Trial intelligence / Profile preview

SRP-9001 with imlifidase in DMD patients with pre-existing AAVrh74 antibodies

NCT06241950Phase 1Terminated

Sponsor
Sarepta Therapeutics
Start
Jan 2024
Primary completion
Oct 2025
Modality
Gene Editing, Gene Silencing, Viral Vectors
Countries
1
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Overview

Phase 1, open-label, single-arm trial of delandistrogene moxeparvovec in Duchenne muscular dystrophy (DMD), sponsored by Sarepta Therapeutics. Primary endpoints: Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content at 12 weeks, Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by Immunofluorescence Fiber Intensity at 12 weeks and 2 more. Started Jan 2024; status: terminated.

02

Drugs

delandistrogene moxeparvovec
03

Conditions

Duchenne muscular dystrophy (DMD)

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