An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy Clinical Trial

— ENDURE  

Official title:

A Long-term Multicenter Prospective Observational Study Evaluating the Comparative Effectiveness and Safety of Sarepta Gene Transfer Therapy vs. Standard of Care in Participants With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06270719
• Other study ID #: SRP-9001-401
• Status: Recruiting
• Start date: February 7, 2024
• Est. completion date: December 31, 2038

Study information

• Verified date: May 2024
• Source: Sarepta Therapeutics, Inc.
• Contact: Sarepta Therapeutics Inc. For Clinical Trial Information, Select
• Phone: 1-888-SAREPTA (1-888-727-3782)
• Email: SareptAlly[@]sarepta.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

This is a multicenter, prospective, observational Phase 4 study in the United States. The study is designed to collect both medical history and prospective data on Duchenne muscular dystrophy (DMD) treatment outcomes in participants receiving delandistrogene moxeparvovec as part of clinical care, compared to participants with DMD receiving or prescribed to start chronic glucocorticoid treatment at baseline in routine clinical practice. In addition, treatment outcomes will be collected prospectively from post-trial participants who have received delandistrogene moxeparvovec through participation in select SRP-9001 studies.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 500
• Est. completion date: December 31, 2038
• Est. primary completion date: December 31, 2029
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 4 Years and older

Eligibility

Inclusion Criteria:

• Has a definitive diagnosis of DMD prior to Screening based on documentation of clinical findings and confirmatory genetic testing.

Among participants recruited from routine clinical practice:

• Is aged 4 through 5 years at the time of enrollment.

• Is ambulatory per protocol specified criteria.

• Is currently receiving or has been prescribed to start chronic glucocorticoid therapy at the time of this observational study enrollment.

For Delandistrogene Moxeparvovec-exposed Participants:

• Will be initiating usual care treatment with delandistrogene moxeparvovec at the time of study enrollment.

For Comparators:

• Is unexposed to DMD gene therapy at the time of study enrollment.

Exclusion Criteria:

Among participants recruited from routine clinical practice:

• Has any deletion of exon 8 and/or exon 9 in the DMD gene.

• Is currently participating in any DMD interventional study at the time of this observational study enrollment.

• Has a medical condition or confounding circumstances (for example, prior traumatic limitation for mobility or significant behavioral comorbidity) that, in the opinion of the Investigator, might compromise:

• The participant's ability to comply with the protocol-required procedures,

• The participant's wellbeing or safety, and/or

• The clinical interpretability of the data collected from the participant.

Other inclusion/exclusion criteria may apply.

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Genetic:
• delandistrogene moxeparvovec
No study medication will be provided by the sponsor during this trial.

Drug:
• Standard of Care
No study medication will be provided by the sponsor during this trial.

Locations

Country	Name	City	State
United States	Cook Children's Hospital	Fort Worth	Texas
United States	Penn State Health Milton S. Hershey Medical Center	Hershey	Pennsylvania
United States	Arkansas Children's Hospital	Little Rock	Arkansas
United States	Nicklaus Children's Hospital	Miami	Florida
United States	Children's Hospital of the King's Daughters	Norfolk	Virginia

Sponsors (1)

Lead Sponsor	Collaborator
Sarepta Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	All Cohorts: Mean Change From Baseline in Time to Walk/Run 10 Meters (and Calculated Velocity) at Month 12		Baseline, Month 12
Secondary	All Cohorts: Time to Rise From Floor (Supine to Stand)		Up to 10 years
Secondary	All Cohorts: Loss of Ambulation (LOA)		Up to 10 years
Secondary	All Cohorts: Performance of Upper Limb (PUL) Version 2.0 Entry Item Score		Up to 10 years
Secondary	All Cohorts: Patient-reported Outcomes Measurement Information (PROMIS) Domain Scores of Mobility, Upper Extremity and Fatigue		Up to 10 years
Secondary	All Cohorts: Pulmonary Function, as Measured Forced Vital Capacity (FVC) (% Predicted)		Up to 10 years
Secondary	All Cohorts: Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF) as Measured by Echocardiogram (ECHO)		Up to 10 years
Secondary	All Cohorts: Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs)		Up to 10 years
Secondary	Delandistrogene Moxeparvovec Cohorts: Time to Walk/Run 10 Meters (Calculated Velocity)		Up to 10 years
Secondary	Delandistrogene Moxeparvovec Post-trial Cohort: North Star Ambulatory Assessment (NSAA)		Up to 10 years