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Trial record 1 of 1 for:    9001-301
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A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 (Delandistrogene Moxeparvovec) in Participants With Duchenne Muscular Dystrophy (DMD) (EMBARK)

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: NCT05096221
Recruitment Status : Active, not recruiting
First Posted : October 27, 2021
Last Update Posted : September 22, 2022
Sponsor:
Collaborator:
Hoffmann-La Roche
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

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.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Genetic: SRP-9001 Genetic: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 125 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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)
Actual Study Start Date : October 27, 2021
Estimated Primary Completion Date : October 31, 2023
Estimated Study Completion Date : November 30, 2024


Arm Intervention/treatment
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.
Other Name: delandistrogene moxeparvovec

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.
Other Name: delandistrogene moxeparvovec

Genetic: Placebo
Single IV infusion of matching placebo.




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 104 ]


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 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.
  • A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive), with the exception of mutation fully contained within exon 45.

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): NCT05096221


Locations
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Sponsors and Collaborators
Sarepta Therapeutics, Inc.
Hoffmann-La Roche
Investigators
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Study Director: Medical Director Sarepta Therapeutics, Inc.
Additional Information:
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Responsible Party: Sarepta Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT05096221    
Other Study ID Numbers: SRP-9001-301
2019-003374-91 ( EudraCT Number )
First Posted: October 27, 2021    Key Record Dates
Last Update Posted: September 22, 2022
Last Verified: September 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.:
Muscular Dystrophies
Duchenne Muscular Dystrophy
DMD
North Star Ambulatory Assessment (NSAA)
Ambulatory
Pediatric
Gene-Delivery
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