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

NCT03375164 | Completed Phase 1 Results DMC FDA Drug

• 2 other identifiers: SRP-9001-1012021-000077-83
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 1

Brief Summary

This study was an open-label single-dose gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec intravenous (IV) administration in boys with DMD. This study was originally designed to consist of 12 patients across 2 Cohorts. Cohort A would have included participants ages 3 months to 3 years, and Cohort B included participants ages 4 to 7 years old. No participants were enrolled in Cohort A.

Conditions

Duchenne Muscular Dystrophy

Keywords

Duchenne; Gene Therapy; DMD; Dystrophin; Pediatric; Gene-Delivery; Gene Transfer Therapy

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Adverse Events (AEs)

  An AE is any untoward medical occurrence in a clinical study participant that does not necessarily have a causal relationship with the study drug. An AE can, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurs during or after administration of a study drug, whether or not considered related to the study drug. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module.

  Up to 5 years

Secondary Outcomes (4)

• Change From Baseline at Day 90 in Delandistrogene Moxeparvovec Dystrophin Expression as Measured by Western Blot

  Baseline, Day 90

• Change From Baseline at Day 90 in Delandistrogene Moxeparvovec Dystrophin Expression as Measured by Immunofluorescence (IF) Fiber Intensity

  Baseline, Day 90

• Change From Baseline at Day 90 in Delandistrogene Moxeparvovec Dystrophin Expression as Measured by IF Percent Dystrophin Positive Fibers (PDPF)

  Baseline, Day 90

• Change From Baseline at Year 5 in the 100 Meter Timed Test

  Baseline, Year 5

Study Arms (2)

Cohort A: Delandistrogene Moxeparvovec

EXPERIMENTAL

Participants will receive a Single IV infusion of delandistrogene moxeparvovec on Day 1.

Genetic: delandistrogene moxeparvovec

Cohort B: Delandistrogene Moxeparvovec

EXPERIMENTAL

Participants will receive a Single IV infusion of delandistrogene moxeparvovec on Day 1.

Genetic: delandistrogene moxeparvovec

Interventions

delandistrogene moxeparvovec GENETIC

Single IV infusion of delandistrogene moxeparvovec.

Also known as: SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS

Cohort A: Delandistrogene Moxeparvovec; Cohort B: Delandistrogene Moxeparvovec

Eligibility Criteria

• Age: 3 Months - 7 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Cohort A participants: 3 months to 3 years of age, inclusive
• Cohort B participants: 4 to 7 years of age, inclusive
• Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
• Ability to cooperate with motor assessment testing.
• Cohort A participants: No previous treatment with corticosteroids.
• Cohort B participants: Stable dose equivalent of oral corticosteroids for at least 12 weeks prior to screening and the dose is expected to remain constant (except for potential modifications to accommodate changes in weight) throughout the first year of the study.
• Cohorts A \& B: A frameshift mutation contained between exons 18 and 58 (inclusive).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Sarepta Therapeutics, Inc.: lead

Study Sites (1)

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Related Publications (2)

• Zaidman CM, Proud CM, McDonald CM, Lehman KJ, Goedeker NL, Mason S, Murphy AP, Guridi M, Wang S, Reid C, Darton E, Wandel C, Lewis S, Malhotra J, Griffin DA, Potter RA, Rodino-Klapac LR, Mendell JR. Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged >/=4 to <8 Years) with Duchenne Muscular Dystrophy: 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR). Ann Neurol. 2023 Nov;94(5):955-968. doi: 10.1002/ana.26755. Epub 2023 Sep 7.

  PMID: 37539981 DERIVED

• Mendell JR, Sahenk Z, Lehman K, Nease C, Lowes LP, Miller NF, Iammarino MA, Alfano LN, Nicholl A, Al-Zaidy S, Lewis S, Church K, Shell R, Cripe LH, Potter RA, Griffin DA, Pozsgai E, Dugar A, Hogan M, Rodino-Klapac LR. Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial. JAMA Neurol. 2020 Sep 1;77(9):1122-1131. doi: 10.1001/jamaneurol.2020.1484.

  PMID: 32539076 DERIVED

Related Links

• SRP-9001-101 Plain Language Study Summary

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Medical Director
• Organization: Sarepta Therapeutics, Inc.

SareptAlly@sarepta.com

888-727-3782

Study Officials

• Medical Director

  Sarepta Therapeutics, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 4, 2017
• First Posted: December 15, 2017
• Study Start: January 4, 2018
• Primary Completion: April 25, 2023
• Study Completion: April 25, 2023
• Last Updated: November 14, 2024
• Results First Posted: May 23, 2024

Record last verified: 2024-11

Locations

US (1)