Duchenne Muscular Dystrophy research hub
23 indexed studies · 11 currently recruiting
- active not recruitingPHASE4
A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy
This study looks at the long-term safety and effects of a medicine called vamorolone for boys with Duchenne Muscular Dystrophy. It includes boys who have already used vamorolone in other studies or special access programmes.
Belgium · Czechia · Greece - active not recruitingNA
Hydrotherapy in Duchenne Muscular Dystrophy (DMD)
This study looks at whether warm water exercise (hydrotherapy) helps young people with Duchenne muscular dystrophy (DMD). We want to see if it improves their daily life, reduces pain, and helps them be more active. We'll also ask families about their experiences.
United Kingdom - recruitingPHASE1, PHASE2
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
This study is looking for boys aged 4-10 with Duchenne muscular dystrophy (DMD) with a specific genetic change. It's testing a new medicine called BMN 351 to see how safe it is and how well the body handles it. They want to find the right dose for future studies.
Canada · Italy · Netherlands - not yet recruiting
The U.K. NorthStar Clinical Network
This UK study tracks Duchenne Muscular Dystrophy (DMD) in children and adults. It records how the condition changes over time, looking at movement, breathing, and general health. The aim is to better understand DMD to help improve future treatments.
United Kingdom - active not recruitingPHASE3
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)
This study is testing a new gene therapy called delandistrogene moxeparvovec for boys with Duchenne Muscular Dystrophy (DMD). It aims to see if it's safe and effective for those who can and cannot walk. Some boys will receive the treatment, others a dummy treatment.
United States · Australia · Belgium - recruitingPHASE3
Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy
This study is testing a new medicine, Givinostat, for boys aged 9-17 with Duchenne muscular dystrophy who use a wheelchair. Researchers want to see if it helps reduce muscle decline and is safe to use. Half the boys will get the medicine, and half a dummy pill for 18 months.
Belgium · Canada · France - ongoing, recruitingTherapeutic confirmatory (Phase III)
The efficacy and safety of Metoprolol as add-on treatment to standard of care in preventing cardiomyopathy in patients with Duchenne Muscular Dystrophy aged 8-17 years. A randomized, double-blind, placebo controlled study
This study looks at whether a medicine called Metoprolol can help prevent heart problems in young people with Duchenne Muscular Dystrophy. It compares Metoprolol to a dummy medicine to see if it makes a difference to heart strength.
Poland - active not recruitingPHASE3
Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
This study is testing a new gene therapy, PF-06939926, for boys aged 4-7 with Duchenne muscular dystrophy. It aims to see if the treatment is safe and effective in improving muscle function. Some boys will get the treatment right away, others will get a dummy treatment first, then the real treatment later.
United States · Australia · Belgium - recruitingPHASE2, PHASE3
Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
This study looks at the long-term safety and effects of a medicine called Givinostat for boys with Duchenne muscular dystrophy. It's for those who have already taken Givinostat in a previous study to see how it helps over time.
United States · Belgium · Canada - recruitingPHASE1, PHASE2
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
This study is looking at a new medicine called WVE-N531 for Duchenne muscular dystrophy. It wants to see if the medicine is safe, how the body uses it, and if it helps patients. It focuses on boys with a specific genetic change.
United States · Jordan · United Kingdom - enrolling by invitationPHASE2
Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
This study is looking at the long-term safety and effects of a medicine called WVE-N531 for Duchenne muscular dystrophy. It's for boys who have already taken part in an earlier study of WVE-N531. They will continue to receive the treatment and be carefully checked.
Jordan · United Kingdom - recruitingPHASE2
Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old
This study is looking at a new medicine called Givinostat for boys with Duchenne Muscular Dystrophy (DMD) between 2 and 6 years old. It aims to see how the medicine works in their bodies and if it is safe for them.
Belgium · Italy · Netherlands - recruitingPHASE2
Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients
This study is testing a new medicine, SAT-3247, for boys aged 7-9 with Duchenne muscular dystrophy. We want to find the best dose, see if it's safe, and check if it helps. Some boys will get the real medicine, others a dummy medicine.
United States · Australia · Belgium - enrolling by invitationPHASE3
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
This study is checking how people who previously received a treatment called delandistrogene moxeparvovec for Duchenne muscular dystrophy are doing over time. Researchers want to see if the treatment is safe and effective in the long run. No new treatment will be given during this study.
United States · Belgium · Germany - recruiting
Natural History of Duchenne Muscular Dystrophy
This study looks at how Duchenne muscular dystrophy (DMD) naturally progresses in boys aged 4 to 9. The goal is to understand the disease better and prepare for future gene therapy trials. Researchers will follow boys for several months to learn more about DMD.
Belgium · France · Spain - active not recruitingPHASE2
A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)
This study looks at a new gene therapy called delandistrogene moxeparvovec for boys with Duchenne Muscular Dystrophy (DMD) under four years old. It checks how safe it is and if it helps the body make a missing protein.
Belgium · France · Germany - recruitingPHASE1, PHASE2
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
This study looks at a new gene therapy, SGT-003, for Duchenne muscular dystrophy. We want to see if it's safe and how well it works. Boys aged 0-17 with Duchenne can take part. It involves a single treatment and long-term follow-up.
United States · Canada · Italy - authorisedPhase I and Phase II (Integrated)- First administration to humans
A PHASE 1/2, OPEN-LABEL, EXPLORATORY CLINICAL TRIAL TO EVALUATE THE SAFETY AND EFFICACY OF DT-DEC01 THERAPY IN PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY
This study is testing a new treatment, DT-DEC01, for Duchenne Muscular Dystrophy. It aims to see if it's safe and how well it helps improve muscle function in both patients who can walk and those who can't.
Poland - authorisedTherapeutic exploratory (Phase II)
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of Weekly Intravenous Infusions of BMN 351 in Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
This study is for men with Duchenne muscular dystrophy, aged 18 and over. It's looking into the long-term safety and how well a new medicine, BMN 351, works. This medicine is given weekly by drip and helps with a specific type of Duchenne.
Spain · Italy · Netherlands - authorisedTherapeutic confirmatory (Phase III)
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Global Study with an Open-Label Extension to Evaluate the Efficacy and Safety of Intravenous AOC 1044 (delpacibart zotadirsen) for the Treatment of DMD with Gene Mutations Amenable to Exon 44 Skipping
This study is testing a new medicine, AOC 1044, for boys and men with Duchenne muscular dystrophy (DMD) who have a specific genetic change. It compares the new medicine to a harmless substance (placebo) to see if it's safe and helps improve muscle function.
Italy · Germany · France - authorisedTherapeutic confirmatory (Phase III)
A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy
This study is looking at a new treatment called SGT-003 for Duchenne muscular dystrophy in boys who can still walk. It aims to see if a single dose helps them to get up faster from the floor over about a year and a half.
Spain · Netherlands · France - ongoing, recruitingTherapeutic exploratory (Phase II)
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
Netherlands · Spain · Belgium - ongoing, recruitingTherapeutic exploratory (Phase II)
A multicentre, randomised, double-blind, placebo-controlled and open label extension study to assess the efficacy, safety, and pharmacokinetic profile of two dose levels of ATL1102 administered by subcutaneous injection in nonambulatory participants with Duchenne Muscular Dystrophy
Bulgaria