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
Status: Recruiting
Location: See all (7) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 2
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.
Eligibility
Participation Requirements
Sex: Male
Maximum Age: 3
Healthy Volunteers: f
View:
• Cohort A: \>=3 years of age to \<4 years of age
• Cohort B: \>=2 years of age to \<3 years of age
• Cohort C: \>6 months to \<2 years of age
• Cohort D: \<=6 months of age
• Has a definitive diagnosis of DMD prior to screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test
• Able to cooperate with age-appropriate motor assessment testing
• A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive)
Locations
Other Locations
Belgium
Chr de La Citadelle
RECRUITING
Liège
France
Hôpital Necker-Enfants Malades
RECRUITING
Paris
Germany
Universitätsklinikum Essen
RECRUITING
Essen
Italy
PU A. Gemelli, Università Cattolica del Sacro Cuore
RECRUITING
Rome
Spain
Hospital Sant Joan De Deu
RECRUITING
Esplugues De Llobregas
United Kingdom
Great Ormond Street Hospital for Children
RECRUITING
London
John Radcliffe Hospital
RECRUITING
Oxford
Contact Information
Primary
Reference Study ID Number: BN43881 https://forpatients.roche.com/
global-roche-genentech-trials@gene.com
888-662-6728 (U.S. and Canada)
Time Frame
Start Date: 2023-11-29
Estimated Completion Date: 2033-05-30
Participants
Target number of participants: 21
Treatments
Experimental: Delandistrogene Moxeparvovec
Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1.
Related Therapeutic Areas
Sponsors
Collaborators: Sarepta Therapeutics, Inc.
Leads: Hoffmann-La Roche