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Trial record 1 of 3 for:    SRP-9001
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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.
 
ClinicalTrials.gov Identifier: NCT03769116
Recruitment Status : Active, not recruiting
First Posted : December 7, 2018
Last Update Posted : October 5, 2020
Sponsor:
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 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 in 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
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
Estimated Primary Completion Date : December 31, 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

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

Genetic: Placebo
Single IV infusion of matching placebo




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 ]


Information from the National Library of Medicine

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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.

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.


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): NCT03769116


Locations
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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.
Investigators
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Study Director: Medical Director Sarepta Therapeutics, Inc.
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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: October 5, 2020
Last Verified: September 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
Gene-Delivery
DMD
Ambulatory
Pediatric
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