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Gene Transfer Therapy

Cohort A: 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
Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
Cohort A participants: 3 months to 3 years of age, inclusive
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights

Study Summary

This trial is testing a new gene therapy for Duchenne Muscular Dystrophy in boys. The boys will receive the therapy through IV. The trial will test the safety of the therapy in two groups of boys, 3 months to 3 years old, and 4 to 7 years old.

Eligible Conditions
  • Duchenne Muscular Dystrophy

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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You have been diagnosed with DMD through medical exams and genetic testing.
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You are between the ages of 3 months and 3 years old.
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You must be between 4 and 7 years old to participate in this study.
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You are able to participate in tests that measure your physical abilities.
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You have not taken corticosteroids as a treatment before.
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If you are in Cohort B, you must have been taking a steady dose of oral corticosteroids for at least 12 weeks before screening, and this dose should not change during the first year of the study except for adjustments for changes in weight.
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You have a certain type of genetic mutation that occurs between exons 18 and 58.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Cohorts A and B: Number of Participants with Adverse Events (AEs)
Secondary outcome measures
Cohort A: Gross Motor Subtest Scaled (Bayley-III) Score
Cohorts A and B: Change From Baseline of Delandistrogene Moxeparvovec Dystrophin Expression Quantification by Immunofluorescence
Cohorts A and B: Change From Baseline of Delandistrogene Moxeparvovec Dystrophin Expression Quantification by Western Blot
+1 more
Other outcome measures
Physical Therapy Assessments North Star Ambulatory Assessment (NSAA)

Trial Design

2Treatment groups
Experimental Treatment
Group I: Cohort B: Delandistrogene MoxeparvovecExperimental Treatment1 Intervention
Participants will receive a Single IV infusion of delandistrogene moxeparvovec on Day 1.
Group II: Cohort A: Delandistrogene MoxeparvovecExperimental Treatment1 Intervention
Participants will receive a Single IV infusion of delandistrogene moxeparvovec on Day 1.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
delandistrogene moxeparvovec
2018
Completed Phase 2
~50

Find a Location

Who is running the clinical trial?

Sarepta Therapeutics, Inc.Lead Sponsor
49 Previous Clinical Trials
23,401 Total Patients Enrolled
Medical DirectorStudy DirectorSarepta Therapeutics, Inc.
2,769 Previous Clinical Trials
8,062,196 Total Patients Enrolled

Frequently Asked Questions

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

Are there any available positions for subjects in this research project?

"This particular study is no longer recruiting patients, however based on the information found on clinicaltrials.gov, it appears that the study was last edited on 7/4/2022 and was originally posted on 1/4/2018. There are 88 other studies that are actively searching for candidates right now."

Answered by AI

Are people of all ages being accepted for this clinical trial?

"3 month old to 7 year olds are the only age group applicable for this particular trial. Out of the total 111 trials, 61 are designed for children and 50 are meant for elderly patients."

Answered by AI

For which kind of patients is this research relevant?

"This clinical trial is testing a new medication for Duchenne muscular dystrophy and is currently looking for 4 participants that meet the following eligibility criteria: 3 months to 7 years old, definitive diagnosis of DMD, ability to cooperate with motor assessment testing, no previous treatment with corticosteroids (for Cohort A), or stable dose of oral corticosteroids for at least 12 weeks prior to screening (for Cohort B)."

Answered by AI

Who else is applying?

What state do they live in?
Georgia
How old are they?
< 18
What site did they apply to?
Nationwide Children's Hospital
What portion of applicants met pre-screening criteria?
Met criteria
~1 spots leftby Mar 2025