Duchenne muscular dystrophy: open trials in Italy

19 open trials have a site in Italy. Registry data; only the trial team decides who can take part.

2023-504520-26-00
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
Trial Treatment · Phase 3 · Italfarmaco S.p.A.
Associazione La Nostra Famiglia, Bosisio Parini · IRCCS Istituto Giannina Gaslini, Genoa · Azienda Ospedaliera Universitaria Gaetano Martino Messina, Messina · Centro Clinico Nemo, Milan · IRCCS Foundation Istituto Neurologico Carlo Besta, Milan · Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Milan · Universita Degli Studi Di Padova, Padova · Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome · Ospedale Pediatrico Bambino Gesù, Rome
Registry updated 2026-11-06
NCT07890883
Bone Health in Adults With Duchenne Muscular Dystrophy: A Survey on Osteoporosis, Fractures and Treatment.
Research · Istituto Giannina Gaslini
Centro Malattie Neuromuscolari dell'età Evolutiva, Bologna, Bologna · Associazione La Nostra Famiglia Eugenio Medea IRCCS, Bosisio Parini, Bosisio Parini · Associazione La Nostra Famiglia Eugenio Medea IRCCS, Conegliano, Conegliano · IRCCS Istituto Giannina Gaslini, Genova · Dipartimento di Scienze Cliniche e Sperimentali, Università di Brescia, Gussago · Azienda Universitaria Policlinico G. Martino, Messina · Università degli Studi di Milano Statale, Milan · Fondazione IRCCS Istituto. Neurologico "C. Besta", Milano, Milan · Università degli Studi della Campania Luigi Vanvitelli, Napoli, Naples · Dipartimento di Neuroscienze Università di Padova, Padova · Fondazione Stella Maris IRCCS-Pisa, Pisa · Dipartimento di Medicina Clinica e Sperimentale, Università di Pisa, Pisa · Fondazione Policlinico Universitario Agostino Gemelli - ROMA, Roma · Dipartimento di Neuroscienze IRCCS Ospedale Bambino Gesù, Roma, Roma · Dipartimento di Scienze Mediche, Chirurgiche e Neuroscienze, Siena, Siena
Registry updated 2026-10-22
NCT07879989
Respiratory Progression in Adults With Duchenne Muscular Dystrophy.
Research · Istituto Giannina Gaslini
Istituto delle Scienze Neurologiche di Bologna - IRCCS, Bologna · Associazione La Nostra Famiglia Eugenio Medea - IRCCS, Bosisio Parini · Fondazione Stella Maris - IRCCS, Calambrone · IRCCS Istituto Giannina Gaslini, Genova · Azienda Ospedaliera Policlinico Universitario "G. Martino", Messina · Centro Clinico Nemo, Milan · Fondazione Istituto Neurologico Carlo Besta - IRCCS, Milan · Università Vita-Salute San Raffaele, Milan · Dipartimento di Neuroscienze DNS, Università di Padova, Padova · Fondazione Istituto Neurologico Nazionale Casimiro Mondino IRCCS, Pavia · Università di Pisa, Pisa · Fondazione Policlinico Universitario Agostino Gemelli, Roma · Dipartimento di Neuroscienze Rita Levi Montalcini, Università di Torino, Torino
Registry updated 2026-10-19
NCT07587242
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
Trial Treatment · Phase 3 · Avidity Biosciences, Inc.
Istituto Giannina Gaslini, Genova · IRCCS Ospedale San Raffaele, Milan · Fondazione Serena ETS - Centro Clinico NeMO Milano, Milan · Fondazione Policlinico Universitario A. Gemelli IRCCS - Universitario Cattolica del Sacro Cuore, Roma
Registry updated 2026-10-13
2024-511823-32-00
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
Trial Treatment · Phase 2 · Italfarmaco S.p.A.
Centro Clinico Nemo, Milan · Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome · Bambino Gesu Childrens Hospital, Rome
Registry updated 2026-10-09
NCT07160634
A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
Trial Treatment · Phase 3 · Solid Biosciences Inc.
Centro Clinico Nemo, Milan · Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome
Registry updated 2026-09-28
NCT06066580
Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy
Trial Treatment · Phase 2 · Edgewise Therapeutics, Inc.
Fondazione IRCCS Ca' Granda Ospedale Maggiore, Milan · Azienda Ospedale Universita Padova, Padova · Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore, Rome
Registry updated 2026-09-17
NCT07037862
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44
Trial Treatment · Phase 1/2 · Entrada Therapeutics, Inc.
San Raffaele Hospital, Milan · Centro Clinico Nemo, Milan · Ospedale Pediatrico Bambino Gesù, Rome
Registry updated 2026-08-13
NCT07038824
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45
Trial Treatment · Phase 1/2 · Entrada Therapeutics, Inc.
Ospedale San Raffaele S.r.l., Milan · Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome · Ospedale Pediatrico Bambino Gesù, Rome
Registry updated 2026-08-13
NCT05933057
Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy
Trial Treatment · Phase 3 · Italfarmaco
Centro Clinico Nemo, Milan · Azienda Ospedale Universita Padova, Padova · Associazione La Nostra Famiglia, Ponte Lambro · Bambino Gesu Childrens Hospital, Rome · Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome
Registry updated 2026-07-24
NCT07573631
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
Trial Treatment · Phase 2 · BioMarin Pharmaceutical
Centro Clinico Nemo, Milan · Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome
Registry updated 2026-07-24
NCT06138639
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Trial Treatment · Phase 1/2 · Solid Biosciences Inc.
Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome
Registry updated 2026-07-08
NCT07682129
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)
Trial Treatment · Phase 2 · Entrada Therapeutics, Inc.
Centro Clinico Nemo, Milan · Ospedale San Raffaele S.r.l., Milan · Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome · Ospedale Pediatrico Bambino Gesù, Rome
Registry updated 2026-07-02
2025-523087-20-00
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
Trial Treatment · Phase 3 · Avidity Biosciences Inc.
IRCCS Istituto Giannina Gaslini, Genoa · Centro Clinico Nemo, Milan · Ospedale San Raffaele S.r.l., Milan · Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome
Registry updated 2026-05-27
NCT05967351
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
Trial Treatment · Phase 3 · Sarepta Therapeutics, Inc.
Giannina Gaslini Institute For Scientific Hospitalization And Care, Genoa · Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Milan · Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome
Registry updated 2026-02-06
NCT03373968
Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
Trial Treatment · Phase 2/3 · Italfarmaco
U.O.S.D. Centro Traslazionale di Miologia e Patologie Neurodegenerative, Building 16 - ground floor IRCCS Istituto Giannina Gaslini,, Genova · A.O.U. Policlinico G. Martino, U.O.C. Neurologia e Malattie Neuromuscolari, Messina · IRCCS Istituto Neurologico Carlo Besta, Milan · Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano, UOS di Neurologia Pediatrica, Milan · Centro Clinico NeMO Fondazione Serena ONLUS Area SUD, Milan · Ospedale Pediatrico Bambino Gesù, Malattie Neuromuscolari e Neurodegenerative, Roma · Fondazione Policlinico Universitario "A.Gemelli", UOC Neuropsichiatria Infantile, Roma
Registry updated 2026-01-21
NCT07129954
Fear of Falling in Muscular Dystrophy
Trial Treatment · Universita di Verona
Azienda Ospedaliera Universitaria Integrata Verona - UOC di Neuroriabilitazione, Verona
Registry updated 2025-08-19
NCT07101185
Transition to Adulthood in People With Muscular Dystrophy
Trial Treatment · IRCCS Eugenio Medea
Unit of Rare Disease- Rehabilitation Dept, Bosisio Parini
Registry updated 2025-08-03
NCT06769633
Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old
Trial Treatment · Phase 2 · Italfarmaco
Fondazione Serena Onlus - Azienda Ospedaliera Niguarda Ca' Granda - NeuroMuscolar Omnicenter, Milan · Ospedale Pediatrico Bambino Gesù, Roma · Policlinico Universitario Agostino Gemelli - Università Cattolica del Sacro Cuore, Roma
Registry updated 2025-07-10

All Duchenne muscular dystrophy trials and the map

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