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Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients

StatusRecruiting
PhasePhase 1/2
Started
View on ClinicalTrials.gov ↗

Record History

  1. 2 versions Re-verified since Sep 23, 2024, no change to tracked fieldsv2-v3
  2. 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  • Stopped: an ongoing trial was terminated, suspended or withdrawn.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 study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 3 (SMA 3) patients.
Trial Details
NCT Number NCT06421831
Lead Sponsor GeneCradle Inc
Conditions Spinal Muscular Atrophy Type 3
Enrollment 21 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov