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A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) (ENVOL)

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ClinicalTrials.gov Identifier: NCT06128564
Recruitment Status : Recruiting
First Posted : November 13, 2023
Last Update Posted : May 1, 2024
Sponsor:
Collaborator:
Sarepta Therapeutics, Inc.
Information provided by (Responsible Party):
Hoffmann-La Roche

Study Description
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Brief Summary:
This open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Genetic: delandistrogene moxeparvovec Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 21 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Two-Part, Open-Label Systemic Gene Delivery Study to Evaluate the Safety and Expression of RO7494222 (SRP-9001) in Subjects Under the Age of Four With Duchenne Muscular Dystrophy
Actual Study Start Date : November 29, 2023
Estimated Primary Completion Date : November 30, 2032
Estimated Study Completion Date : November 30, 2032

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Delandistrogene Moxeparvovec
Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1.
 Genetic: delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec
Other Names:
  • SRP-9001
  • ELEVIDYS
  • RO7494222
  • delandistrogene moxeparvovec-rokl


Outcome Measures
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Primary Outcome Measures :
  1. Percentage 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 260 ]

Secondary Outcome Measures :
  1. Change in Quantity of Delandistrogene Moxeparvovec Dystrophin as Measured by Western Blot [ Time Frame: Baseline, Week 12 ]

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

Inclusion Criteria:

  • Cohort A: >=3 years of age to <4 years of age
  • Cohort B: >=2 years of age to <3 years of age
  • Cohort C: >6 months to <2 years of age
  • Cohort D: <=6 months of age
  • Has a definitive diagnosis of DMD prior to screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test
  • Able to cooperate with age-appropriate motor assessment testing
  • A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive)

Exclusion Criteria:

  • Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression, within protocol-specified time limits
  • Recombinant Adeno-Associated Virus Serotype rh74 (rAArh74) antibody titers are elevated, as per protocol-specified criteria
  • Receiving regular oral corticosteroids as a treatment for DMD or planning to receive oral corticosteroids as a treatment for DMD within 1 year of baseline
  • 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
  • Medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the participant's ability to comply with the protocol required testing or procedures, or compromise the participant's well-being or safety, or clinical interpretability

Other inclusion or exclusion criteria could apply

Contacts and Locations
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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): NCT06128564


Contacts
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Contact: Reference Study ID Number: BN43881 https://forpatients.roche.com/ 888-662-6728 (U.S. and Canada) global-roche-genentech-trials@gene.com

Locations
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Germany
Universitätsklinikum Essen Recruiting
Essen, Germany, 45147
Spain
Hospital Sant Joan De Deu Recruiting
Esplugues De Llobregas, Barcelona, Spain, 08950
Sponsors and Collaborators
Hoffmann-La Roche
Sarepta Therapeutics, Inc.
Investigators
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Study Director: Clinical Trials Hoffmann-La Roche
More Information
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Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT06128564    
Other Study ID Numbers: BN43881
2022-000691-19 ( EudraCT Number )
First Posted: November 13, 2023    Key Record Dates
Last Update Posted: May 1, 2024
Last Verified: April 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/innovation/process/clinical-trials/data-sharing/).

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Keywords provided by Hoffmann-La Roche:
DMD
Duchenne
Dystrophin
Gene-Delivery
Pediatric
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