Duchenne muscular dystrophy

• An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants with Duchenne Muscular Dystrophy Pending
  2024-511656-41-00 4 "Duchenne Muscular Dystrophy"
• A PHASE 1/2, OPEN-LABEL, EXPLORATORY CLINICAL TRIAL TO EVALUATE THE SAFETY AND EFFICACY OF DT-DEC01 THERAPY IN PATIENTS WITH DUCHENNE MUSCUL … Pending
  2024-519004-27-00 7 "Duchenne Muscular Dystrophy"
• A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy Pending
  2025-522949-22-00 5
• A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Global Study with an Open-Label Extension to Evaluate the Efficacy and Safety of In … Pending
  2025-523087-20-00 5
• A 2-Part, Randomized, Double-Blind, Placebo-Controlled Study in Participants with Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping t … Recruiting
  2024-517499-39-00 7 2025-07-16 "Duchenne Muscular Dystrophy"
• Phase 1/2a study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of SQY51 in Paediatric and Adult Patients with a Genetically … Recruiting
  2022-500703-49-01 7 2025-11-05
• A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy Recruiting
  2024-514501-57-00 7 2025-06-20
• A 2-Part, Randomized, Double-Blind, Placebo-Controlled Study in Participants with Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping t … Recruiting
  2024-517584-23-00 9 2025-05-16 "Duchenne Muscular Dystrophy"
• A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study (EXPEDITION) Recruiting
  2023-505043-39-00 5 2024-05-24 "Duchenne Muscular Dystrophy"
• A clinical study in 3 parts with a microdystrophin (called GNT0004), a new gene therapy in boys with Duchenne disease who can still walk. Th … Recruiting
  2023-505187-11-00 7 2021-03-01 "Duchenne Muscular Dystrophy"
• Study in which all the patients take the same investigational drug with the aim of evaluate the long-term safety, tolerability, and efficacy … Recruiting
  2023-504520-26-00 5 2017-10-19 "Duchenne Muscular Dystrophy (DMD)"
• A PHASE 3, RANDOMIZED, DOUBLE-BLIND,PLACEBO-CONTROLLED STUDY OF PF-06939926 IN DMD Recruitment ended
  2023-508510-42-00 5 2025-03-24 "DUCHENNE MUSCULAR DYSTROPHY (DMD)"
• An open-label study to collect safety and effectiveness information on long-term treatment with vamorolone in boys with Duchenne Muscular Dy … Recruitment ended
  2024-512828-12-00 6 2024-12-05 "Duchenne Muscular Dystrophy"
• A study to check the effect of satralizumab in children and adolescents living with Duchenne muscular dystrophy, to check if it is safe and … Recruitment ended
  2024-512383-65-00 4 2024-09-26 "Duchenne Muscular Dystrophy (DMD)"
• A Phase 1/2, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Intrav … Recruitment ended
  2023-506737-30-00 7 2024-03-08 "Duchenne Muscular Dystrophy"
• Study of DYNE-251 in Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Recruitment ended
  2023-510351-31-00 7 2023-01-06
• A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscu … Recruitment ended
  2024-511492-15-00 5 2021-12-09 "Duchenne Muscular Dystrophy"
• A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulator … Halted
  2024-512626-28-00 5 2024-03-28 "Duchenne Muscular Dystrophy"
• Study to assess the safety and expression of RO7494222 (SRP-9001) in participants under the age of 4 with Duchenne muscular dystrophy Halted
  2023-509901-57-00 4 2023-10-26 "Duchenne Muscular Dystrophy"
• Evaluation of vamorolone CYP3A4 induction on midazolam (a sensitive CYP3A4 substrate) pharmacokinetics Ended
  2024-513845-36-00 3 2024-08-13 "Phase I study in healthy male volunteers. Intended indicatio"
• A study to learn how vamorolone affects the body, how safe it is and how it moves into, through and out of the body in healthy male particip … Ended
  2024-512101-60-00 3 2024-06-05 "Phase I study in healthy male volunteers. Intended indicatio"
• A multicentre, randomised, double-blind, placebo-controlled and open label extension study to assess the efficacy, safety, and pharmacokinet … Ended
  2024-512265-13-00 4 2023-04-13 "Duchenne Muscular Dystrophy"
• Metoprolol Safety and Efficacy in the Prevention of Cardiomyopathy in Patients with Duchenne Muscular Dystrophy (DMD) Ended
  2024-515057-19-00 5 2021-07-12 "Duchenne muscular dystrophy; Cardiomyopathy; Tachycardia"
• "A Phase 3 study of Viltolarsen in boys with Duchenne Muscular Dystrophy (DMD)" Ended
  2023-507146-91-00 5 2021-06-07 "Duchenne Muscular Dystrophy (DMD)"

Showing 1–24 of 25 trials