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Exogenous Gene Transfer

delandistrogene moxeparvovec for Duchenne Muscular Dystrophy

Phase 1 & 2
Waitlist Available
Research Sponsored by Sarepta Therapeutics, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline

Summary

This trial is testing a new gene therapy for patients with Duchenne Muscular Dystrophy. The therapy aims to help their bodies produce a missing protein needed for healthy muscles. The study will measure safety and effectiveness over time.

Eligible Conditions
  • Duchenne Muscular Dystrophy
  • Muscular Dystrophy

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, week 48 (part 1)
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 48 (part 1) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content
Change From Baseline at Week 48 in NSAA Total Score
Secondary study objectives
Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression Measured by IF Percent Dystrophin Positive Fibers (PDPF)
Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression Measured by Immunofluorescence (IF) Fiber Intensity
Change From Baseline at Week 48 in Time of 10-meter Timed Test
+4 more
Other study objectives
Baseline NSAA Total Score by Age Group
Change From Baseline at Week 48 in NSAA Total Score by Age Group

Trial Design

2Treatment groups
Experimental Treatment
Group I: Placebo in Part 1 Followed by Delandistrogene Moxeparvovec in Part 2Experimental Treatment2 Interventions
Participant will receive matching placebo at Part 1 followed by delandistrogene moxeparvovec at Part 2 followed by an open-label extension at Part 3.
Group II: Delandistrogene Moxeparvovec in Part 1 Followed by Placebo in Part 2Experimental Treatment2 Interventions
Participant will receive delandistrogene moxeparvovec at Part 1 followed by matching placebo at Part 2 followed by an open-label extension at Part 3.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
delandistrogene moxeparvovec
2018
Completed Phase 2
~50
placebo
2010
Completed Phase 4
~6580

Find a Location

Who is running the clinical trial?

Sarepta Therapeutics, Inc.Lead Sponsor
52 Previous Clinical Trials
33,927 Total Patients Enrolled
Medical DirectorStudy DirectorSarepta Therapeutics, Inc.
2,892 Previous Clinical Trials
8,089,281 Total Patients Enrolled

Media Library

Duchenne Muscular Dystrophy Clinical Trial 2023: SRP-9001 Highlights & Side Effects. Trial Name: NCT03769116 — Phase 1 & 2
~6 spots leftby Dec 2025
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