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

July 28, 2023 updated by: Sarepta Therapeutics, Inc.

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

4

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Ohio
      • Columbus, Ohio, United States, 43205
        • Nationwide Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

3 months to 7 years (Child)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort A: Delandistrogene Moxeparvovec
Participants will receive a Single IV infusion of delandistrogene moxeparvovec on Day 1.
Genetic: delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec.
Other Names:
  • SRP-9001
  • delandistrogene moxeparvovec-rokl
  • ELEVIDYS
Experimental: Cohort B: Delandistrogene Moxeparvovec
Participants will receive a Single IV infusion of delandistrogene moxeparvovec on Day 1.
Genetic: delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec.
Other Names:
  • SRP-9001
  • delandistrogene moxeparvovec-rokl
  • ELEVIDYS

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sarepta Therapeutics, Inc.

Investigators

  • Study Director: Medical Director, Sarepta Therapeutics, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 4, 2018

Primary Completion (Actual)

April 25, 2023

Study Completion (Actual)

April 25, 2023

Study Registration Dates

First Submitted

December 4, 2017

First Submitted That Met QC Criteria

December 14, 2017

First Posted (Actual)

December 15, 2017

Study Record Updates

Last Update Posted (Actual)

August 1, 2023

Last Update Submitted That Met QC Criteria

July 28, 2023

Last Verified

July 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SRP-9001-101
  • 2021-000077-83 (EudraCT Number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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