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

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ClinicalTrials.gov Identifier: NCT03375164
Recruitment Status : Completed
First Posted : December 15, 2017
Last Update Posted : August 1, 2023
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Study Description
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Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Genetic: delandistrogene moxeparvovec Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 4 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Systemic Gene Delivery Phase I/IIa Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MHCK7.Micro-dystrophin (microDys-IV-001)
Actual Study Start Date : January 4, 2018
Actual Primary Completion Date : April 25, 2023
Actual Study Completion Date : April 25, 2023

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Arms and Interventions
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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


Outcome Measures
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Primary Outcome Measures :
  1. Cohorts A and B: Number of Participants with Adverse Events (AEs) [ Time Frame: Up to 5 Years ]

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

Eligibility Criteria
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Ages Eligible for Study:   3 Months to 7 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03375164


Locations
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United States, Ohio
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Sponsors and Collaborators
Sarepta Therapeutics, Inc.
Investigators
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Study Director: Medical Director Sarepta Therapeutics, Inc.
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Sarepta Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03375164    
Other Study ID Numbers: SRP-9001-101
2021-000077-83 ( EudraCT Number )
First Posted: December 15, 2017    Key Record Dates
Last Update Posted: August 1, 2023
Last Verified: July 2023

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Sarepta Therapeutics, Inc.:
Duchenne
Gene Therapy
DMD
Dystrophin
 Pediatric
Gene-Delivery
Gene Transfer Therapy
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
 Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked