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A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 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. Identifier: NCT03769116
Recruitment Status : Active, not recruiting
First Posted : December 7, 2018
Last Update Posted : March 30, 2020
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

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 two parts: a 48-week randomized, double-blinded, placebo-controlled period (Part 1), and a 96-week, double-blinded extension period (Part 2).

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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 41 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A 48-Week, Randomized, Double-Blind, Placebo-Controlled, Systemic, Gene-Delivery Clinical Trial for Duchenne Muscular Dystrophy Using SRP-9001 With a 96-Week Extension
Actual Study Start Date : December 22, 2018
Estimated Primary Completion Date : October 10, 2022
Estimated Study Completion Date : October 10, 2022

Arm Intervention/treatment
Experimental: SRP-9001
Single IV infusion of SRP-9001.
Genetic: SRP-9001
Single IV infusion of SRP-9001

Placebo Comparator: Placebo
Placebo IV infusion 10 mL/kg.
Genetic: Placebo
Single IV infusion of placebo

Primary Outcome Measures :
  1. Incidence of Serious Adverse Events (SAEs) [ Time Frame: Up to Week 144 ]
  2. Incidence of Treatment Emergent Adverse Events (TEAEs) [ Time Frame: Up to Week 144 ]
  3. Change From Baseline in Quantity of Microdystrophin Protein Expression Measured by Western Blot [ Time Frame: Baseline up to Week 12 ]

Secondary Outcome Measures :
  1. Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score [ Time Frame: Baseline up to Week 48 ]
  2. Change From Baseline in Time to Rise From the Floor and Ascend 4 Steps [ Time Frame: Baseline up to Week 48 ]
  3. Change From Baseline in Time of 10 Meter and 100 Meter Timed Test [ Time Frame: Baseline up to Week 48 ]

Information from the National Library of Medicine

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.

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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.

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 months.
  • Exposure to an investigational or commercial gene therapy product.
  • Abnormal liver or renal function by protocol-specified criteria

Other inclusion/exclusion criteria apply.

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 identifier (NCT number): NCT03769116

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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.
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Study Director: Medical Director Sarepta Therapeutics, Inc.
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Responsible Party: Sarepta Therapeutics, Inc. Identifier: NCT03769116    
Other Study ID Numbers: SRP-9001-102
First Posted: December 7, 2018    Key Record Dates
Last Update Posted: March 30, 2020
Last Verified: March 2020

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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 Muscular Dystrophy
North Star Ambulatory Assessment (NSAA)
Percent Dystrophin Positive Fibers (PDPF)
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