Back to resultsA Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) (EMBARK)
Primary Purpose
Duchenne Muscular Dystrophy
Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
delandistrogene moxeparvovec
placebo
Sponsored by
About this trial
This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring Muscular Dystrophies, Duchenne Muscular Dystrophy, DMD, North Star Ambulatory Assessment (NSAA), Ambulatory, Pediatric, Gene-Delivery
Eligibility 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.
Sites / Locations
- Arkansas Children's
- UC San Diego Altman Clinical and Translational Research Institute
- UCLA Medical Center
- Lucile Packard Children's Hospital at Stanford
- University of California, Davis
- Children's Hospital Colorado
- University of Florida
- Lurie Children's Hospital of Chicago
- University of Iowa Stead Family Children's Hospital
- The Johns Hopkins Hospital
- Boston Children's Hospital
- Washington University of St. Louis
- Columbia University/NYPH
- University of Rochester
- Duke University Medical Center, Lenox Baker Children's Hospital
- Nationwide Children's Hospital
- Oregon Health & Science University
- The Children's Hospital of Philadelphia
- Vanderbilt University Medical Center
- University of Texas Southwestern
- University of Utah Hospital
- Children's Hospital of the King's Daughters
- Children's Wisconsin
- University Hospital Ghent
- LMU - Klinikum der Universitaet Muenchen - Kinderklinik und
- Universitätsklinikum Essen - Klinik für Kinderheilkunde I
- University Hospital Hamburg- Eppendorf
- Hong Kong Children's Hospital
- IRCCS Istituto G.Gaslini, U.O.
- UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
- UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gamelli IRCCS
- Kobe University Hospital
- National Center for Child Health and Development
- Tokyo Women's Medical University Hospital - Pediatrics
- National Center of Neurology and Psychiatry
- Hospital Universitari i Politécnico La Fe
- Hospital Sant Joan de Déu
- Kaohsiung Medical University Chung-Ho Memorial Hospital
- National Taiwan University Hospital
- Oxford University Hospitals NHS Foundation Trust
- Great Ormond Street Hospital for Children NHS Foundation Trust
- The Newcastle Upon Tyne NHS Hospital NHS Foundation Trust, Royal Victoria Infirmary
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Delandistrogene Moxeparvovec followed by Placebo
Placebo followed by Delandistrogene Moxeparvovec
Arm Description
Participants will receive single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1. Then, participants will receive a single IV infusion of matching placebo at Year 2.
Participants will receive matching placebo IV infusion on Day 1. Then, participants will have the opportunity to receive a single IV infusion of delandistrogene moxeparvovec at Year 2.
Outcomes
Primary Outcome Measures
Part 1: Change From Baseline in NSAA Total Score at Week 52
Secondary Outcome Measures
Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot, in a Subset of Participants
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
Part 1: Change From Baseline in Stride Velocity 95th Centile (SV95C) Measured by a Wearable Device
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.
Part 1: Number of Skills Gained or Improved at Week 52 as Measured by the NSAA
Number of Participants with a Treatment Emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)
Full Information
NCT ID
NCT05096221
First Posted
October 14, 2021
Last Updated
September 6, 2023
Sponsor
Sarepta Therapeutics, Inc.
Collaborators
Hoffmann-La Roche
1. Study Identification
Unique Protocol Identification Number
NCT05096221
Brief Title
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
Acronym
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)
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
October 27, 2021 (Actual)
Primary Completion Date
October 31, 2023 (Anticipated)
Study Completion Date
November 30, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Sarepta Therapeutics, Inc.
Collaborators
Hoffmann-La Roche
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
Keywords
Muscular Dystrophies, Duchenne Muscular Dystrophy, DMD, North Star Ambulatory Assessment (NSAA), Ambulatory, Pediatric, Gene-Delivery
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
126 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Delandistrogene Moxeparvovec followed by Placebo
Arm Type
Experimental
Arm Description
Participants will receive single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1. Then, participants will receive a single IV infusion of matching placebo at Year 2.
Arm Title
Placebo followed by Delandistrogene Moxeparvovec
Arm Type
Placebo Comparator
Arm Description
Participants will receive matching placebo IV infusion on Day 1. Then, participants will have the opportunity to receive a single IV infusion of delandistrogene moxeparvovec at Year 2.
Intervention Type
Genetic
Intervention Name(s)
delandistrogene moxeparvovec
Other Intervention Name(s)
SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS
Intervention Description
Single IV infusion of delandistrogene moxeparvovec.
Intervention Type
Genetic
Intervention Name(s)
placebo
Intervention Description
Single IV infusion of matching placebo.
Primary Outcome Measure Information:
Title
Part 1: Change From Baseline in NSAA Total Score at Week 52
Time Frame
Baseline, Week 52
Secondary Outcome Measure Information:
Title
Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot, in a Subset of Participants
Time Frame
Week 12
Title
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
Title
Part 1: Change From Baseline in Stride Velocity 95th Centile (SV95C) Measured by a Wearable Device
Time Frame
Baseline, Week 52
Title
Part 1: Change from Baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Score in Mobility and Upper Extremity Function to Week 52
Description
PROMIS is a family of instruments developed and validated to assess health-related quality of life.
Time Frame
Baseline, Week 52
Title
Part 1: Number of Skills Gained or Improved at Week 52 as Measured by the NSAA
Time Frame
Baseline, Week 52
Title
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
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
7 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Sarepta Therapeutics, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Arkansas Children's
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Facility Name
UC San Diego Altman Clinical and Translational Research Institute
City
La Jolla
State/Province
California
ZIP/Postal Code
92037
Country
United States
Facility Name
UCLA Medical Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
Lucile Packard Children's Hospital at Stanford
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States
Facility Name
University of California, Davis
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
Children's Hospital Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
University of Florida
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32608
Country
United States
Facility Name
Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
University of Iowa Stead Family Children's Hospital
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
The Johns Hopkins Hospital
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Washington University of St. Louis
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Columbia University/NYPH
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
University of Rochester
City
Rochester
State/Province
New York
ZIP/Postal Code
14642
Country
United States
Facility Name
Duke University Medical Center, Lenox Baker Children's Hospital
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27705
Country
United States
Facility Name
Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43210
Country
United States
Facility Name
Oregon Health & Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
The Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Vanderbilt University Medical Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
University of Texas Southwestern
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
University of Utah Hospital
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
Children's Hospital of the King's Daughters
City
Norfolk
State/Province
Virginia
ZIP/Postal Code
23510
Country
United States
Facility Name
Children's Wisconsin
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
Facility Name
University Hospital Ghent
City
Ghent
ZIP/Postal Code
9000
Country
Belgium
Facility Name
LMU - Klinikum der Universitaet Muenchen - Kinderklinik und
City
Bayern
ZIP/Postal Code
80337
Country
Germany
Facility Name
Universitätsklinikum Essen - Klinik für Kinderheilkunde I
City
Essen
ZIP/Postal Code
45147
Country
Germany
Facility Name
University Hospital Hamburg- Eppendorf
City
Hamburg
ZIP/Postal Code
20251
Country
Germany
Facility Name
Hong Kong Children's Hospital
City
Kowloon
Country
Hong Kong
Facility Name
IRCCS Istituto G.Gaslini, U.O.
City
Genoa
ZIP/Postal Code
16147
Country
Italy
Facility Name
UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
City
Milano
ZIP/Postal Code
20122
Country
Italy
Facility Name
UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gamelli IRCCS
City
Roma
ZIP/Postal Code
00168
Country
Italy
Facility Name
Kobe University Hospital
City
Kobe
ZIP/Postal Code
650-0017
Country
Japan
Facility Name
National Center for Child Health and Development
City
Tokyo
ZIP/Postal Code
157-8535
Country
Japan
Facility Name
Tokyo Women's Medical University Hospital - Pediatrics
City
Tokyo
ZIP/Postal Code
162-8666
Country
Japan
Facility Name
National Center of Neurology and Psychiatry
City
Tokyo
ZIP/Postal Code
187-8551
Country
Japan
Facility Name
Hospital Universitari i Politécnico La Fe
City
Valencia
State/Province
Comunidad Valencia
Country
Spain
Facility Name
Hospital Sant Joan de Déu
City
Barcelona
ZIP/Postal Code
08950
Country
Spain
Facility Name
Kaohsiung Medical University Chung-Ho Memorial Hospital
City
Kaohsiung
ZIP/Postal Code
807
Country
Taiwan
Facility Name
National Taiwan University Hospital
City
Taipei
ZIP/Postal Code
100225
Country
Taiwan
Facility Name
Oxford University Hospitals NHS Foundation Trust
City
Headington
State/Province
Oxford
ZIP/Postal Code
OX3 9DU
Country
United Kingdom
Facility Name
Great Ormond Street Hospital for Children NHS Foundation Trust
City
London
ZIP/Postal Code
WC1N3JK
Country
United Kingdom
Facility Name
The Newcastle Upon Tyne NHS Hospital NHS Foundation Trust, Royal Victoria Infirmary
City
Newcastle Upon Tyne
ZIP/Postal Code
NE1 4LP
Country
United Kingdom
12. IPD Sharing Statement
Learn more about this trial
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
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