A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 (Delandistrogene Moxeparvovec) for Duchenne Muscular Dystrophy (DMD)
![]() |
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT03769116 |
Recruitment Status :
Active, not recruiting
First Posted : December 7, 2018
Last Update Posted : July 1, 2022
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Muscular Dystrophy, Duchenne | Genetic: SRP-9001 Genetic: Placebo | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 41 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Intervention Model Description: | Parallel up to the measurement of the primary outcome at Week 48. At the beginning of Part 2, patients who were originally assigned to placebo will have the opportunity to receive SRP-9001. All patients will be followed for 5 years following treatment with SRP-9001. |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial for Duchenne Muscular Dystrophy Using SRP-9001 |
Actual Study Start Date : | December 22, 2018 |
Actual Primary Completion Date : | December 8, 2020 |
Estimated Study Completion Date : | April 2, 2026 |

Arm | Intervention/treatment |
---|---|
Experimental: SRP-9001 in Part 1 Followed by Placebo in Part 2
Patient will receive SRP-9001 at Part 1 followed by matching Placebo at Part 2 followed by an open-label extension at Part 3.
|
Genetic: SRP-9001
Single IV infusion of SRP-9001
Other Name: delandistrogene moxeparvovec Genetic: Placebo Single IV infusion of matching placebo |
Experimental: Placebo in Part 1 Followed by SRP-9001 in Part 2
Patient will receive matching Placebo at Part 1 followed by SRP-9001 at Part 2 followed by an open-label extension at Part 3.
|
Genetic: SRP-9001
Single IV infusion of SRP-9001
Other Name: delandistrogene moxeparvovec Genetic: Placebo Single IV infusion of matching placebo |
- Change From Baseline in Quantity of Micro-dystrophin Protein Expression as Measured by Western Blot [ Time Frame: Baseline up to Week 12 (Part 1) ]
- Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score [ Time Frame: Baseline up to Week 48 (Part 1) ]
- Change From Baseline in Time to Rise From the Floor [ Time Frame: Baseline up to Week 48 (Part 1) ]
- Change From Baseline in Time to Ascend 4 Steps [ Time Frame: Baseline up to Week 48 (Part 1) ]
- Change From Baseline in Time of 10 Meter Timed Test [ Time Frame: Baseline up to Week 48 (Part 1) ]
- Change From Baseline in Time of 100 Meter Timed Test [ Time Frame: Baseline up to Week 48 (Part 1) ]
- Change From Baseline in Quantity of Micro-dystrophin Expression Measured by Immunofluorescence (IF) Fiber Intensity [ Time Frame: Baseline up to Week 12 (Part 1) ]
- Change From Baseline in Quantity of Micro-dystrophin Expression Measured by IF Percent Dystrophin Positive Fibers (PDPF) [ Time Frame: Baseline up to Week 12 (Part 1) ]
- Incidence of Serious Adverse Events (SAEs) and Treatment Emergent Adverse Events (TEAEs) [ Time Frame: Up to Week 260 ]

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 4 Years to 7 Years (Child) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Established clinical diagnosis of DMD and documented dystrophin gene mutation of DMD phenotype.
- Indication of symptomatic muscular dystrophy by protocol-specified criteria.
- Ability to cooperate with motor assessment testing.
- Stable dose equivalent of oral corticosteroids for at least 12 weeks.
- A frameshift mutation contained between exons 18 and 58 (inclusive).
Exclusion Criteria:
- Impaired cardiovascular function on ECHO.
- Prior or ongoing medical condition on physical examination, ECG, or laboratory findings that could adversely affect subject safety, compromise completion of follow-up, or impair assessment of study results.
- Exposure to another investigational drug or exon skipping medication within 6 months of screening.
- Exposure to an investigational or commercial gene therapy product.
- Abnormal liver or renal function by protocol-specified criteria
Other inclusion/exclusion criteria apply.

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): NCT03769116
United States, California | |
David Geffen School of Medicine at UCLA | |
Los Angeles, California, United States, 90095 | |
United States, Ohio | |
Nationwide Children's Hospital | |
Columbus, Ohio, United States, 43205 |
Study Director: | Medical Director | Sarepta Therapeutics, Inc. |
Responsible Party: | Sarepta Therapeutics, Inc. |
ClinicalTrials.gov Identifier: | NCT03769116 |
Other Study ID Numbers: |
SRP-9001-102 |
First Posted: | December 7, 2018 Key Record Dates |
Last Update Posted: | July 1, 2022 |
Last Verified: | June 2022 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Duchenne Muscular Dystrophy Gene-Delivery DMD Ambulatory |
Pediatric North Star Ambulatory Assessment (NSAA) Percent Dystrophin Positive Fibers (PDPF) |
Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked |