A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy Clinical Trial

— ENVISION  

Official title:

A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP- 9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05881408
• Other study ID #: SRP-9001-303
• Secondary ID: 2020-002372-13
• Status: Recruiting
• Phase: Phase 3
• Start date: May 31, 2023
• Est. completion date: January 31, 2027

Study information

• Verified date: May 2024
• Source: Sarepta Therapeutics, Inc.
• Contact: Sarepta Therapeutics Inc., For Clinical Trial Information, Selec
• Phone: 1-888-SAREPTA (1-888-727-3782)
• Email: SareptAlly[@]sarepta.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 148
• Est. completion date: January 31, 2027
• Est. primary completion date: January 31, 2026
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
• Cohort 1 only: Non-ambulatory per protocol specified criteria.
• Cohort 2 only: Ambulatory per protocol specified criteria and =8 to <18 years of age at the time of Screening.
• 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).
• Recombinant Adeno-Associated Virus Serotype rh74 (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).

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.

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Genetic:
• delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec
• placebo
Single IV infusion of matching placebo

Locations

Country	Name	City	State
Belgium	Universitair Ziekenhuis Gent	Gent	Oost-Vlaanderen
Italy	U.O.S.D Centro Traslazionale di Miologia e Patologie Neurodegenerative, Istituto G. Gaslini, Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico	Genova
Italy	UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico	Milano
Italy	UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gemelli IRCCS	Roma
Japan	National Center of Neurology and Psychiatry	Kodaira	Tokyo
Japan	Tokyo Women's Medical University Hospital	Shinjuku-ku	Tokyo
Japan	National Hospital Organization Osaka Toneyama Medical Center	Toyonaka-shi	Osaka
Spain	Hospital Sant Joan de Deu	Esplugues de LLobregat	Barcelona
Spain	Hospital Universitari Politecnic La Fe	Valencia
United Kingdom	Great Ormond Street Hospital for Children Foundation Trust	London	Greater London
United Kingdom	Institute of Translational and Clinical Research	Newcastle Upon Tyne
United States	The Johns Hopkins Hospital, Charlotte R. Bloomberg Children's Center, Pediatric Clinical Research Unit	Baltimore	Maryland
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	Lenox Baker Children's Hospital (Duke University)	Durham	North Carolina
United States	University of Florida, UF Health Center for Pediatric Neuromuscular and Rare Diseases	Gainesville	Florida
United States	University of Iowa Hospitals and Clinics, Dept of Pediatrics	Iowa City	Iowa
United States	Arkansas Children's Hospital	Little Rock	Arkansas
United States	Children's Hospital of the King's Daughters	Norfolk	Virginia
United States	Lucile Packard Children's Hospital Stanford	Palo Alto	California
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	University of Rochester, Department of Neurology	Rochester	New York
United States	University of California at Davis Medical Center	Sacramento	California
United States	Washington University of St. Louis, St. Louis Children's Hospital	Saint Louis	Missouri
United States	Rady Children's Hospital-San Diego	San Diego	California

Sponsors (2)

Lead Sponsor	Collaborator
Sarepta Therapeutics, Inc.	Hoffmann-La Roche

Countries where clinical trial is conducted

United States,  Belgium,  Italy,  Japan,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72		Baseline, Week 72
Secondary	Part 1: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 72		Baseline, Week 72
Secondary	Part 1: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72		Baseline, Week 72
Secondary	Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot		Week 12
Secondary	Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 72		Baseline, Week 72
Secondary	Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE)		Baseline up to Week 124
Secondary	Part 1 (For Cohort 2 Only): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 72		Baseline, Week 72
Secondary	Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72		Baseline, Week 72

External Links

•   Click here to access the website, clinicaltrials.sarepta.com/ENVISION, for additional information for the study.