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A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)
Sponsor: Hoffmann-La Roche
Summary
This open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks.
Official title: A Two-Part, Open-Label Systemic Gene Delivery Study to Evaluate the Safety and Expression of RO7494222 (SRP-9001) in Subjects Under the Age of Four With Duchenne Muscular Dystrophy
Key Details
Gender
MALE
Age Range
2 Years - 3 Years
Study Type
INTERVENTIONAL
Enrollment
13
Start Date
2023-11-29
Completion Date
2034-01-30
Last Updated
2026-02-10
Healthy Volunteers
No
Conditions
Interventions
delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec
Locations (7)
Chr de La Citadelle
Liège, Belgium
Hôpital Necker-Enfants Malades
Paris, France
Universitätsklinikum Essen
Essen, Germany
PU A. Gemelli, Università Cattolica del Sacro Cuore
Rome, Lazio, Italy
Hospital Sant Joan De Deu
Esplugues de Llobregas, Barcelona, Spain
Great Ormond Street Hospital for Children
London, United Kingdom
John Radcliffe Hospital
Oxford, United Kingdom