History of Changes for Study: NCT05881408

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

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Study Record Versions

Version	Submitted Date	Changes
1	May 19, 2023	None (earliest Version on record)
2	June 7, 2023	Contacts/Locations and Study Status
3	June 30, 2023	Recruitment Status, Arms and Interventions, Study Status, Contacts/Locations, Outcome Measures, Study Description and Study Identification
4	July 28, 2023	Contacts/Locations, Study Status and Outcome Measures
5	September 6, 2023	Contacts/Locations and Study Status
6	October 6, 2023	Contacts/Locations and Study Status
7	November 21, 2023	Contacts/Locations, Study Status and References
8	December 20, 2023	Study Status and Contacts/Locations
9	February 27, 2024	Contacts/Locations and Study Status
10	March 22, 2024	Contacts/Locations and Study Status
11	May 9, 2024	Contacts/Locations and Study Status

Comparison Format:

Merged
Side-by-Side

Study Identification


Unique Protocol ID:	SRP-9001-303
Brief Title:	A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 (Delandistrogene Moxeparvovec) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) (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)
Secondary IDs:	2020-002372-13 [EudraCT Number]

Study Status


Record Verification:	May 2023
Overall Status:	Not yet recruiting
Study Start:	June 30, 2023
Primary Completion:	January 31, 2026 [Anticipated]
Study Completion:	January 31, 2027 [Anticipated]

First Submitted:	May 19, 2023
First Submitted that Met QC Criteria:	May 19, 2023
First Posted:	May 31, 2023 [Actual]

Last Update Submitted that Met QC Criteria:	May 19, 2023
Last Update Posted:	May 31, 2023 [Actual]

Sponsor/Collaborators


Sponsor:	Sarepta Therapeutics, Inc.
Responsible Party:	Sponsor
Collaborators:	Hoffmann-La Roche

Oversight


U.S. FDA-regulated Drug:	Yes
U.S. FDA-regulated Device:	No
Data Monitoring:	Yes

Study Description

Brief Summary:

The study will evaluate the safety and efficacy of SRP-9001 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) SRP-9001 (delandistrogene moxeparvovec) in either Part 1 or Part 2.

Detailed Description:

Conditions


Conditions:	Duchenne Muscular Dystrophy
Keywords:	Ambulatory Non-ambulatory DMD Gene-Delivery Pediatric North Star Ambulatory Assessment (NSAA) Performance of Upper Limb (PUL) Duchenne

Study Design


Study Type:	Interventional
Primary Purpose:	Treatment
Study Phase:	Phase 3
Interventional Study Model:	Parallel Assignment
Number of Arms:	2
Masking:	Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Allocation:	Randomized
Enrollment:	148 [Anticipated]

Arms and Interventions

Arms	Assigned Interventions
Experimental: SRP-9001 followed by Placebo Participants will receive single IV infusion of SRP-9001 on Day 1. Then, participants will receive a single IV infusion of matching placebo at approximately 72 weeks.	Genetic: SRP-9001 Single IV infusion of SRP-9001 Other Names: delandistrogene moxeparvovec Genetic: Placebo Single IV infusion of matching placebo
Placebo Comparator: Placebo followed by SRP-9001 Participants will receive matching placebo IV infusion on Day 1. Then, participants will have the opportunity to receive a single IV infusion of SRP-9001 at approximately 72 weeks.	Genetic: SRP-9001 Single IV infusion of SRP-9001 Other Names: delandistrogene moxeparvovec Genetic: Placebo Single IV infusion of matching placebo

Outcome Measures


Primary Outcome Measures:
1.	Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72 [ Time Frame: Baseline, Week 72 ]
Secondary Outcome Measures:
1.	Part 1: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 72 [ Time Frame: Baseline, Week 72 ]
2.	Part 1: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72 [ Time Frame: Baseline, Week 72 ]
3.	Part 1: Quantity of SRP-9001 Protein Expression at Week 12 as Measured by Western Blot [ Time Frame: Week 12 ]
4.	Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 72 [ Time Frame: Baseline, Week 72 ]
5.	Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE) [ Time Frame: Baseline up to Week 124 ]
6.	Part 1 (For Cohort 2 Only): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 72 [ Time Frame: Baseline, Week 72 ]
7.	Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72 [ Time Frame: Baseline, Week 72 ]

Eligibility


Minimum Age:
Maximum Age:
Sex:	Male
Gender Based:
Accepts Healthy Volunteers:	No
Criteria:	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.

Contacts/Locations


Central Contact Person:	Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4 Telephone: 1-888-SAREPTA (1-888-727-3782) Email: SareptAlly@sarepta.com
Study Officials:	Medical Director Study Director Sarepta Therapeutics, Inc.
Locations:	United States, Arkansas
	Arkansas Children's Little Rock, Arkansas, United States, 72202 Contact:Contact: 501-364-1850 AVeerapandiyan@uams.edu Contact:Principal Investigator: Aravindhan Veerapandiyan, MD
	United States, California
	Rady Children's Hospital-San Diego San Diego, California, United States, 92123 Contact:Contact: 619-736-1373 tpkhounani@health.ucsd.edu, mastechschulte@health.ucsd.edu, ben003@health.ucsd.edu Contact:Principal Investigator: Chamindra Laverty, MD
	United States, Florida
	University of Florida, UF Health Center for Pediatric Neuromuscular and Rare Diseases Gainesville, Florida, United States, 32608 Contact:Contact: SareptAlly@sarepta.com Contact:Principal Investigator: Carmen Leon-Astudillo, MD
	United States, Illinois
	Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States, 60611 Contact:Contact: Kiking@luriechildrens.org Contact:Principal Investigator: Nancy Kuntz, MD
	United States, Iowa
	University of Iowa Hospitals and Clinics, Dept of Pediatrics Iowa City, Iowa, United States, 52242 Contact:Contact: 319-384-9618 ciara-gibbs@uiowa.edu Contact:Principal Investigator: Katherine Mathews, MD
	United States, Maryland
	The Johns Hopkins Hospital, Charlotte R. Bloomberg Children's Center, Pediatric Clinical Research Unit Baltimore, Maryland, United States, 21287 Contact:Contact: 443-287-6294 aking2@jhmi.edu Contact:Principal Investigator: Jessica Nance, MD
	United States, Massachusetts
	Boston Children's Hospital Boston, Massachusetts, United States, 02115 Contact:Contact: alyssa.rohan@childrens.harvard.edu Contact:Principal Investigator: Partha Ghosh, MD
	United States, Missouri
	Washington University of St. Louis, St. Louis Children's Hospital Saint Louis, Missouri, United States, 63110 Contact:Contact: 314-362-6981 NeuromusclePediatricResearch@wustl.edu Contact:Principal Investigator: Craig Zaidman, MD
	United States, New York
	University of Rochester, Dept of Neurology Rochester, New York, United States, 14642 Contact:Contact: Emma_Ciafaloni@URMC.Rochester.edu Contact:Principal Investigator: Emma Ciafaloni, MD
	United States, Ohio
	Nationwide Children's Hospital Columbus, Ohio, United States, 43205 Contact:Contact: 614-722-2661 Jerry.Mendell@nationwidechildrens.org, Leah.Johnson2@nationwidechildrens.org Contact:Principal Investigator: Jerry Mendell, MD
	United States, Pennsylvania
	Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States, 19104 Contact:Contact: brizzolars@chop.edu Contact:Principal Investigator: John Brandesma, MD
	United States, Tennessee
	VUMC- Monroe Carell Jr. Children's Hospital at Vanderbilt Nashville, Tennessee, United States, 37203 Contact:Contact: 615-936-5536 william.b.burnette@vumc.org Contact:Principal Investigator: William B Burnette, MD
	United States, Virginia
	Children's Hospital of the King's Daughters Norfolk, Virginia, United States, 23510 Contact:Contact: 757-668-6981 Proud.research@chkd.org Contact:Principal Investigator: Crystal Proud, MD
	United States, Wisconsin
	Children's Wisconsin Milwaukee, Wisconsin, United States, 53226 Contact:Contact: 414-266-7543 pedsneuroresearch@mcw.edu, admueller@mcw.edu Contact:Principal Investigator: Matthew Harmelink, MD

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