Clinical Trial

A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 SMA

Enrolling by Invitation
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Summary
An Open-label, Long-term Follow-Up Study to Evaluate the Safety and Tolerability of Gene Therapy with EXG001-307 in SMA1 patients ,who joined the parent study (EXG001-307-102)
Protocol Amendment History 1 amendment
This ClinicalTrials.gov record has been amended once since 2024-09-06.
Status change: Not Yet Recruiting → Enrolling by Invitation 2025-02-28
Trial Details
NCT Number NCT06588803
Lead Sponsor Guangzhou Jiayin Biotech Ltd
Conditions Spinal Muscular Atrophy
Enrollment 18 participants
Start Date 2025-03
Primary Completion 2030-09-10 (estimated)
Study Completion 2030-12-31 (estimated)
Updated on ClinicalTrials.gov 2025-03-05