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Abstract Details

EMBARK, a Phase 3 Trial Evaluating Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Duchenne Muscular Dystrophy (DMD): Study Design and Baseline Characteristics
Neuromuscular and Clinical Neurophysiology (EMG)
P5 - Poster Session 5 (11:45 AM-12:45 PM)
10-012
To describe the EMBARK (Study 301; NCT05096221) study design, a Phase 3, global, randomized, double-blind, two-part, placebo-controlled study assessing the safety and efficacy of intended commercial process delandistrogene moxeparvovec (SRP-9001) material in ambulatory individuals with a confirmed Duchenne muscular dystrophy (DMD) mutation within exons 18-79 (excluding individuals with a mutation fully contained within exon 45), aged ≥4 to <8 years (N=125). 
Delandistrogene moxeparvovec is an investigational gene transfer therapy developed to address the root cause of DMD through targeted skeletal and cardiac muscle expression of SRP-9001 dystrophin protein, which contains key functional domains of dystrophin.

In Part 1, participants will be stratified by age at randomization (≥4 to <6 years or ≥6 to <8 years) and North Star Ambulatory Assessment total score (NSAA ≤22 points or >22 points) at screening and randomized (1:1) to receive a single intravenous dose of intended commercial process delandistrogene moxeparvovec material (1.33x1014 vg/kg by linear standard qPCR) or placebo. Participants will be evaluated at Week 52. In Part 2 (52-week follow-up period), participants randomized to placebo in Part 1 will receive delandistrogene moxeparvovec and participants randomized to delandistrogene moxeparvovec in Part 1 will receive placebo.

The primary endpoint is change from baseline to Week 52 in NSAA total score (Part 1). Secondary endpoints include safety; SRP-9001 dystrophin protein production at Week 12 by western blot and change from baseline to Week 52 in: key timed function tests, stride velocity 95th centile measured by a wearable device, and Patient-Reported Outcomes Measurement Information Score®.

For the first time, we present baseline characteristics of participants enrolled in EMBARK, a Phase 3 study of delandistrogene moxeparvovec.
EMBARK will provide placebo-controlled information about efficacy and safety of delandistrogene moxeparvovec in a large population of ambulatory patients with DMD aged ≥4 to <8 years. 
Authors/Disclosures
Francesco Muntoni, MD (UCL Institute of Child Health)
PRESENTER
Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sarepta. Dr. Muntoni has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Biogen. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Novartis. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche. The institution of Dr. Muntoni has received research support from European Commission. The institution of Dr. Muntoni has received research support from Medical Research Council. The institution of Dr. Muntoni has received research support from Biogen. The institution of Dr. Muntoni has received research support from Muscular Dystrophy UK. The institution of Dr. Muntoni has received research support from MDA USA. The institution of Dr. Muntoni has received research support from Sarepta. The institution of Dr. Muntoni has received research support from Association Francoise Myopathies. Dr. Muntoni has received personal compensation in the range of $0-$499 for serving as a Clinical expert with UK NICE Committee.
No disclosure on file
No disclosure on file
Hirofumi Komaki No disclosure on file
No disclosure on file
Tejdip Singh (Sarepta) Tejdip Singh has received personal compensation for serving as an employee of Sarepta Therapeutics . Tejdip Singh has received stock or an ownership interest from Sarepta Therapeutics.
No disclosure on file
Stefanie Mason, MD Dr. Mason has received personal compensation for serving as an employee of Sarepta. Dr. Mason has stock in Sarepta. An immediate family member of Dr. Mason has stock in Sarepta. The institution of an immediate family member of Dr. Mason has received research support from Biogen. The institution of Dr. Mason has received research support from NIH. The institution of Dr. Mason has received research support from Chest Foundation.
No disclosure on file
No disclosure on file
No disclosure on file
Eddie Darton (Sarepta Therapeutics) No disclosure on file
Christoph Wandel, MD, PhD Christoph Wandel, MD, PhD has received personal compensation for serving as an employee of F Hoffmann-LaRoche AG. Christoph Wandel, MD, PhD has stock in F Hoffmann-La Roceh AG, Basel, CH; Bayer AG, Leverkusen, FRG.
Jerry R. Mendell, MD, FAAN (The Research Institute at Nationwide Children's Hospital) Dr. Mendell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Vertex. Dr. Mendell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Novartis. Dr. Mendell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta Therapeutics . The institution of Dr. Mendell has received research support from Sarepta.