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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) (ENVISION)

Primary Purpose

Duchenne Muscular Dystrophy

Status

Recruiting

Phase

Phase 3

Locations

United States

Study Type

Interventional

Intervention

delandistrogene moxeparvovec

placebo

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring Ambulatory, Non-ambulatory, DMD, Gene-Delivery, Pediatric, North Star Ambulatory Assessment (NSAA), Performance of Upper Limb (PUL), Duchenne

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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.

Sites / Locations

Arkansas Children's HospitalRecruiting
Lucile Packard Children's Hospital StanfordRecruiting
University of California at Davis Medical CenterRecruiting
Rady Children's Hospital-San DiegoRecruiting
University of Florida, UF Health Center for Pediatric Neuromuscular and Rare DiseasesRecruiting
Ann & Robert H. Lurie Children's Hospital of ChicagoRecruiting
University of Iowa Hospitals and Clinics, Dept of PediatricsRecruiting
Washington University of St. Louis, St. Louis Children's HospitalRecruiting
University of Rochester, Department of NeurologyRecruiting
Lenox Baker Children's Hospital (Duke University)Recruiting
Children's Hospital of PhiladelphiaRecruiting
Children's Hospital of the King's DaughtersRecruiting

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 IV infusion of delandistrogene moxeparvovec on Day 1. Then, participants will receive a single IV infusion of matching placebo at approximately 72 weeks.

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.

Outcomes

Primary Outcome Measures

Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72

Secondary Outcome Measures

Part 1: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 72

Part 1: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72

Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot

Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 72

Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE)

Part 1 (For Cohort 2 Only): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 72

Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72

Full Information

NCT ID

NCT05881408

First Posted

May 19, 2023

Last Updated

October 6, 2023

Sponsor

Sarepta Therapeutics, Inc.

Collaborators

Hoffmann-La Roche

1. Study Identification

Unique Protocol Identification Number

NCT05881408

Brief 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)

Acronym

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)

Study Type

Interventional

2. Study Status

Record Verification Date

October 2023

Overall Recruitment Status

Recruiting

Study Start Date

May 31, 2023 (Actual)

Primary Completion Date

January 31, 2026 (Anticipated)

Study Completion Date

January 31, 2027 (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

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Duchenne Muscular Dystrophy

Keywords

Ambulatory, Non-ambulatory, DMD, Gene-Delivery, Pediatric, North Star Ambulatory Assessment (NSAA), Performance of Upper Limb (PUL), Duchenne

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

148 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Delandistrogene Moxeparvovec followed by Placebo

Arm Type

Experimental

Arm Description

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.

Arm Title

Placebo followed by Delandistrogene Moxeparvovec

Arm Type

Placebo Comparator

Arm Description

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 the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72

Time Frame

Baseline, Week 72

Secondary Outcome Measure Information:

Title

Part 1: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 72

Time Frame

Baseline, Week 72

Title

Part 1: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72

Time Frame

Baseline, Week 72

Title

Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot

Time Frame

Week 12

Title

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

Title

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

Title

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

Title

Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72

Time Frame

Baseline, Week 72

10. Eligibility

Sex

Male

Accepts Healthy Volunteers

Eligibility Criteria

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4

Phone

1-888-SAREPTA (1-888-727-3782)

SareptAlly@sarepta.com

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 Hospital

City

Little Rock

State/Province

Arkansas

ZIP/Postal Code

72202

Country

United States

Individual Site Status

Recruiting

Facility Contact:

Phone

501-364-1850

AVeerapandiyan@uams.edu

First Name & Middle Initial & Last Name & Degree

Aravindhan Veerapandiyan, MD

Facility Name

Lucile Packard Children's Hospital Stanford

City

Palo Alto

State/Province

California

ZIP/Postal Code

94304

Country

United States

Individual Site Status

Recruiting

Facility Contact:

Phone

650-725-4341

neuromuscularresearch@stanford.edu

First Name & Middle Initial & Last Name & Degree

Carolina Tesi-Rocha, MD

Facility Name

University of California at Davis Medical Center

City

Sacramento

State/Province

California

ZIP/Postal Code

95817

Country

United States

Individual Site Status

Recruiting

Facility Contact:

Phone

916-734-5057

nmrl@ucdavis.edu

First Name & Middle Initial & Last Name & Degree

Craig McDonald, MD

Facility Name

Rady Children's Hospital-San Diego

City

San Diego

State/Province

California

ZIP/Postal Code

92123

Country

United States

Individual Site Status

Recruiting

Facility Contact:

Phone

619-736-1373

tpkhounani@health.ucsd.edu

First Name & Middle Initial & Last Name & Degree

Chamindra Laverty, MD

Facility Name

University of Florida, UF Health Center for Pediatric Neuromuscular and Rare Diseases

City

Gainesville

State/Province

Florida

ZIP/Postal Code

32608

Country

United States

Individual Site Status

Recruiting

Facility Contact:

SareptAlly@sarepta.com

First Name & Middle Initial & Last Name & Degree

Carmen Leon-Astudillo, MD

Facility Name

Ann & Robert H. Lurie Children's Hospital of Chicago

City

Chicago

State/Province

Illinois

ZIP/Postal Code

60611

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Kiana King

Kiking@luriechildrens.org

First Name & Middle Initial & Last Name & Degree

Nancy Kuntz, MD

Facility Name

University of Iowa Hospitals and Clinics, Dept of Pediatrics

City

Iowa City

State/Province

Iowa

ZIP/Postal Code

52242

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Ciara Gibbs

Phone

319-384-9618

ciara-gibbs@uiowa.edu

First Name & Middle Initial & Last Name & Degree

Katherine Mathews, MD

Facility Name

Washington University of St. Louis, St. Louis Children's Hospital

City

Saint Louis

State/Province

Missouri

ZIP/Postal Code

63110

Country

United States

Individual Site Status

Recruiting

Facility Contact:

Phone

314-362-6981

neuromusclepediatricresearch@wustl.edu

First Name & Middle Initial & Last Name & Degree

Craig Zaidman, MD

Facility Name

University of Rochester, Department of Neurology

City

Rochester

State/Province

New York

ZIP/Postal Code

14642

Country

United States

Individual Site Status

Recruiting

Facility Contact:

Emma_Ciafaloni@URMC.Rochester.edu

First Name & Middle Initial & Last Name & Degree

Emma Ciafaloni, MD

Facility Name

Lenox Baker Children's Hospital (Duke University)

City

Durham

State/Province

North Carolina

ZIP/Postal Code

27705

Country

United States

Individual Site Status

Recruiting

Facility Contact:

SareptAlly@sarepta.com

First Name & Middle Initial & Last Name & Degree

Mai Kamal ElMallah, MD

Facility Name

Children's Hospital of Philadelphia

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19104

Country

United States

Individual Site Status

Recruiting

Facility Contact:

brizzolars@chop.edu

First Name & Middle Initial & Last Name & Degree

John Brandsema, MD

Facility Name

Children's Hospital of the King's Daughters

City

Norfolk

State/Province

Virginia

ZIP/Postal Code

23510

Country

United States

Individual Site Status

Recruiting

Facility Contact:

SareptAlly@sarepta.com

First Name & Middle Initial & Last Name & Degree

Crystal Proud, MD

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 Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

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