Back to resultsA Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
Primary Purpose
Duchenne Muscular Dystrophy
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
delandistrogene moxeparvovec
Sponsored by
About this trial
This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring Duchenne, Gene Therapy, DMD, Dystrophin, Pediatric, Gene-Delivery, Gene Transfer Therapy
Eligibility 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.
Sites / Locations
- Nationwide Children's Hospital
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
Cohort A: Delandistrogene Moxeparvovec
Cohort B: Delandistrogene Moxeparvovec
Arm Description
Participants will receive a Single IV infusion of delandistrogene moxeparvovec on Day 1.
Participants will receive a Single IV infusion of delandistrogene moxeparvovec on Day 1.
Outcomes
Primary Outcome Measures
Cohorts A and B: Number of Participants with Adverse Events (AEs)
Secondary Outcome Measures
Cohort A: Gross Motor Subtest Scaled (Bayley-III) Score
Cohorts A and B: The 100 Meter Timed Test (100m) Physical Therapy Assessment
Cohorts A and B: Change From Baseline of Delandistrogene Moxeparvovec Dystrophin Expression Quantification by Immunofluorescence
Cohorts A and B: Change From Baseline of Delandistrogene Moxeparvovec Dystrophin Expression Quantification by Western Blot
Full Information
NCT ID
NCT03375164
First Posted
December 4, 2017
Last Updated
July 28, 2023
Sponsor
Sarepta Therapeutics, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT03375164
Brief Title
A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
Official Title
Systemic Gene Delivery Phase I/IIa Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MHCK7.Micro-dystrophin (microDys-IV-001)
Study Type
Interventional
2. Study Status
Record Verification Date
July 2023
Overall Recruitment Status
Completed
Study Start Date
January 4, 2018 (Actual)
Primary Completion Date
April 25, 2023 (Actual)
Study Completion Date
April 25, 2023 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Sarepta Therapeutics, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
Keywords
Duchenne, Gene Therapy, DMD, Dystrophin, Pediatric, Gene-Delivery, Gene Transfer Therapy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
4 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Cohort A: Delandistrogene Moxeparvovec
Arm Type
Experimental
Arm Description
Participants will receive a Single IV infusion of delandistrogene moxeparvovec on Day 1.
Arm Title
Cohort B: Delandistrogene Moxeparvovec
Arm Type
Experimental
Arm Description
Participants will receive a Single IV infusion of delandistrogene moxeparvovec on Day 1.
Intervention Type
Genetic
Intervention Name(s)
delandistrogene moxeparvovec
Other Intervention Name(s)
SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS
Intervention Description
Single IV infusion of delandistrogene moxeparvovec.
Primary Outcome Measure Information:
Title
Cohorts A and B: Number of Participants with Adverse Events (AEs)
Time Frame
Up to 5 Years
Secondary Outcome Measure Information:
Title
Cohort A: Gross Motor Subtest Scaled (Bayley-III) Score
Time Frame
Day 30 up to 3 Years
Title
Cohorts A and B: The 100 Meter Timed Test (100m) Physical Therapy Assessment
Time Frame
Up to 5 Years
Title
Cohorts A and B: Change From Baseline of Delandistrogene Moxeparvovec Dystrophin Expression Quantification by Immunofluorescence
Time Frame
Baseline, Day 90
Title
Cohorts A and B: Change From Baseline of Delandistrogene Moxeparvovec Dystrophin Expression Quantification by Western Blot
Time Frame
Baseline, Day 90
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
3 Months
Maximum Age & Unit of Time
7 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Sarepta Therapeutics, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
32539076
Citation
Mendell JR, Sahenk Z, Lehman K, Nease C, Lowes LP, Miller NF, Iammarino MA, Alfano LN, Nicholl A, Al-Zaidy S, Lewis S, Church K, Shell R, Cripe LH, Potter RA, Griffin DA, Pozsgai E, Dugar A, Hogan M, Rodino-Klapac LR. Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial. JAMA Neurol. 2020 Sep 1;77(9):1122-1131. doi: 10.1001/jamaneurol.2020.1484.
Results Reference
derived
Learn more about this trial
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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