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

Duchenne Muscular Dystrophy Clinical Trial

— 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 Subjects With Duchenne Muscular Dystrophy (EMBARK)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05096221
• Other study ID #: SRP-9001-301
• Secondary ID: 2019-003374-91
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: October 27, 2021
• Est. completion date: November 30, 2024

Study information

• Verified date: November 2023
• Source: Sarepta Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 126
• Est. completion date: November 30, 2024
• Est. primary completion date: October 4, 2023
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 4 Years to 7 Years

Eligibility

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.

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	University Hospital Ghent	Ghent
Germany	LMU - Klinikum der Universitaet Muenchen - Kinderklinik und	Bayern
Germany	Universitätsklinikum Essen - Klinik für Kinderheilkunde I	Essen
Germany	University Hospital Hamburg- Eppendorf	Hamburg
Hong Kong	Hong Kong Children's Hospital	Kowloon
Italy	IRCCS Istituto G.Gaslini, U.O.	Genoa
Italy	UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico	Milano
Italy	UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gamelli IRCCS	Roma
Japan	Kobe University Hospital	Kobe
Japan	National Center for Child Health and Development	Tokyo
Japan	National Center of Neurology and Psychiatry	Tokyo
Japan	Tokyo Women's Medical University Hospital - Pediatrics	Tokyo
Spain	Hospital Sant Joan de Déu	Barcelona
Spain	Hospital Universitari i Politécnico La Fe	Valencia	Comunidad Valencia
Taiwan	Kaohsiung Medical University Chung-Ho Memorial Hospital	Kaohsiung
Taiwan	National Taiwan University Hospital	Taipei
United Kingdom	Oxford University Hospitals NHS Foundation Trust	Headington	Oxford
United Kingdom	Great Ormond Street Hospital for Children NHS Foundation Trust	London
United Kingdom	The Newcastle Upon Tyne NHS Hospital NHS Foundation Trust, Royal Victoria Infirmary	Newcastle Upon Tyne
United States	Children's Hospital Colorado	Aurora	Colorado
United States	The Johns Hopkins Hospital	Baltimore	Maryland
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	University of Texas Southwestern	Dallas	Texas
United States	Duke University Medical Center, Lenox Baker Children's Hospital	Durham	North Carolina
United States	University of Florida	Gainesville	Florida
United States	University of Iowa Stead Family Children's Hospital	Iowa City	Iowa
United States	UC San Diego Altman Clinical and Translational Research Institute	La Jolla	California
United States	Arkansas Children's	Little Rock	Arkansas
United States	UCLA Medical Center	Los Angeles	California
United States	Children's Wisconsin	Milwaukee	Wisconsin
United States	Vanderbilt University Medical Center	Nashville	Tennessee
United States	Columbia University/NYPH	New York	New York
United States	Children's Hospital of the King's Daughters	Norfolk	Virginia
United States	Lucile Packard Children's Hospital at Stanford	Palo Alto	California
United States	The Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Oregon Health & Science University	Portland	Oregon
United States	University of Rochester	Rochester	New York
United States	University of California, Davis	Sacramento	California
United States	Washington University of St. Louis	Saint Louis	Missouri
United States	University of Utah Hospital	Salt Lake City	Utah

Sponsors (2)

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

Countries where clinical trial is conducted

United States,  Belgium,  Germany,  Hong Kong,  Italy,  Japan,  Spain,  Taiwan,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Part 1: Change From Baseline in NSAA Total Score at Week 52		Baseline, Week 52
Secondary	Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot, in a Subset of Participants		Week 12
Secondary	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		Baseline, Week 52
Secondary	Part 1: Change From Baseline in Stride Velocity 95th Centile (SV95C) Measured by a Wearable Device		Baseline, Week 52
Secondary	Part 1: Change from Baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Score in Mobility and Upper Extremity Function to Week 52	PROMIS is a family of instruments developed and validated to assess health-related quality of life.	Baseline, Week 52
Secondary	Part 1: Number of Skills Gained or Improved at Week 52 as Measured by the NSAA		Baseline, Week 52
Secondary	Number of Participants with a Treatment Emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)		Baseline up to Week 104