drugset
drugset / Trial / NCT06421831

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

NCT06421831 ↗

Phase 1/2 Recruiting 21 enrolled GeneCradle Inc
NaSingle-groupOpen-labelTreatment

Summary

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.

Timeline

Start
2024-05-10
Primary completion
2026-12
Completion
2028-12

Drugs

EvaluationDrugModalityDoseRoute
Subject GC101 Gene therapy (AAV / viral vector) 1.2e+14 vg Intrathecal

Indications

spinal muscular atrophy, type III