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)

Research summary

This study will evaluate long-term safety and efficacy in approximately 400 subjects who were dosed with SRP-9001 in a previous clinical study. The study will consist of 2 periods as follows: • An Eligibility Confirmation Visit of up to 2 days in length, during which eligibility confirmation, informed consent, disease characteristics, and current therapy/ concomitant medication data will be collected. • A Long-Term Follow-Up Period that will begin the day eligibility is confirmed and continue for at least 5 years post-infusion of SRP-9001, during which safety and efficacy will be evaluated. Subjects will be expected to attend in-person visits every 6 months to complete required procedures/assessments.

Principal Investigator

Prof Laurent Servais

Contact us

Email: childrensresearch@ouh.nhs.uk

IRAS number

1008633