Duchenne muscular dystrophy trials in Italy

In Italy 19 Also abroad 87

19 trials · From ClinicalTrials.gov and EU CTIS (euclinicaltrials.eu), checked 11 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
Genova, Milan, Roma
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
Bosisio Parini, Genoa, Messina, Milan, Padova, Rome
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
No phaseTaking people
Bone Health in Adults With Duchenne Muscular Dystrophy: A Survey on Osteoporosis, Fractures and Treatment.
Where
Bologna, Bosisio Parini, Conegliano, Genova, Gussago, Messina, Milan, Naples, Padova, Pisa, Roma, Siena
Age
18 years to 44 years
People
200
Run by
Istituto Giannina Gaslini
See details and how to joinNCT07890883
No phaseTaking people
Respiratory Progression in Adults With Duchenne Muscular Dystrophy.
Where
Bologna, Bosisio Parini, Calambrone, Genova, Messina, Milan, Padova, Pavia, Pisa, Roma, Torino
Age
18 years and over
People
150
Run by
Istituto Giannina Gaslini
See details and how to joinNCT07879989
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
Milan, Rome
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
Milan, Rome
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
Milan, Padova, Rome
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
Milan, Rome
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
Milan, Rome
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
Milan, Padova, Ponte Lambro, Rome
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
Milan, Rome
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
Rome
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 Italy
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
Milan, Rome
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
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
Genoa, Milan, Rome
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
Phase 3Enrolling by invitation
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
Where
Genoa, Milan, Rome
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
Genova, Messina, Milan, Roma
Age
7 years and over
People
206 worldwide
Run by
Italfarmaco
See details and how to joinNCT03373968
No phaseTaking people
Fear of Falling in Muscular Dystrophy
Where
Verona
Age
16 years to 65 years
People
100
Run by
Universita di Verona
See details and how to joinNCT07129954
No phaseNot yet taking people in Italy
Transition to Adulthood in People With Muscular Dystrophy
Where
Bosisio Parini
Age
14 years and over
People
250
Run by
IRCCS Eugenio Medea
See details and how to joinNCT07101185
Phase 2Taking people
Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old
Where
Milan, Roma
Age
2 years to 6 years
People
18 worldwide
Run by
Italfarmaco
See details and how to joinNCT06769633