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Clinical trial

Microdystrophin (GNT0004) Gene Therapy Clinical Trial in Duchenne Muscular Dystrophy: A Phase I/II/III Study With a Dose Determination Part Followed by an Efficacy and Safety Evaluation, Quadruple Blind Placebo-controlled Part and Then by a Long-term Safety Follow up Part, in Ambulant Boys

NCT07856329 is registered in ClinicalTrials.gov with Genethon as lead sponsor.

NCT NCT07856329
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Registry record

As filed in ClinicalTrials.gov.

Title
Microdystrophin (GNT0004) Gene Therapy Clinical Trial in Duchenne Muscular Dystrophy: A Phase I/II/III Study With a Dose Determination Part Followed by an Efficacy and Safety Evaluation, Quadruple Blind Placebo-controlled Part and Then by a Long-term Safety Follow up Part, in Ambulant Boys
Lead sponsor
Genethon
Phase
Phase 2/3
Overall status
Recruiting
Start date
March 1, 2021
Completion date
October 1, 2028
Enrollment
77 (estimated)
Sites
14

Conditions

As the registry lists them; a condition with its own page is linked.

duchene muscular dystrophy