臨床研究等提出・公開システム

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 (EXPEDITION)

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

O Malley Patrick

Sarepta Therapeutics, Inc.

215 First Street, Cambridge, MA 02142 USA

SareptAlly@sarepta.com

Rosario Chikako

Parexel International Inc.

Kayabacho Tower, 1-21-2, Shinkawa, Chuo-ku, Tokyo, 104-0033

+81-80-8929-3137

Clinicaltrial-registration@parexel.com

Pending

June. 10, 2024

Observational

single arm study

open(masking not used)

no treatment control/standard of care control

single assignment

other

- 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.

Not applicable

Male

Duchenne muscular dystrophy (DMD)

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 after dosing.

- Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline of Delandistrogene
Moxeparvovec to the End of the Study Participation 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 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 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 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 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 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 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 up to 10 Years - Change in Musculoskeletal MRI Findings From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of
the Study Participation up to 10 Years

81-78-382-5111

June. 27, 2024

No

US/UK/Belgium/Spain/Germany/Italy/Hong Kong/Taiwan/France/Sweden/Israel/Australia/Canada/South Korea