Duchenne Muscular Dystrophy research hub
31 indexed studies · 17 currently recruiting
- recruitingTherapeutic confirmatory (Phase III)
A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of High Doses of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping
This clinical trial is testing whether higher doses of a medicine called eteplirsen are safe and helpful for boys with Duchenne muscular dystrophy. It is for males with a specific genetic change that can be treated by skipping exon 51, aiming to protect muscle strength over nearly three years.
Italy · Denmark · Czechia - active not recruitingPHASE3
Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)
This study is testing a medicine called viltolarsen for boys with Duchenne muscular dystrophy who can still walk. It is an extension study for children who already completed an earlier trial. Every participant receives the medicine through a weekly drip for nearly two years to check its safety and long-term effects.
Australia · Canada · Chile - 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
This study is testing a new medicine called VESLETEPLIRSEN for Duchenne muscular dystrophy in males over 18. It aims to find the best dose and check how safe it is, while also measuring its effect on a muscle protein called dystrophin.
Netherlands · Spain · Belgium - 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
This study is looking at a new medicine called ATL1102 for Duchenne Muscular Dystrophy in boys who can't walk. It wants to see if the medicine helps improve arm and hand function and if it's safe. Some boys will get the real medicine, and some will get a dummy medicine at first.
Bulgaria - 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 - 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 - not yet recruitingPhase II
A Phase II, open-label long-term extension study in participants with Duchenne muscular dystrophy amenable to exon skipping to assess the long-term safety, tolerability, pharmacokinetics, and efficacy of endosomal escape vehicle phosphorodiamidate morpholino oligomer platform products (ELEVATE-LTE)
This clinical trial is for males with Duchenne muscular dystrophy who took part in an earlier study of the experimental medicines ENTR-601-44 or ENTR-601-45. Researchers want to see if continuing this treatment over a longer time is safe and helps protect muscle strength. Every participant receives the active medicine.
United Kingdom · England · Belgium - recruitingPhase I/II
A 2-part, randomized, double-blind, placebo-controlled study in participants with Duchenne muscular dystrophy amenable to exon 45 skipping to evaluate the safety and efficacy of ENTR-601-45 (ELEVATE-45)
This study, ELEVATE-45, is testing a new medicine called ENTR-601-45 for Duchenne muscular dystrophy (DMD). It aims to see if it's safe, what side effects it has, and if it helps by increasing a muscle-building protein called dystrophin in boys and young men aged 4-20.
United Kingdom · England · Belgium - active not 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 - active not 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 - recruitingPHASE2, PHASE3
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
This study is testing a new gene therapy called RGX-202 for boys with Duchenne muscular dystrophy. It aims to see if it's safe and helps muscles work better by delivering a special protein that's missing.
United States · Canada - 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 - 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 - available
Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy
This program helps boys with Duchenne muscular dystrophy in Canada continue to receive a medicine called vamorolone. It's for those who have already been in earlier studies, allowing them to keep taking the medication while it awaits wider approval. It is not currently looking for new participants.
United States · Canada · Israel - recruitingPHASE2
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
This study is testing a new medicine, NS-089/NCNP-02, for Duchenne muscular dystrophy (DMD) in boys aged 4 to 14. It's for those with a specific gene change (exon 44 skipping) and involves weekly IV infusions to see if the medicine is safe and helps their condition.
United States · Australia · Canada - 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 - 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 - active not 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.
Italy · Netherlands · Spain - recruitingPHASE3
A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
This study is testing a new gene therapy, SGT-003, for boys aged 7-11 with Duchenne Muscular Dystrophy who can still walk. It's comparing SGT-003 to a dummy treatment (placebo) to see if it helps and is safe. Participants will be monitored for at least 5 years.
United States · Australia · Canada - 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 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
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 - 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 · Czechia - 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 - 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 - 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 - 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 - 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 - 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 - 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 - 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