Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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 : January 22, 2021
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Tracking Information
First Submitted Date  ICMJE December 6, 2018
First Posted Date  ICMJE December 7, 2018
Last Update Posted Date January 22, 2021
Actual Study Start Date  ICMJE December 22, 2018
Actual Primary Completion Date December 8, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 30, 2020)
  • 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) ]
Original Primary Outcome Measures  ICMJE
 (submitted: December 6, 2018)
  • Incidence of Serious Adverse Events (SAEs) [ Time Frame: Up to 144 weeks ]
  • Incidence of Treatment Emergent Adverse Events (TEAEs) [ Time Frame: Up to 144 weeks ]
  • Incidence of Adverse Events of Special Interest (AESIs) [ Time Frame: Up to 144 weeks ]
  • Change from Baseline in Quantity of Microdystrophin Protein Expression [ Time Frame: Baseline, Week 12 ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: September 30, 2020)
  • 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) ]
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures
 (submitted: September 30, 2020)
Incidence of Serious Adverse Events (SAEs) and Treatment Emergent Adverse Events (TEAEs) [ Time Frame: Up to Week 260 ]
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD)
Official Title  ICMJE A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial for Duchenne Muscular Dystrophy Using SRP-9001
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE 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
Condition  ICMJE Muscular Dystrophy, Duchenne
Intervention  ICMJE
  • Genetic: SRP-9001
    Single IV infusion of SRP-9001
  • Genetic: Placebo
    Single IV infusion of matching placebo
Study Arms  ICMJE
  • 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.
    Interventions:
    • Genetic: SRP-9001
    • Genetic: 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.
    Interventions:
    • Genetic: SRP-9001
    • Genetic: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: January 23, 2020)
41
Original Estimated Enrollment  ICMJE
 (submitted: December 6, 2018)
24
Estimated Study Completion Date  ICMJE April 2, 2026
Actual Primary Completion Date December 8, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.

Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 4 Years to 7 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03769116
Other Study ID Numbers  ICMJE SRP-9001-102
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Sarepta Therapeutics, Inc.
Study Sponsor  ICMJE Sarepta Therapeutics, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Medical Director Sarepta Therapeutics, Inc.
PRS Account Sarepta Therapeutics, Inc.
Verification Date January 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP