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A Gene Delivery Study to Evaluate the Safety of and Expression From SRP-9001 in Duchenne Muscular Dystrophy (DMD) (ENDEAVOR)

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ClinicalTrials.gov Identifier: NCT04626674
Recruitment Status : Enrolling by invitation
First Posted : November 12, 2020
Last Update Posted : June 22, 2021
Sponsor:
Collaborator:
Roche Pharma AG
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Brief Summary:
This is an open-label gene delivery study evaluating the safety of and expression from SRP-9001 in participants with DMD over 260 weeks.

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 32 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label, Systemic Gene Delivery Study Using Commercial Process Material to Evaluate the Safety of and Expression From SRP-9001 in Subjects With Duchenne Muscular Dystrophy (ENDEAVOR)
Actual Study Start Date : November 23, 2020
Estimated Primary Completion Date : December 31, 2021
Estimated Study Completion Date : July 30, 2026


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




Primary Outcome Measures :
  1. Part 1: Change From Baseline in Quantity of Micro-Dystrophin Protein Expression at Week 12, as Measured by Western Blot [ Time Frame: Baseline, Week 12 ]

Secondary Outcome Measures :
  1. Vector Shedding, Measured in Urine, Saliva, and Stool Samples Post-Infusion [ Time Frame: Day 1 up to Week 52 ]
  2. Level of Antibody Titers to Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74) [ Time Frame: Day 2 up to Week 260 ]
  3. Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events (AEs) of Special Interest [ Time Frame: Baseline up to Week 260 ]
  4. Change From Baseline in Quantity of Micro-Dystrophin Protein Expression at Week 12, as Measured by Immunofluorescence (IF) Fiber Intensity [ Time Frame: Baseline, Week 12 ]
  5. Change From Baseline in Quantity of Micro-Dystrophin Expression at Week 12, as Measured by IF Percent Dystrophin Positive Fibers (PDPF) [ Time Frame: Baseline, Week 12 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Cohort 1 only: Is ambulatory, and ≥4 to <8 years of age at the time of Screening.
  • Cohort 2 only: Is ambulatory, and ≥8 to <18 years of age at the time of Screening.
  • Cohort 3 only: Non-ambulatory per protocol specified criteria.
  • Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
  • Ability to cooperate with motor assessment testing.
  • Stable dose equivalent of oral corticosteroids for at least 12 weeks.
  • rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.

Exclusion Criteria:

  • Has a concomitant illness, autoimmune disease, chronic drug treatment, and/or cognitive delay/impairment that in the opinion of the Investigator creates unnecessary risks for gene transfer.
  • 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.

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): NCT04626674


Locations
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United States, California
Stanford University
Palo Alto, California, United States, 94304
University of California, Davis
Sacramento, California, United States, 95616
United States, Missouri
Washington University in St. Louis
Saint Louis, Missouri, United States, 21205
United States, Ohio
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
United States, Virginia
Children's Hospital of The King's Daughters
Norfolk, Virginia, United States, 23507
Sponsors and Collaborators
Sarepta Therapeutics, Inc.
Roche Pharma AG
Investigators
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Study Director: Medical Director Sarepta Therapeutics, Inc.
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Responsible Party: Sarepta Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT04626674    
Other Study ID Numbers: SRP-9001-103
First Posted: November 12, 2020    Key Record Dates
Last Update Posted: June 22, 2021
Last Verified: June 2021

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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
Micro-dystrophin
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