A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 (Delandistrogene Moxeparvovec) for Duchenne Muscular Dystrophy (DMD)

• ClinicalTrials.gov Identifier: NCT03769116
• Recruitment Status: Active, not recruiting
• First Posted: December 7, 2018
• Last Update Posted: July 1, 2022
• Sponsor: Sarepta Therapeutics, Inc.
• Information provided by (Responsible Party): Sarepta Therapeutics, Inc.

Study Description

Brief Summary:

The purpose of this study is to evaluate the safety and efficacy of exogenous gene transfer in DMD patients by measuring biological and clinical endpoints in three parts: two 48-week randomized, double-blinded, placebo-controlled periods (Part 1 and Part 2), and an open-label follow-up period (Part 3). Patients who are randomized to placebo in Part 1 will have the opportunity for treatment with SRP-9001 (delandistrogene moxeparvovec) in Part 2.

Condition or disease	Intervention/treatment	Phase
Muscular Dystrophy, Duchenne	Genetic: SRP-9001; Genetic: Placebo	Phase 2

Study Design

• 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

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures :

1. Change From Baseline in Quantity of Micro-dystrophin Protein Expression as Measured by Western Blot [ Time Frame: Baseline up to Week 12 (Part 1) ]
2. Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score [ Time Frame: Baseline up to Week 48 (Part 1) ]

Secondary Outcome Measures :

1. Change From Baseline in Time to Rise From the Floor [ Time Frame: Baseline up to Week 48 (Part 1) ]
2. Change From Baseline in Time to Ascend 4 Steps [ Time Frame: Baseline up to Week 48 (Part 1) ]
3. Change From Baseline in Time of 10 Meter Timed Test [ Time Frame: Baseline up to Week 48 (Part 1) ]
4. Change From Baseline in Time of 100 Meter Timed Test [ Time Frame: Baseline up to Week 48 (Part 1) ]
5. Change From Baseline in Quantity of Micro-dystrophin Expression Measured by Immunofluorescence (IF) Fiber Intensity [ Time Frame: Baseline up to Week 12 (Part 1) ]
6. 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) ]

Other Outcome Measures:

1. Incidence of Serious Adverse Events (SAEs) and Treatment Emergent Adverse Events (TEAEs) [ Time Frame: Up to Week 260 ]

Eligibility Criteria

• Ages Eligible for Study: 4 Years to 7 Years (Child)
• Sexes Eligible for Study: Male
• Accepts Healthy Volunteers: No

Criteria

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.

Contacts and Locations

Locations

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

Sponsors and Collaborators

Sarepta Therapeutics, Inc.

Investigators

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

More Information

• Responsible Party: Sarepta Therapeutics, Inc.
• ClinicalTrials.gov Identifier: NCT03769116
• Other Study ID Numbers: SRP-9001-102
• First Posted: December 7, 2018
• 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

Keywords provided by Sarepta Therapeutics, Inc.:

Duchenne Muscular Dystrophy; Gene-Delivery; DMD; Ambulatory; Pediatric; North Star Ambulatory Assessment (NSAA); Percent Dystrophin Positive Fibers (PDPF)

Additional relevant MeSH terms:

Muscular Dystrophies; Muscular Dystrophy, Duchenne; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked