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

Muscular Dystrophy, Duchenne Clinical Trial

— ENDEAVOR  

Official title:

An Open-Label, Systemic Gene Delivery Study Using Commercial Process Material to Evaluate the Safety of and Expression From SRP-9001 in Subjects With Duchenne Muscular Dystrophy (ENDEAVOR)

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT04626674
• Other study ID #: SRP-9001-103
• Status: Enrolling by invitation
• Phase: Phase 1
• Start date: November 23, 2020
• Est. completion date: July 31, 2026

Study information

• Verified date: July 2023
• Source: Sarepta Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with DMD. The maximum participant duration for this study is 156 weeks.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 58
• Est. completion date: July 31, 2026
• Est. primary completion date: November 30, 2024
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 2 Years and older

Eligibility

Inclusion Criteria:

• For Cohorts 1-7: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
• Cohort 1: Is ambulatory, and =4 to <8 years of age at the time of Screening.
• Cohort 2: Is ambulatory, and =8 to <18 years of age at the time of Screening.
• Cohort 3: Non-ambulatory per protocol specified criteria at the time of Screening.
• Cohort 4: Is ambulatory and =3 to <4 years of age at the time of Screening.
• Cohort 5a: Is ambulatory and =4 to <9 years of age.
• Cohort 5b: Non-ambulatory per protocol specified criteria at the time of Screening.
• Cohort 6: Is ambulatory, and =2 to <3 years of age at the time of Screening.
• Cohort 7: Non-ambulatory per protocol-specified criteria at the time of Screening.
• Ability to cooperate with motor assessment testing.
• Cohorts 1, 2, 3, 5, and 7 only: Stable dose equivalent of oral glucocorticoids for at least 12 weeks before screening and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the first year of the study.
• Cohorts 4 and 6: Do not yet require use of chronic steroids for treatment of their DMD, in the opinion of the Investigator, and are not receiving steroids at the time of Screening.
• rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
• Genetic mutation inclusion criteria vary by cohort.

Exclusion Criteria:

• Has a concomitant illness, autoimmune disease, chronic drug treatment, and/or cognitive delay/impairment that in the opinion of the Investigator creates unnecessary risks for gene transfer.
• 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. Other inclusion/exclusion criteria apply.

Related Conditions & MeSH terms

• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Genetic:
• delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec

Locations

Country	Name	City	State
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	Children's Hospital of The King's Daughters	Norfolk	Virginia
United States	Stanford University	Palo Alto	California
United States	University of California, Davis	Sacramento	California
United States	Washington University in St. Louis	Saint Louis	Missouri

Sponsors (2)

Lead Sponsor	Collaborator
Sarepta Therapeutics, Inc.	Hoffmann-La Roche

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Part 1: Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12, as Measured by Western Blot		Baseline, Week 12
Primary	Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot		Week 12
Secondary	Vector Shedding, Measured in Urine, Saliva, and Stool Samples Post-Infusion		Day 1 up to Week 104
Secondary	Level of Antibody Titers to Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74)		Day 2 up to Week 156
Secondary	Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events (AEs) of Special Interest		Baseline up to Week 156
Secondary	Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12, as Measured by Immunofluorescence (IF) Fiber Intensity		Baseline, Week 12
Secondary	Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12, as Measured by IF Percent Dystrophin Positive Fibers (PDPF)		Baseline, Week 12
Secondary	Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by IF Fiber Intensity:		Week 12
Secondary	Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by IF PDPF		Week 12