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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 up to week 48 (part 1)
Awards & highlights

Study Summary

This trial will test a new gene therapy for Duchenne Muscular Dystrophy. The safety and effectiveness will be measured in three parts over the course of several years.

Eligible Conditions
  • Duchenne 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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
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 North Star Ambulatory Assessment (NSAA) Total Score
Secondary outcome measures
Dystrophin
Change From Baseline in Quantity of Micro-dystrophin Expression Measured by Immunofluorescence (IF) Fiber Intensity
Change From Baseline in Time of 10 Meter Timed Test
+3 more
Other outcome measures
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
49 Previous Clinical Trials
33,498 Total Patients Enrolled
Medical DirectorStudy DirectorSarepta Therapeutics, Inc.
2,777 Previous Clinical Trials
8,063,445 Total Patients Enrolled

Media Library

Duchenne Muscular Dystrophy Clinical Trial 2023: SRP-9001 Highlights & Side Effects. Trial Name: NCT03769116 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is eligibility for this experiment open to me?

"Candidates for the trial must possess Becker Muscular dystrophy and be between 4-7 years old. Currently, 41 participants are required for this research project."

Answered by AI

Are participants still being accepted into this investigation?

"Clinicaltrials.gov reveals that this trial, which initially launched on December 22nd 2018 and was last updated June 29th 2022, is no longer recruiting patients. Fortunately, there are 86 other medical studies actively enrolling participants at the present time."

Answered by AI

Does this clinical trial accept participants aged below 70 years?

"The enrollment requirements of this medical trial restrict participants to children between the ages 4 and 7. Overall, there are 60 clinical trials targeting minors while 49 remain available for seniors 65 or older."

Answered by AI

What risk factors are associated with administering SRP-9001 to patients?

"The safety of SRP-9001 has been judged a 2 on our scale as this is currently in Phase 2, meaning that there are data available to support its safety but none confirming efficacy."

Answered by AI

Who else is applying?

How old are they?
< 18
What portion of applicants met pre-screening criteria?
Met criteria
Recent research and studies
clinicaltrials.govStudy
A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)
2018. "A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03769116.
~6 spots leftby Apr 2025
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