Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
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 : March 30, 2020
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Study Description
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
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

Study Design
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
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

Resource links provided by the National Library of Medicine


Arms and Interventions
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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


Outcome Measures
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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 ]

Eligibility Criteria
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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.


Layout table for eligibility information
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 months.
  • 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
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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
Layout table for location information
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
Layout table for investigator information
Study Director: Medical Director Sarepta Therapeutics, Inc.
More Information
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Layout table for additonal information
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: March 30, 2020
Last Verified: March 2020

Layout table for additional information
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:
Layout table for 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