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A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9001 in Participants With 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: NCT03375164
Recruitment Status : Active, not recruiting
First Posted : December 15, 2017
Last Update Posted : February 25, 2022
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Brief Summary:
This is an open-label single-dose gene transfer therapy study evaluating the safety of SRP-9001 intravenous (IV) administration in boys with DMD. This study will consist of 2 Cohorts. Cohort A will include participants ages 3 months to 3 years, and Cohort B will include participants ages 4 to 7 years old. All participants in the study will receive IV SRP-9001.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Genetic: SRP-9001 Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 4 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Systemic Gene Delivery Phase I/IIa Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MHCK7.Micro-dystrophin (microDys-IV-001)
Actual Study Start Date : January 4, 2018
Estimated Primary Completion Date : April 30, 2023
Estimated Study Completion Date : April 30, 2023


Arm Intervention/treatment
Experimental: Cohort A: SRP-9001
Participants will receive a single IV infusion of SRP-9001 on Day 1.
Genetic: SRP-9001
Single IV infusion of SRP-9001.

Experimental: Cohort B: SRP-9001
Participants will receive a single IV infusion of SRP-9001 on Day 1.
Genetic: SRP-9001
Single IV infusion of SRP-9001.




Primary Outcome Measures :
  1. Cohorts A and B: Number of Participants with Adverse Events (AEs) [ Time Frame: Up to 5 Years ]

Secondary Outcome Measures :
  1. Cohort A: Gross Motor Subtest Scaled (Bayley-III) Score [ Time Frame: Day 30 up to 3 Years ]
  2. Cohorts A and B: The 100 Meter Timed Test (100m) Physical Therapy Assessment [ Time Frame: Up to 5 Years ]
  3. Cohorts A and B: Change From Baseline of Micro-dystrophin Gene Expression Quantification by Immunofluorescence [ Time Frame: Baseline, Day 90 ]
  4. Cohorts A and B: Change From Baseline of Micro-dystrophin Gene Expression Quantification by Western Blot [ Time Frame: Baseline, Day 90 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   3 Months to 7 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Cohort A participants: 3 months to 3 years of age, inclusive
  • Cohort B participants: 4 to 7 years of age, inclusive
  • Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
  • Ability to cooperate with motor assessment testing.
  • Cohort A participants: No previous treatment with corticosteroids.
  • Cohort B participants: Stable dose equivalent of oral corticosteroids for at least 12 weeks prior to screening and the dose is expected to remain constant (except for potential modifications to accommodate changes in weight) throughout the first year of the study.

Exclusion Criteria:

  • Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol specified time limits.
  • Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
  • Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer.

Other inclusion or exclusion criteria could 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): NCT03375164


Locations
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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.
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Sarepta Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03375164    
Other Study ID Numbers: SRP-9001-101
First Posted: December 15, 2017    Key Record Dates
Last Update Posted: February 25, 2022
Last Verified: February 2022

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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
Gene Therapy
DMD
Dystrophin
Pediatric
Gene-Delivery
Gene Transfer Therapy
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