The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
148
Single IV infusion of delandistrogene moxeparvovec
Single IV infusion of matching placebo
Arkansas Children's Hospital
Little Rock, Arkansas, United States
Lucile Packard Children's Hospital Stanford
Palo Alto, California, United States
University of California at Davis Medical Center
Sacramento, California, United States
Rady Children's Hospital-San Diego
San Diego, California, United States
University of Florida, UF Health Center for Pediatric Neuromuscular and Rare Diseases
Gainesville, Florida, United States
Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72
Time frame: Baseline, Week 72
Part 1: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 72
Time frame: Baseline, Week 72
Part 1: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72
Time frame: Baseline, Week 72
Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot
Time frame: Week 12
Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 72
Time frame: Baseline, Week 72
Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE)
Time frame: Baseline up to Week 124
Part 1 (For Cohort 2 Only): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 72
Time frame: Baseline, Week 72
Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72
Time frame: Baseline, Week 72
Part 1: Change From Baseline in the Middle Domain Score of PUL (Version 2.0) at Week 72
Time frame: Baseline, Week 72
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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
The Johns Hopkins Hospital, Charlotte R. Bloomberg Children's Center, Pediatric Clinical Research Unit
Baltimore, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Washington University of St. Louis, St. Louis Children's Hospital
St Louis, Missouri, United States
University of Rochester, Department of Neurology
Rochester, New York, United States
...and 36 more locations