Clinical Trial

Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1

Enrolling by Invitation Phase 1/2
View on ClinicalTrials.gov →
Record status
This record was last updated September 19, 2024 (before its estimated August 2025 completion). Its status may not reflect the trial's current state.
Summary
The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of EXG001-307 as a treatment of spinal muscular atrophy Type 1 (SMN1).
Protocol Amendment History 1 amendment
This ClinicalTrials.gov record has been amended once since 2022-11-07.
Status change: Recruiting → Enrolling by Invitation 2024-09-06
Trial Details
NCT Number NCT05614531
Lead Sponsor Guangzhou Jiayin Biotech Ltd
Conditions Spinal Muscular Atrophy Type I
Enrollment 12 participants
Start Date 2023-02-16
Primary Completion 2025-08 (estimated)
Study Completion 2025-08 (estimated)
Updated on ClinicalTrials.gov 2024-09-19