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Duchenne muscular dystrophy

63 clinical trials

55 products

1 drug

Product
Viltolarsen
Clinical trial
A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)
Status: Active (not recruiting), Estimated PCD: 2025-10-01
Product
NS-050/NCNP-03
Clinical trial
A Phase 1/2, First in Human, Multiple-dose, 2-part Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NS-050/NCNP-03 in Boys With Duchenne Muscular Dystrophy (DMD)
Status: Not yet recruiting, Estimated PCD: 2027-05-01
Product
Placebo
Clinical trial
Randomised, Double-blind, Placebo-controlled, Multicentre Study to Evaluate the Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy
Status: Recruiting, Estimated PCD: 2028-02-01
Product
ATL1102
Clinical trial
A Multicentre, Randomised, Double-blind, Placebo-controlled and Open Label Extension Study to Assess the Efficacy, Safety, and Pharmacokinetic Profile of of ATL1102 in Non-ambulatory Participants With Duchenne Muscular Dystrophy
Status: Recruiting, Estimated PCD: 2024-11-13
Clinical trial
Long-term Follow-up Safety and Efficacy Study in Participants With Duchenne Muscular Dystrophy Who Have Received Fordadistrogene Movaparvovec in a Preceding Clinical Study
Status: Recruiting, Estimated PCD: 2039-05-15
Product
Fordadistrogene Movaparvovec
Product
Givinostat
Clinical trial
A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP- 9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)
Status: Recruiting, Estimated PCD: 2026-01-31
Product
placebo
Product
SRP-4045
Clinical trial
A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy
Status: Active (not recruiting), Estimated PCD: 2025-10-03
Product
Delandistrogene
Product
SRP-4053
Product
AOC 1044
Product
EDG-5506
Clinical trial
A 2-part Phase 2 Study of Safety, Pharmacokinetics and Biomarkers in Children With Duchenne Muscular Dystrophy Including a Randomized, Double-Blind, Placebo-Controlled Part A, Followed by an Open-Label Part B
Status: Recruiting, Estimated PCD: 2026-02-01
Clinical trial
A Phase 2 Open-label Study to Evaluate the Pharmacodynamics and Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants With Mutations Amenable to Exon 44 Skipping
Status: , Estimated PCD: 2025-12-31
Product
NS-089/NCNP-02
Clinical trial
A Phase 2 Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NS-089/NCNP-02 in Boys With Duchenne Muscular Dystrophy (DMD)
Status: Recruiting, Estimated PCD: 2025-11-28
Clinical trial
A Phase 2 Study to Evaluate the Effect of EDG-5506 on Safety, Pharmacokinetics, and Biomarkers in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy
Status: Recruiting, Estimated PCD: 2025-06-01
Product
SGT-003
Clinical trial
A Phase 1/2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Status: Recruiting, Estimated PCD: 2026-01-15
Clinical trial
An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of SRP-9001 in Association With Imlifidase in Subjects With Duchenne Muscular Dystrophy With Pre-existing Antibodies to rAAVrh74
Status: Active (not recruiting), Estimated PCD: 2025-01-31
Product
imlifidase
Product
Delandistrogene Moxeparvovec
Clinical trial
A Phase 1/2 Open-label, Dose Escalation and Dose Expansion Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Intravenous RGX-202 Gene Therapy in Males With Duchenne Muscular Dystrophy (DMD)
Status: Recruiting, Estimated PCD: 2025-12-01
Product
RGX-202
Product
WVE-N531
Clinical trial
An Open-label Phase 1b/2a Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
Status: Active (not recruiting), Estimated PCD: 2025-01-01
Product
Standard of Care
Clinical trial
A Long-term Multicenter Prospective Observational Study Evaluating the Comparative Effectiveness and Safety of Sarepta Gene Transfer Therapy vs. Standard of Care in Participants With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice
Status: Recruiting, Estimated PCD: 2029-12-31
Clinical trial
A Phase 3, Randomized, Double-Blind Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD)
Status: Terminated, Estimated PCD: 2023-02-13
Product
Pamrevlumab
Product
Corticosteroids
Product
Vamorolone
Product
BMN 351
Clinical trial
A Phase 1/2, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Intravenous Doses of BMN 351 in Participants With Duchenne Muscular Dystrophy
Status: Recruiting, Estimated PCD: 2025-12-01
Clinical trial
A Phase II Open-Label, Multiple Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)
Status: Active (not recruiting), Estimated PCD: 2024-07-01
Clinical trial
A Two-Part, Open-Label Systemic Gene Delivery Study to Evaluate the Safety and Expression of RO7494222 (SRP-9001) in Subjects Under the Age of Four With Duchenne Muscular Dystrophy
Status: Recruiting, Estimated PCD: 2032-11-30
Product
DS-5141b
Clinical trial
Phase I/II Study of DS-5141b: Open-label Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
Status: Completed, Estimated PCD: 2020-10-20
Product
PGN-EDO51
Clinical trial
A Phase 2, Open-Label, Multiple Ascending Dose Study of PGN-EDO51 With a Long-Term Extension in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (CONNECT1-EDO51)
Status: Recruiting, Estimated PCD: 2025-03-01
Product
PF-06939926
Clinical trial
A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF 06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
Status: Active (not recruiting), Estimated PCD: 2024-05-31
Product
EN001
Clinical trial
Long-term, Open-label Extension Study for Patients With Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen
Status: Terminated, Estimated PCD: 2023-07-26
Product
Golodirsen
Product
CAP-1002
Product
Casimersen
Clinical trial
An Exploratory, Open-label Study to Assess the Effect of P-188 NF (Carmeseal-MD) on Safety, on Respiratory and Cardiac Dysfunction and on Upper Limb Strength in Non-ambulatory Patients With Duchenne Muscular Dystrophy (DMD)
Status: Terminated, Estimated PCD: 2021-09-01
Clinical trial
A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, Then Dose Expansion, in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
Status: Active (not recruiting), Estimated PCD: 2023-10-30
Clinical trial
Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)
Status: Active (not recruiting), Estimated PCD: 2032-09-01
Product
P-188
Clinical trial
A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)
Status: Active (not recruiting), Estimated PCD: 2023-06-28
Clinical trial
A Phase II, Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
Status: Active (not recruiting), Estimated PCD: 2027-01-31
Clinical trial
An Open-Label, Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy Who Have Completed the Long-Term Extension (VBP15-LTE) or VBP15-004 or VBP15-006 Studies
Status:
Product
Vesleteplirsen
Product
Prednisone
Clinical trial
Open-label, Dose-escalation, Phase 1 Clinical Trial to Determine the Safety and Dose of EN001 in Patients With Duchenne Muscular Dystrophy(DMD)
Status: Completed, Estimated PCD: 2022-12-28
Clinical trial
A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study With Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)
Status: Completed, Estimated PCD: 2021-02-23
Product
ACE-031
Clinical trial
A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular Dystrophy
Status: Terminated, Estimated PCD: 2011-06-01
Clinical trial
Open-Label Extension of the HOPE-2 Duchenne Muscular Dystrophy Trial
Status: Active (not recruiting), Estimated PCD: 2022-02-16
Clinical trial
A Phase II, Open-Label, Extension Study to Assess the Safety and Efficacy of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
Status: Completed, Estimated PCD: 2021-10-20
Clinical trial
An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients With Duchenne Muscular Dystrophy Who Have Completed Study 4658-102
Status: Terminated, Estimated PCD: 2022-08-31
Clinical trial
A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)
Status: Completed, Estimated PCD: 2023-10-19
Product
Idebenone
Product
NS-065/NCNP-01
Product
Eteplirsen
Clinical trial
A PHASE 1B MULTICENTER, OPEN-LABEL, SINGLE ASCENDING DOSE STUDY TO EVALUATE THE SAFETY AND TOLERABILITY OF PF-06939926 IN AMBULATORY AND NON-AMBULATORY SUBJECTS WITH DUCHENNE MUSCULAR DYSTROPHY
Status: Active (not recruiting), Estimated PCD: 2022-03-28
Clinical trial
Trial of Pamrevlumab (FG-3019), a Monoclonal Antibody to Connective Tissue Growth Factor, in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy
Status: Terminated, Estimated PCD: 2020-05-07
Clinical trial
Expanded Access Protocol (EAP) of Idebenone in Patients With Duchenne Muscular Dystrophy
Status:
Clinical trial
Systemic Gene Delivery Phase I/IIa Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MHCK7.Micro-dystrophin (microDys-IV-001)
Status: Completed, Estimated PCD: 2023-04-25
Product
givinostat
Clinical trial
Randomised, Double Blind, Placebo Controlled, Multicentre Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy
Status: Completed, Estimated PCD: 2022-02-22
Clinical trial
A Randomized, Controlled, Open-label, Single-ascending Dose, Phase I/II Study to Investigate the Safety and Tolerability, and Efficacy of Intravenous SGT-001 in Male Adolescents and Children With Duchenne Muscular Dystrophy
Status: Active (not recruiting), Estimated PCD: 2026-12-01
Clinical trial
A Phase 3, Randomized, Placebo-controlled, Double-blind and Open-label, Extension Study of TAS-205 in Patients With Duchenne Muscular Dystrophy
Status: Recruiting, Estimated PCD: 2027-05-01
Product
SGT-001
Clinical trial
A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK)
Status: Active (not recruiting), Estimated PCD: 2023-10-04
Product
TAS-205
Clinical trial
Open Label, Long-term Safety, Tolerability, and Efficacy Study of GIVINOSTAT in All DMD Patients Who Have Been Previously Treated in One of the GIVINOSTAT Studies
Status: , Estimated PCD: 2025-12-01
Clinical trial
A Phase 2 Open-label Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With Duchenne Muscular Dystrophy (DMD) Compared to Natural History Controls
Status: Completed, Estimated PCD: 2023-06-20
Clinical trial
Vasodilators and Exercise as Adjuvant Therapy for Duchenne Muscular Dystrophy (VASO-REx Study)
Status: Not yet recruiting, Estimated PCD: 2026-11-01
Product
Tadalafil
Clinical trial
A Phase II Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Satralizumab in Pediatric Patients With Duchenne Muscular Dystrophy (SHIELD DMD)
Status: Not yet recruiting, Estimated PCD: 2026-06-30
Product
Satralizumab
Clinical trial
Prospective Cardiac Magnetic Resonance Imaging Study in Duchenne Muscular Dystrophy (DMD-CMP)
Status: Not yet recruiting, Estimated PCD: 2029-02-01
Product
Blood assays
Clinical trial
Phase I/II Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy
Status: Completed, Estimated PCD: 2017-11-01
Product
rAAV1.CMV.huFollistin344
Clinical trial
Phase-2 Trial of 5mg/kg/Week Prednisolone in Young Boys With DMD
Status: Recruiting, Estimated PCD: 2026-08-30
Product
Prednisolone
Clinical trial
Phase 1/2a, Monocentric, Open Label Study to Evaluate the Safety, PK and PD of SQY51 in Paediatric and Adult Patients With a Genetically Confirmed Diagnosis of Duchenne Muscular Dystrophy
Status: Recruiting, Estimated PCD: 2025-02-01
Product
SQY51
Clinical trial
Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD)
Status: Recruiting, Estimated PCD: 2025-12-01
Product
Genetic characterization
Clinical trial
Phase I/IIa Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2
Status: Active (not recruiting), Estimated PCD: 2020-11-04
Product
rAAVrh74.MCK.GALGT2
Clinical trial
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.
Status: Completed, Estimated PCD: 2021-09-01
Product
Amondys
Product
Exondys 51
Product
Vyondys 53
Clinical trial
A Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) Assessing Safety and Biomarker Changes in Boys With Duchenne Muscular Dystrophy
Status: Active (not recruiting), Estimated PCD: 2024-04-01
Product
Canakinumab
Clinical trial
Tadalafil as an Adjuvant to Therapy for Duchenne Muscular Dystrophy
Status: Recruiting, Estimated PCD: 2024-10-20
Clinical trial
A Phase 3, Multinational, Long-term Follow-up Study to Evaluate Safety and Efficacy in Subjects Who Have Previously Received SRP-9001 in a Clinical Study
Status: , Estimated PCD: 2030-11-30
Clinical trial
An Open-Label, Safety Study for Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy
Status: , Estimated PCD: 2024-04-30
Product
Ataluren
Clinical trial
Open-Label Extension of the Halt Cardiomyopathy Progression in Duchenne (HOPE-Duchenne) Trial (CAP-1002-DMD-03)
Status: Completed, Estimated PCD: 2019-03-06
Product
Allogeneic Cardiosphere-Derived Cells
Clinical trial
A Phase 1/2, Randomized, Placebo-controlled, Double-blind, Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Effects of Single and Multiple Ascending Doses of AOC 1044 Administered Intravenously to Healthy Adult Volunteers and Participants With DMD Mutations Amenable to Exon 44 Skipping
Status: Recruiting, Estimated PCD: 2025-03-01
Drug
Varlilumab
Clinical trial
Phase I/IIa Systemic Gene Delivery Clinical Trial of scAAV9.U7.ACCA for Exon 2 Duplication-Associated Duchenne Muscular Dystrophy
Status: Active (not recruiting), Estimated PCD: 2023-11-19
Product
scAAV9.U7.ACCA