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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 : Recruiting
First Posted : November 12, 2020
Last Update Posted : December 24, 2020
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 individuals with Duchenne Muscular Dystrophy over 260 weeks. The primary endpoint is the change in quantity of micro-dystrophin protein expression from baseline to Week 12.

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 : 10 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 : May 31, 2021
Estimated Study Completion Date : December 2025


Arm Intervention/treatment
Experimental: SRP-9001
Patients 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 Measured by Western Blot [ Time Frame: Baseline up to Week 12 ]

Secondary Outcome Measures :
  1. Vector Shedding as Measured by Polymerase Chain Reaction in Urine, Saliva and Stool Samples Post-infusion [ Time Frame: Baseline up to Week 260 ]
  2. Immunogenicity of SRP-9001 Determined by Level of Antibody Titers to rAAVrh74 [ Time Frame: Screening up to Week 260 ]
  3. Incidence of Treatment Emergent Adverse Events (TEAEs), Serious AEs and Adverse Events of Special Interest [ Time Frame: Baseline up to Week 260 ]
  4. Number of Participants with Clinically Significant Abnormalities in Vital Signs, Physical Examinations, Laboratory Parameters, Electrocardiograms (ECG) and Echocardiograms (ECHO) Findings [ Time Frame: Baseline up to Week 260 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years to 7 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Is male at birth, ambulatory and from 4 to under 8 years of age at time of randomization.
  • Definitive diagnosis of Duchenne Muscular Dystrophy 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


Contacts
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Contact: Medical Information +1-800-690-2003 clinicaltrials@sarepta.com

Locations
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United States, California
University of California, Davis Recruiting
Sacramento, California, United States, 95616
Contact    916-734-3993    omsarwary@ucdavis.edu   
Principal Investigator: Craig McDonald, MD         
United States, Missouri
Washington University in St. Louis Recruiting
Saint Louis, Missouri, United States, 21205
Contact: Alyssa Sonsoucie    314-362-1566    sonsouciea@wustl.edu   
Principal Investigator: Craig Zaidman, MD         
United States, Ohio
Nationwide Children's Hospital Not yet recruiting
Columbus, Ohio, United States, 43205
Contact: Jerry Mendell         
Principal Investigator: Jerry Mendell         
United States, Virginia
Children's Hospital of The King's Daughters Recruiting
Norfolk, Virginia, United States, 23507
Contact: Cara Headrick    757-668-6417    Cara.Headrick@CHKD.ORG   
Principal Investigator: Crystal Proud, MD         
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: December 24, 2020
Last Verified: December 2020

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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