Skip to main content
OpenTrials
Enrolling by Invitation

NCT Number: NCT05967351

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

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.

Enrolling by Invitation

Interested in participating?

Request Info

Key information

Conditions

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

Sex eligibility

Male

Study type

Interventional

Phase

Phase 3

Primary location

Universitair Ziekenhuis Gent, Neuromuscular Reference Centre (NMRC), Ghent, East Flanders, Belgium

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

  • Not applicable

Treatment and study plan

delandistrogene moxeparvovec

Genetic

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.

Other names: SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS

Primary outcomes

  1. Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)

    Time frame: Up to 10 years

Secondary outcomes

  1. Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation

    Time frame: Baseline, up to 10 years

  2. Change in Time to Rise From Floor From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation

    Time frame: Baseline, up to 10 years

  3. Change in the Time of 10-meter Walk/Run (10MWR) From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation

    Time frame: Baseline, up to 10 years

  4. Change in Performance of Upper Limb (PUL) (Version 2.0) Total Scores From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation

    Time frame: Baseline, up to 10 years

  5. Change in PUL (Version 2.0) Domain Specific Scores From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation

    Time frame: Baseline, up to 10 years

  6. Change in Forced Vital Capacity Percent (FVC%) Predicted From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation

    Time frame: Baseline, up to 10 years

  7. Change in Peak Expiratory Flow Percent (PEF%) Predicted From Pre-infusion Baseline to the End of the Study Participation of Delandistrogene Moxeparvovec

    Time frame: Baseline, up to 10 years

  8. Change in Cardiac Magnetic Resonance Imaging (MRI) Findings From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation

    Time frame: Baseline, up to 10 years

  9. Change in Musculoskeletal MRI Findings From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation

    Time frame: Baseline, up to 10 years

Sponsors and collaborators

Lead sponsor

Sarepta Therapeutics, Inc.

Industry

Collaborators

  • Hoffmann-La Roche

Registry information

Official study 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

Acronym: EXPEDITION

Important dates

Study start
2023
Primary completion
2033
Study completion
2033
First posted
Aug 1, 2023
Registry last updated
Feb 6, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.