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History of Changes for Study: NCT05096221
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Participants With Duchenne Muscular Dystrophy (DMD) (EMBARK)
Latest version (submitted November 3, 2023) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 October 14, 2021 None (earliest Version on record)
2 November 3, 2021 Study Status and Contacts/Locations
3 December 14, 2021 Study Status and Contacts/Locations
4 February 11, 2022 Study Status and Contacts/Locations
5 March 10, 2022 Contacts/Locations and Study Status
6 April 8, 2022 Study Status and Contacts/Locations
7 May 13, 2022 Contacts/Locations and Study Status
8 June 11, 2022 Contacts/Locations, Study Status, Eligibility, Arms and Interventions and Study Identification
9 July 19, 2022 Contacts/Locations, Study Status and References
10 August 17, 2022 Study Status and Contacts/Locations
11 September 20, 2022 Recruitment Status, Study Status, Contacts/Locations, Outcome Measures and Study Design
12 November 2, 2022 Outcome Measures, Study Design, Study Status, References and Study Identification
13 November 8, 2022 Study Status and Outcome Measures
14 November 10, 2022 Outcome Measures and Study Status
15 July 6, 2023 Arms and Interventions, Study Status, Outcome Measures and Study Identification
16 July 28, 2023 Outcome Measures and Study Status
17 September 6, 2023 Study Status and Contacts/Locations
18 November 2, 2023 Study Status
19 November 3, 2023 Outcome Measures and Study Status
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Study NCT05096221
Submitted Date:  October 14, 2021 (v1)
Open or close this module Study Identification
Unique Protocol ID: SRP-9001-301
Brief Title: A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Participants With Duchenne Muscular Dystrophy (DMD) (EMBARK)
Official Title: A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Patients With Duchenne Muscular Dystrophy (EMBARK)
Secondary IDs: 2019-003374-91 [EudraCT Number]
Open or close this module Study Status
Record Verification: October 2021
Overall Status: Recruiting
Study Start: October 12, 2021
Primary Completion: October 31, 2023 [Anticipated]
Study Completion: November 30, 2024 [Anticipated]
First Submitted: October 14, 2021
First Submitted that
Met QC Criteria:		 October 14, 2021
First Posted: October 27, 2021 [Actual]
Last Update Submitted that
Met QC Criteria:		 October 14, 2021
Last Update Posted: October 27, 2021 [Actual]
Open or close this module Sponsor/Collaborators
Sponsor: Sarepta Therapeutics, Inc.
Responsible Party: Sponsor
Collaborators: Hoffmann-La Roche
Open or close this module Oversight
U.S. FDA-regulated Drug: Yes
U.S. FDA-regulated Device: No
Data Monitoring: Yes
Open or close this module Study Description
Brief Summary: The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene transfer therapy at the beginning of the second year.
Detailed Description:
Open or close this module Conditions
Conditions: Duchenne Muscular Dystrophy
Keywords: Muscular Dystrophies
Duchenne Muscular Dystrophy
DMD
North Star Ambulatory Assessment (NSAA)
Ambulatory
Pediatric
Gene-Delivery
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Treatment
Study Phase: Phase 3
Interventional Study Model: Parallel Assignment
Number of Arms: 2
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Allocation: Randomized
Enrollment: 120 [Anticipated]
Open or close this module Arms and Interventions
Arms Assigned Interventions
Experimental: SRP-9001 followed by Placebo
Participants will receive single intravenous (IV) infusion of SRP-9001 on Day 1. Then, participants will receive a single IV infusion of matching placebo at Year 2.
 Genetic: SRP-9001
Single IV infusion of SRP-9001.
Genetic: Placebo
Single IV infusion of matching placebo.
Placebo Comparator: Placebo followed by SRP-9001
Participants will receive matching placebo IV infusion on Day 1. Then, participants will have the opportunity to receive a single IV infusion of SRP-9001 at Year 2.
 Genetic: SRP-9001
Single IV infusion of SRP-9001.
Genetic: Placebo
Single IV infusion of matching placebo.
Open or close this module Outcome Measures
Primary Outcome Measures:
1. Part 1: Change From Baseline in NSAA Total Score at Week 52
[ Time Frame: Baseline, Week 52 ]
Secondary Outcome Measures:
1. Part 1: Quantity of Micro-Dystrophin Protein Expression at Week 12 as Measured by Western Blot, in a Subset of Participants
[ Time Frame: Week 12 ]
2. Part 1: Change From Baseline in Time to Rise From the Floor, Time to Complete 100 and 10 meter Walk/Run, and the Timed Stair Ascend 4 Steps Test at Week 52
[ Time Frame: Baseline, Week 52 ]
3. Part 1: Change From Baseline in Stride Velocity 95th Centile (SV95C) Measured by a Wearable Device
[ Time Frame: Baseline up to Week 52 ]
4. Part 1: Change from Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score per Domain at Week 52
[ Time Frame: Baseline, Week 52 ]

PROMIS is a family of instruments developed and validated to assess health-related quality of life. Parents will be asked "Taking into account all aspects of your child's observable symptoms, physical ability, ability to perform daily activities and overall health, how would you rate the change in clinical status for your child since the study start? using the following rating scale 1=very much improved, 2=much improved, 3=minimally improved, 4=no change, 5=minimally worse, 6=much worse, and 7=very much worse."
5. Part 1: Number of Skills Gained or Improved at Week 52 as Measured by the NSAA
[ Time Frame: Baseline up to Week 52 ]
6. Number of Participants with a Treatment Emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)
[ Time Frame: Baseline up to Week 52 ]
Open or close this module Eligibility
Minimum Age: 4 Years
Maximum Age: 7 Years
Sex: Male
Gender Based:
Accepts Healthy Volunteers: No
Criteria:

Inclusion Criteria:

  • Is ambulatory and from 4 to under 8 years of age at time of randomization.
  • Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
  • Ability to cooperate with motor assessment testing.
  • Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).
  • rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.

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.
Open or close this module Contacts/Locations
Central Contact Person: Medical Information
Telephone: +1-800-690-2003
Email: SareptAlly@sarepta.com
Study Officials: Medical Director
Study Director
Sarepta Therapeutics, Inc.
Locations: United States, Missouri
Washington University of St. Louis
[Recruiting]
Saint Louis, Missouri, United States, 63110
Contact:Contact: 314-362-6981 NeuromusclePediatricResearch@wustl.edu
Contact:Principal Investigator: Craig Zaidman
Open or close this module IPDSharing
Plan to Share IPD:
Open or close this module References
Citations:
Links:
Available IPD/Information:
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U.S. National Library of Medicine | U.S. National Institutes of Health | U.S. Department of Health & Human Services