A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study

Duchenne Muscular Dystrophy Clinical Trial

— EXPEDITION  

Official title:

A Phase 3, Multinational, Long-term Follow-up Study to Evaluate Safety and Efficacy in Subjects Who Have Previously Received SRP-9001 in a Clinical Study

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT05967351
• Other study ID #: SRP-9001-305
• Secondary ID: 2023-505043-39-0
• Status: Enrolling by invitation
• Phase: Phase 3
• Start date: September 27, 2023
• Est. completion date: November 30, 2030

Study information

• Verified date: April 2024
• Source: Sarepta Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received delandistrogene moxeparvovec in a previous clinical study. No study drug will be administered as part of this study. Pre-infusion baseline will be defined as the timepoint just prior to infusion of delandistrogene moxeparvovec from a previous clinical study. Each participant will be followed for a minimum of 5 years post-infusion of delandistrogene moxeparvovec from a previous clinical study. The duration of participation in this study is dependent on the length of follow-up the participant completed post-infusion of delandistrogene moxeparvovec from a previous clinical study.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 400
• Est. completion date: November 30, 2030
• Est. primary completion date: November 30, 2030
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Received delandistrogene moxeparvovec for Duchenne muscular dystrophy in a previous clinical study.
• Has (a) parent(s) or legal caregiver(s) or is =18 years of age and able to understand and comply with the study visit schedule and all other protocol requirements.

Exclusion Criteria:

• Participant or family does not want to disclose participation with general practitioner/primary care physician and other medical providers. Other inclusion/exclusion criteria may apply.

Related Conditions & MeSH terms

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

Intervention

Genetic:
• delandistrogene moxeparvovec
No study drug will be administered as part of this study. Eligible participants who received treatment with delandistrogene moxeparvovec during a previous clinical study will be included.

Locations

Country	Name	City	State
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	UCLA Medical Center	Los Angeles	California
United States	Children's Hospital Wisconsin	Milwaukee	Wisconsin
United States	Children's Hospital of The King's Daughters	Norfolk	Virginia
United States	Lucile Packard Children's Hospital Stanford (LPCH)	Palo Alto	California
United States	University of Rochester Medical Center	Rochester	New York
United States	Washington University School of Medicine	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	Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)		Up to 5 years after dosing
Secondary	Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec		Baseline, 5 years
Secondary	Change in Time to Rise From Floor From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec		Baseline, 5 years
Secondary	Change in the Time of 10-meter Walk/Run (10MWR) From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec		Baseline, 5 years
Secondary	Change in Performance of Upper Limb (PUL) (Version 2.0) Total Scores From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec		Baseline, 5 years
Secondary	Change in PUL (Version 2.0) Domain Specific Scores From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec		Baseline, 5 years
Secondary	Change in Forced Vital Capacity Percent (FVC%) Predicted From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec		Baseline, 5 years
Secondary	Change in Peak Expiratory Flow Percent (PEF%) Predicted From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec		Baseline, 5 years
Secondary	Change in Cardiac Magnetic Resonance Imaging (MRI) Findings From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec		Baseline, 5 years
Secondary	Change in Musculoskeletal MRI Findings From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec		Baseline, 5 years