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

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

4 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-01-04

Study Completion Date

2023-04-25

Brief Summary

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This study was an open-label single-dose gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec intravenous (IV) administration in boys with DMD. This study was originally designed to consist of 12 patients across 2 Cohorts. Cohort A would have included participants ages 3 months to 3 years, and Cohort B included participants ages 4 to 7 years old. No participants were enrolled in Cohort A.

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Detailed Description

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Conditions

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Duchenne Muscular Dystrophy

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort A: Delandistrogene Moxeparvovec

Participants will receive a Single IV infusion of delandistrogene moxeparvovec on Day 1.

Group Type EXPERIMENTAL

delandistrogene moxeparvovec

Intervention Type GENETIC

Single IV infusion of delandistrogene moxeparvovec.

Cohort B: Delandistrogene Moxeparvovec

Participants will receive a Single IV infusion of delandistrogene moxeparvovec on Day 1.

Group Type EXPERIMENTAL

delandistrogene moxeparvovec

Intervention Type GENETIC

Single IV infusion of delandistrogene moxeparvovec.

Interventions

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delandistrogene moxeparvovec

Single IV infusion of delandistrogene moxeparvovec.

Intervention Type GENETIC

Other Intervention Names

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SRP-9001 delandistrogene moxeparvovec-rokl ELEVIDYS

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.

Minimum Eligible Age

3 Months

Maximum Eligible Age

7 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No