchenne Muscular Dystrophy | — | — | — | — | 2018-02-28 |
| BMRN | BioMarin Pharmaceutical | $13.0B | Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)NCT02958202Number of subjects with 1 or more treatment emergent adverse events following BMN044 dosing | Phase 2 | Terminatedsponsor's stated reason: Regulatory approval was not obtained for drisapersen, hence BioMarin is stopping the development of all exon skipping oligonucleotides in DMD. | Drug: BMN 044 IV 6 mg/kg, BMN 044 IV 9 mg/kg, BMN 044 SC 6 mg/kg | Duchenne Muscular Dystrophy | 7 | Apr 2016 | Sep 2016actual | — | 2018-01-26 |
| BMRN | BioMarin Pharmaceutical | $13.0B | Extension Study of Drisapersen in DMD SubjectsNCT02636686expanded access | — | No longer available | Drug: Drisapersen | Duchenne Muscular Dystrophy | — | — | — | — | 2018-01-24 |
| BMRN | BioMarin Pharmaceutical | $13.0B | A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.NCT01753804observational6 minute walk distance | — | Terminated | Other: Observational study | Duchenne Muscular Dystrophy | 269 | 2012-09-01 | 2016-10-01actual | — | 2017-12-08 |
| BMRN | BioMarin Pharmaceutical | $13.0B | Phase IIb Study of PRO045 in Subjects With Duchenne Muscular DystrophyNCT01826474Change from baseline in 6 minute walk test | Phase 1/2 | Terminated | Drug: PRO045, 0.15 mg/kg/week, PRO045, 1.0 mg/kg/week, PRO045, 3.0 mg/kg/week, PRO045, 6.0 mg/kg/week, PRO045, 9.0 mg/kg/week, PRO045, selected dose | Duchenne Muscular Dystrophy | 15 | Jan 2013 | 2016-08-31actual | — | 2017-12-08 |
| BMRN | BioMarin Pharmaceutical | $13.0B | A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)NCT01957059Change from baseline in 6 minute walk test | Phase 1/2 | Terminated | Drug: Regimen Selection Phase Group 2, Regimen Selection Phase Group 3, Treatment Phase Group 4, Regimen Selection Phase Group 1 (COMPLETED), Dosing Extension | Duchenne Muscular Dystrophy | 9 | Jun 2013 | 2016-08-03actual | — | 2017-12-08 |
| PTCT | PTC Therapeutics | $6.1B | An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability DeflazacortNCT02295748Number, frequency, and severity of adverse events | Phase 1 | Completed | Drug: Deflazacort | Duchenne Muscular Dystrophy | 24 | Dec 2014 | 2017-08-24actual | — | 2017-12-08 |
| BMRN | BioMarin Pharmaceutical | $13.0B | Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)NCT02329769Efficacy of PRO044 (composite of several measures) | Phase 2 | Terminated | Drug: PRO044 SC 6 mg/kg, PRO044 IV 6 mg/kg, PRO044 IV 9 mg/kg | Duchenne Muscular Dystrophy | 15 | Dec 2014 | 2016-07-01actual | — | 2017-12-08 |
| PTCT | PTC Therapeutics | $6.1B | A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular DystrophyNCT02251600The area under the plasma concentration time curve, from time 0 to the last measurable concentration non-zero, for single-state pharmacokinetics on Day 1 of deflazacort and 21-desacetyl-DFZ, the active metabolite | Phase 1 | Completed | Drug: Deflazacort | Duchenne Muscular Dystrophy | 24 | Dec 2014 | Oct 2015actual | — | 2017-08-18 |
| SRPT | Sarepta Therapeutics, Inc. | $1.9B | Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) PatientsNCT00844597results2015-10-06Safety and Tolerability | Phase 1/2 | Completed | Drug: AVI-4658 for Injection | Duchenne Muscular Dystrophy | 19 | Jan 2009 | Jun 2010actual | — | 2015-10-06 |
| SMMT | Summit Therapeutics | $10.2B | Modified Diet Trial: A Study of SMT C1100 in Paediatric Patients With DMD Who Follow a Balanced DietNCT02383511Pharmacokinetic parameters at different dose levels of SMT C1100 | Phase 1 | Completed | Drug: SMT C1100, SMT C1100, SMT C1100 | Muscular Dystrophy, Duchenne | 12 | Feb 2015 | Aug 2015actual | — | 2015-08-26 |
| SMMT | Summit Therapeutics | $10.2B | A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD)NCT02056808Safety and tolerability | Phase 1 | Completed | Drug: SMT C1100 | Duchenne Muscular Dystrophy | 12 | Nov 2013 | May 2014actual | — | 2014-08-27 |
| PTCT | PTC Therapeutics | $6.1B | Safety and Efficacy Study of PTC124 in Duchenne Muscular DystrophyNCT00264888Dystrophin expression as assessed by immunofluorescence evaluation of tissue obtained by biopsy of the extensor digitorum brevis (EDB) muscle of the foot or tibialis anterior (TA) muscle of the leg | Phase 2 | Completed | Drug: PTC124 | Duchenne Muscular Dystrophy | 38 | Dec 2005 | May 2007actual | — | 2009-01-14 |
Primary completion is the date the sponsor filed with the registry — their own projection, revised whenever they revise it, unless the row is marked actual.
How Duchenne Muscular Dystrophy is matched — 4 exact registry spellings
A study is on this page when its ClinicalTrials.gov record names one of these conditions exactly (letter case aside). Nothing is matched by substring or stem, so a longer condition that merely contains one of these words is a different page. The list was curated by hand and last measured 2026-08-19; a spelling filed by only one study was not curated, so the counts here are a floor rather than the registry's own total for the disease.
- Duchenne Muscular Dystrophy
- Duchenne Muscular Dystrophy (DMD)
- Muscular Dystrophy, Duchenne
- DMD
Permalink · All conditions · Catalyst calendar · Every registered study · Search the whole pipeline for “Duchenne Muscular Dystrophy” (a text search, wider than this page)