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

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ClinicalTrials.gov Identifier: NCT03769116
Recruitment Status : Recruiting
First Posted : December 7, 2018
Last Update Posted : January 15, 2019
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics

Tracking Information
First Submitted Date  ICMJE December 6, 2018
First Posted Date  ICMJE December 7, 2018
Last Update Posted Date January 15, 2019
Actual Study Start Date  ICMJE December 22, 2018
Estimated Primary Completion Date December 31, 2021   (Final data collection date for primary outcome measure)
Current 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 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03769116 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
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 48-Week, Randomized, Double-Blind, Placebo-Controlled, Systemic, Gene-Delivery Clinical Trial for Duchenne Muscular Dystrophy Using SRP-9001 With a 96-Week Extension
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).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Muscular Dystrophy, Duchenne
Intervention  ICMJE
  • Drug: SRP-9001
    Single IV infusion of SRP-9001
  • Drug: Placebo
    Single IV infusion of placebo
Study Arms  ICMJE
  • Experimental: SRP-9001
    Single IV infusion of SRP-9001.
    Intervention: Drug: SRP-9001
  • Placebo Comparator: Placebo
    Placebo IV infusion 10 mL/kg.
    Intervention: Drug: 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 Recruiting
Estimated Enrollment  ICMJE
 (submitted: December 6, 2018)
24
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 31, 2021
Estimated Primary Completion Date December 31, 2021   (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 months.
  • 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
Gender Based Eligibility: Yes
Gender Eligibility Description: Inheritance of DMD follows an X-linked recessive pattern. Birth prevalence has been estimated at 1 in 3500 to 5000 live male births.
Ages  ICMJE 4 Years to 7 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Medical Information +1-888-727-3782 clinicaltrials@sarepta.com
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
Study Sponsor  ICMJE Sarepta Therapeutics
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Medical Director Sarepta Therapeutics, Inc.
PRS Account Sarepta Therapeutics
Verification Date January 2019

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