Duchenne muscular dystrophy trials in United Kingdom

In United Kingdom 17 Also abroad 87

17 trials · From ClinicalTrials.gov, checked 12 November 2026. Ask the study team whether a trial fits you.

Phase 3Taking people
A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping
Where
Newcastle, Oxford
Age
7 years to 16 years
People
70 worldwide
Run by
Avidity Biosciences, Inc.
See details and how to joinNCT07587242
Phase 2/3Taking people
A Clinical Study to Assess Safety, Efficacy and Pharmacodynamic Profile of GNT0004 in Duchenne Muscular Dystrophy
Where
London, Newcastle upon Tyne
Age
6 years to 10 years
People
77 worldwide
Run by
Genethon
See details and how to joinNCT07856329 · EU 2023-505187-11-00
Phase 2Enrolling by invitation
Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy
Where
London, Newcastle, Salford
People
260 worldwide
Run by
Edgewise Therapeutics, Inc.
See details and how to joinNCT06066580
Phase 1/2Taking people
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44
Where
Leeds, Liverpool, London, Manchester, Newcastle upon Tyne, Oxford
Age
4 years to 20 years
People
24 worldwide
Run by
Entrada Therapeutics, Inc.
See details and how to joinNCT07037862 · EU 2024-517584-23-00
Phase 1/2Taking people
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45
Where
Leeds, Liverpool, London, Manchester, Oxford
Age
4 years to 20 years
People
24 worldwide
Run by
Entrada Therapeutics, Inc.
See details and how to joinNCT07038824 · EU 2024-517499-39-00
Phase 3Taking people
Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy
Where
Glasgow, Newcastle upon Tyne, Oxford
Age
9 years to 17 years
People
138 worldwide
Run by
Italfarmaco
See details and how to joinNCT05933057 · EU 2023-503521-19-00
Phase 2Enrolling by invitation
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
Where
London
Age
4 years to 64 years
People
18 worldwide
Run by
BioMarin Pharmaceutical
See details and how to joinNCT07573631 · EU 2024-511656-41-00
Phase 1/2Taking people
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Where
London
Age
0 years to 17 years
People
60 worldwide
Run by
Solid Biosciences Inc.
See details and how to joinNCT06138639 · EU 2024-514501-57-00
Phase 2Not yet taking people in United Kingdom
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)
Where
Leeds, Liverpool, London, Manchester, Newcastle upon Tyne, Oxford
Age
4 years to 20 years
People
80 worldwide
Run by
Entrada Therapeutics, Inc.
See details and how to joinNCT07682129 · EU 2025-525124-10-00
No phaseTaking people
A Remote Study Using Technology to Assess Outcomes in DMD
Where
Oxford
Age
1 year to 3 years
People
60
Run by
University of Oxford
See details and how to joinNCT07423026
Phase 2Enrolling by invitation
Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
Where
Headington
People
175 worldwide
Run by
Wave Life Sciences USA, Inc.
See details and how to joinNCT07209332
No phaseTaking people
Natural History of Duchenne Muscular Dystrophy
Where
London, Newcastle
Age
4 years to 9 years
People
220 worldwide
Run by
Genethon
See details and how to joinNCT03882827
Phase 3Enrolling by invitation
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
Where
London, Newcastle upon Tyne, Oxford
Age
Up to 64 years
People
400 worldwide
Run by
Sarepta Therapeutics, Inc.
See details and how to joinNCT05967351 · EU 2023-505043-39-00
Phase 2/3Taking people
Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
Where
Gobowen, Liverpool, London, Newcastle upon Tyne
Age
7 years and over
People
206 worldwide
Run by
Italfarmaco
See details and how to joinNCT03373968
Phase 2Not yet taking people in United Kingdom
Repurposing Empagliflozin for DMD-associated Cardiomyopathy in Children 6-18 Years of Age
Where
London
Age
6 years to 18 years
People
12
Run by
Sebastiano Lava
See details and how to joinNCT06643442
Phase 2Taking people
Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old
Where
Leeds, London, Newcastle upon Tyne, Oxford
Age
2 years to 6 years
People
18 worldwide
Run by
Italfarmaco
See details and how to joinNCT06769633
No phaseNot yet taking people in United Kingdom
The U.K. NorthStar Clinical Network
Where
Aberdeen, Belfast, Birmingham, Bristol, Cambridge, Cardiff, Dundee, Glasgow, Lancaster, Leeds, Leicester, Liverpool, London, Manchester, Newcastle, Nottingham, Oswestry, Oxford, Plymouth, Sheffield, Southampton, Swansea
Age
Up to 99 years
People
300
Run by
University College, London
See details and how to joinNCT06711692