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

Sarepta Therapeutics

Status and phase

Completed

Phase 2

Phase 1

Conditions

Duchenne Muscular Dystrophy

Treatments

Genetic: delandistrogene moxeparvovec

Study type

Interventional

Funder types

Industry

Identifiers

NCT03375164

SRP-9001-101

2021-000077-83 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment

4 patients

Sex

Male

Ages

3 months to 7 years old

Volunteers

No Healthy Volunteers

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.