A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)

Study Overview
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Brief Summary
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This is an open-label single-dose gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec intravenous (IV) administration in boys with DMD. This study will consist of 2 Cohorts. Cohort A will include participants ages 3 months to 3 years, and Cohort B will include participants ages 4 to 7 years old. All participants in the study will receive IV delandistrogene moxeparvovec.

Official Title
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Systemic Gene Delivery Phase I/IIa Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MHCK7.Micro-dystrophin (microDys-IV-001)

Conditions
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Duchenne Muscular Dystrophy

Intervention / Treatment
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* Genetic: delandistrogene moxeparvovec


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Cohort A participants: 3 months to 3 years of age, inclusive Cohort B participants: 4 to 7 years of age, inclusive Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing. Ability to cooperate with motor assessment testing. Cohort A participants: No previous treatment with corticosteroids. Cohort B participants: Stable dose equivalent of oral corticosteroids for at least 12 weeks prior to screening and the dose is expected to remain constant (except for potential modifications to accommodate changes in weight) throughout the first year of the study. Cohorts A & B: A frameshift mutation contained between exons 18 and 58 (inclusive). Exclusion Criteria: Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol specified time limits. Abnormality in protocol-specified diagnostic evaluations or laboratory tests. Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer. Other inclusion or exclusion criteria could apply.

Ages Eligible for Study
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Minimum Age: 3 Months
Maximum Age: 7 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Cohort A: Delandistrogene Moxeparvovec<br>Participants will receive a Single IV infusion of delandistrogene moxeparvovec on Day 1. | Genetic: delandistrogene moxeparvovec<br>* Single IV infusion of delandistrogene moxeparvovec.<br>* Other names: ELEVIDYS;|
| Experimental: Cohort B: Delandistrogene Moxeparvovec<br>Participants will receive a Single IV infusion of delandistrogene moxeparvovec on Day 1. | Genetic: delandistrogene moxeparvovec<br>* Single IV infusion of delandistrogene moxeparvovec.<br>* Other names: ELEVIDYS;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Cohorts A and B: Number of Participants with Adverse Events (AEs) |  | Up to 5 Years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Cohort A: Gross Motor Subtest Scaled (Bayley-III) Score |  | Day 30 up to 3 Years | 
| Cohorts A and B: The 100 Meter Timed Test (100m) Physical Therapy Assessment |  | Up to 5 Years | 
| Cohorts A and B: Change From Baseline of Delandistrogene Moxeparvovec Dystrophin Expression Quantification by Immunofluorescence |  | Baseline, Day 90 | 
| Cohorts A and B: Change From Baseline of Delandistrogene Moxeparvovec Dystrophin Expression Quantification by Western Blot |  | Baseline, Day 90 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Duchenne, Dystrophin, Pediatric, Gene Therapy, DMD, Gene-Delivery, Gene Transfer Therapy