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

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

Status

Enrolling by invitation

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT05967351

Acronym

EXPEDITION

Enrollment

400

Registered

2023-08-01

Start date

2023-09-27

Completion date

2033-10-31

Last updated

2026-02-06

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne Muscular Dystrophy

Keywords

Duchenne Muscular Dystrophy, DMD, Pediatric, Long-term, Follow-up, Safety, Efficacy

Brief 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.

Interventions

GENETICdelandistrogene 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.

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

OTHER

Masking

NONE

Eligibility

Sex/Gender

MALE

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame
Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)	Up to 10 years

Secondary

Measure	Time frame
Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation	Baseline, up to 10 years
Change in Time to Rise From Floor From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation	Baseline, up to 10 years
Change in the Time of 10-meter Walk/Run (10MWR) From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation	Baseline, up to 10 years
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	Baseline, up to 10 years
Change in PUL (Version 2.0) Domain Specific Scores From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation	Baseline, up to 10 years
Change in Forced Vital Capacity Percent (FVC%) Predicted From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation	Baseline, up to 10 years
Change in Peak Expiratory Flow Percent (PEF%) Predicted From Pre-infusion Baseline to the End of the Study Participation of Delandistrogene Moxeparvovec	Baseline, up to 10 years
Change in Cardiac Magnetic Resonance Imaging (MRI) Findings From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation	Baseline, up to 10 years
Change in Musculoskeletal MRI Findings From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation	Baseline, up to 10 years

Countries

Belgium, Germany, Hong Kong, Italy, Japan, Spain, Taiwan, United Kingdom, United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 7, 2026