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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05881408




Registration number
NCT05881408
Ethics application status
Date submitted
19/05/2023
Date registered
31/05/2023
Date last updated
24/07/2024

Titles & IDs
Public title
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)
Scientific 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)
Secondary ID [1] 0 0
2020-002372-13
Secondary ID [2] 0 0
SRP-9001-303
Universal Trial Number (UTN)
Trial acronym
ENVISION
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Duchenne Muscular Dystrophy 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - delandistrogene moxeparvovec
Treatment: Other - placebo

Experimental: Delandistrogene Moxeparvovec followed by Placebo - Participants will receive single IV infusion of delandistrogene moxeparvovec on Day 1. Then, participants will receive a single IV infusion of matching placebo at approximately 72 weeks.

Placebo comparator: Placebo followed by Delandistrogene Moxeparvovec - 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 approximately 72 weeks.


Treatment: Other: delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec

Treatment: Other: placebo
Single IV infusion of matching placebo
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72
Timepoint [1] 0 0
Baseline, Week 72
Secondary outcome [1] 0 0
Part 1: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 72
Timepoint [1] 0 0
Baseline, Week 72
Secondary outcome [2] 0 0
Part 1: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72
Timepoint [2] 0 0
Baseline, Week 72
Secondary outcome [3] 0 0
Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot
Timepoint [3] 0 0
Week 12
Secondary outcome [4] 0 0
Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 72
Timepoint [4] 0 0
Baseline, Week 72
Secondary outcome [5] 0 0
Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE)
Timepoint [5] 0 0
Baseline up to Week 124
Secondary outcome [6] 0 0
Part 1 (For Cohort 2 Only): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 72
Timepoint [6] 0 0
Baseline, Week 72
Secondary outcome [7] 0 0
Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72
Timepoint [7] 0 0
Baseline, Week 72

Eligibility
Key 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).
Minimum age
No limit
Maximum age
No limit
Sex
Males
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
31/05/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/01/2027
Actual
Sample size
Target
148
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
The Children's Hospital at Westmead - Westmead
Recruitment hospital [2] 0 0
The Royal Children's Hospital - Parkville
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arkansas
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Iowa
Country [6] 0 0
United States of America
State/province [6] 0 0
Maryland
Country [7] 0 0
United States of America
State/province [7] 0 0
Massachusetts
Country [8] 0 0
United States of America
State/province [8] 0 0
Missouri
Country [9] 0 0
United States of America
State/province [9] 0 0
New York
Country [10] 0 0
United States of America
State/province [10] 0 0
North Carolina
Country [11] 0 0
United States of America
State/province [11] 0 0
Ohio
Country [12] 0 0
United States of America
State/province [12] 0 0
Pennsylvania
Country [13] 0 0
United States of America
State/province [13] 0 0
Virginia
Country [14] 0 0
Belgium
State/province [14] 0 0
Oost-Vlaanderen
Country [15] 0 0
Italy
State/province [15] 0 0
Genova
Country [16] 0 0
Italy
State/province [16] 0 0
Milano
Country [17] 0 0
Italy
State/province [17] 0 0
Roma
Country [18] 0 0
Japan
State/province [18] 0 0
Osaka
Country [19] 0 0
Japan
State/province [19] 0 0
Tokyo
Country [20] 0 0
Spain
State/province [20] 0 0
Barcelona
Country [21] 0 0
Spain
State/province [21] 0 0
Valencia
Country [22] 0 0
United Kingdom
State/province [22] 0 0
Greater London
Country [23] 0 0
United Kingdom
State/province [23] 0 0
Newcastle Upon Tyne

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Sarepta Therapeutics, Inc.
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Hoffmann-La Roche
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief 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.
Trial website
https://clinicaltrials.gov/study/NCT05881408
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Sarepta Therapeutics, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4
Address 0 0
Country 0 0
Phone 0 0
1-888-SAREPTA (1-888-727-3782)
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05881408