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

StatusUnknown
PhasePhase 1/2
Started
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.

Record History

  1. Changed Registry marked status Unknown (sponsor has not verified the record since Sep 2024); last known: Enrolling by Invitation
  2. Start date changed from estimate of Nov 1, 2022 to Feb 16, 2023 Trial status: Recruiting to Enrolling by Invitation · Primary and study completion dates: Mar 2025 to Aug 2025 · Site details revised at 2 of 2 sitesv2 Show 3 more changesShow less
    • Trial status: Recruiting to Enrolling by Invitation
    • Primary and study completion dates: Mar 2025 to Aug 2025
    • Site details revised at 2 of 2 sites
  3. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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).
Trial Details
NCT Number NCT05614531
Lead Sponsor Guangzhou Jiayin Biotech Ltd
Conditions Spinal Muscular Atrophy Type I
Enrollment 12 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov