Duchenne muscular dystrophy (DMD) research hub
10 indexed studies · 5 currently recruiting
- ongoing, recruitingHuman Pharmacology (Phase I)- Other
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
This study is looking at a new gene therapy for Duchenne muscular dystrophy in males who already have certain antibodies. It's testing if the treatment is safe, how well people tolerate it, and if the therapy's active ingredient can be produced in their muscles.
Spain - enrolling by invitationPHASE2
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
This study looks at the long-term effects of a medicine called BMN 351 for boys and young men with Duchenne Muscular Dystrophy. It's for those who have already started taking BMN 351 in an earlier study and will continue to check its safety and how well it helps with physical movement.
Italy · Netherlands · Spain - recruitingPHASE1, PHASE2
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44
This study looks at a new medicine, ENTR-601-44, for Duchenne muscular dystrophy (DMD). We want to see if it's safe, what side effects it might have, and if it helps people with DMD. It involves regular check-ups and is for males aged 4-20.
Belgium · Italy · Spain - ongoing, recruitingTherapeutic confirmatory (Phase III)
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)
This study is looking at a medicine called Viltolarsen for boys with Duchenne Muscular Dystrophy (DMD) who can still walk. It aims to check if the medicine is safe and if it helps them. This is an ongoing study where participants who have been in previous trials can continue with the treatment.
Spain · Italy · Greece - active not recruitingPHASE1, PHASE2
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
This study is looking at a new medicine called DYNE-251 for boys aged 4-16 with Duchenne Muscular Dystrophy (DMD). It aims to see if the medicine is safe, well-tolerated, and helps the body make more dystrophin protein.
United States · Australia · Belgium - not yet recruitingPHASE2
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)
This study looks at new trial medicines, ENTR-601-44 and ENTR-601-45, for boys and young men with Duchenne Muscular Dystrophy (DMD). It checks their long-term safety and how well they work to improve muscle health.
Belgium · Italy · Netherlands - recruitingPHASE1, PHASE2
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45
This study looks at a new medicine, ENTR-601-45, for boys aged 4-20 with Duchenne muscular dystrophy (DMD) where skipping exon 45 is possible. We want to see if it's safe, what side effects it might have, and if it could help. It involves regular clinic visits and infusions.
Belgium · Italy · Netherlands - active not recruitingPHASE1
A Trial to Investigate the Safety and Pharmacokinetics of GRT6019 in Healthy Male Participants
This study is looking at a new medicine called GRT6019, which might help people with Duchenne Muscular Dystrophy. We want to see if it's safe and how it works in healthy men. This is the first step to understand this new medicine.
France - recruiting
A Remote Study Using Technology to Assess Outcomes in DMD
This study uses a watch-like device to track movement in young boys with Duchenne muscular dystrophy (DMD) and healthy boys, aged 1-3. It aims to see if this technology can effectively measure movement changes, helping to test new DMD medicines sooner and reduce hospital visits for families.
United Kingdom - recruitingPhase I/II
A 2-part, randomized, double-blind, placebo-controlled study in participants with Duchenne muscular dystrophy amenable to exon 44 skipping to evaluate the safety and efficacy of ENTR-601-44 (ELEVATE-44)
This study looks at a new medicine, ENTR-601-44, for Duchenne muscular dystrophy (DMD) in boys and young men aged 4-20. We want to see if it's safe, what side effects it has, and if it helps muscles work better by increasing a protein called dystrophin.
United Kingdom · Belgium · Italy