Duchenne muscular dystrophy trials in Belgium

In Belgium 19 Also abroad 87

19 trials · From ClinicalTrials.gov and EU CTIS (euclinicaltrials.eu), 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
Leuven, Liège
Age
7 years to 16 years
People
70 worldwide
Run by
Avidity Biosciences, Inc.
See details and how to joinNCT07587242
Phase 3Taking people
Study in which all the patients take the same investigational drug with the aim of evaluate the long-term safety, tolerability, and efficacy of GIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophy who have been already treated in one of the GIVINOSTAT studies in the past
Where
Leuven, Liège
Age
Up to 64 years
People
207 worldwide
Run by
Italfarmaco S.p.A.
See details and how to join2023-504520-26-00 · EU 2023-504520-26-00
Phase 2/3Taking people
A Clinical Study to Assess Safety, Efficacy and Pharmacodynamic Profile of GNT0004 in Duchenne Muscular Dystrophy
Where
Brussels, Leuven, Liège
Age
6 years to 10 years
People
77 worldwide
Run by
Genethon
See details and how to joinNCT07856329 · EU 2023-505187-11-00
Phase 2Taking people
A Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Than 6 Years Old
Where
Brussels
Age
Up to 17 years
People
10 worldwide
Run by
Italfarmaco S.p.A.
See details and how to join2024-511823-32-00 · EU 2024-511823-32-00
Phase 3Taking people
A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
Where
Brussels, Leuven
Age
7 years to 11 years
People
80 worldwide
Run by
Solid Biosciences Inc.
See details and how to joinNCT07160634 · EU 2025-522949-22-00
Phase 2Enrolling by invitation
Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy
Where
Ghent, Leuven, Liège
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
Gent, Leuven, Liège
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
Gent, Leuven, Liège
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
Leuven
Age
9 years to 17 years
People
138 worldwide
Run by
Italfarmaco
See details and how to joinNCT05933057 · EU 2023-503521-19-00
Phase 2Not yet taking people in Belgium
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
Gent, Leuven, Liège
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 phaseNot yet taking people in Belgium
Digital Monitoring of Upper Limb Function in Non-Ambulant DMD
Where
Liège
People
50
Run by
Centre Hospitalier Universitaire de Liege
See details and how to joinNCT07664124
Phase 3Taking people
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
Where
Leuven, Liège
Age
Up to 17 years
People
42 worldwide
Run by
Avidity Biosciences Inc.
See details and how to join2025-523087-20-00 · EU 2025-523087-20-00
No phaseTaking people
Natural History of Duchenne Muscular Dystrophy
Where
Brussels, Leuven, Liège
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
Gent
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
Leuven, Liège
Age
7 years and over
People
206 worldwide
Run by
Italfarmaco
See details and how to joinNCT03373968
No phaseTaking people
Active NBS Study: Decentralised Monitoring Motor Development in Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified by Newborn Screening
Where
Liège
Age
4 months and over
People
100
Run by
Centre Hospitalier Universitaire de Liege
See details and how to joinNCT07286565
Phase 2Taking people
Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old
Where
Brussels
Age
2 years to 6 years
People
18 worldwide
Run by
Italfarmaco
See details and how to joinNCT06769633
No phaseTaking people
Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study
Where
Liège
Age
1 year to 80 years
People
300 worldwide
Run by
Centre Hospitalier Universitaire de Liege
See details and how to joinNCT05982119
No phaseTaking people
Follow-up of NIV At Home in Patients with Duchenne Muscular Dystrophy
Where
Leuven
Age
18 years and over
People
17
Run by
Universitaire Ziekenhuizen KU Leuven
See details and how to joinNCT06773988