A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

13 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-11-29

Study Completion Date

2034-01-30

Brief Summary

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This open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks.

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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

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Delandistrogene Moxeparvovec

Participants will receive a single intravenous (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 ELEVIDYS RO7494222 delandistrogene moxeparvovec-rokl

Eligibility Criteria

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Inclusion Criteria

* Cohort A: \>=3 years of age to \<4 years of age
* Cohort B: \>=2 years of age to \<3 years of age
* Has a definitive diagnosis of DMD prior to screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test
* Able to cooperate with age-appropriate motor assessment testing
* A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive)

Exclusion Criteria

* Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression, within protocol-specified time limits
* Recombinant Adeno-Associated Virus Serotype rh74 (rAArh74) antibody titers are elevated, as per protocol-specified criteria
* Receiving regular oral corticosteroids as a treatment for DMD or planning to receive oral corticosteroids as a treatment for DMD within 1 year of baseline
* 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
* Medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the participant's ability to comply with the protocol required testing or procedures, or compromise the participant's well-being or safety, or clinical interpretability

Minimum Eligible Age

2 Years

Maximum Eligible Age

3 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No