drugset
drugset / Trial / NCT05824169

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients

NCT05824169 ↗

Phase 1/2 Recruiting 18 enrolled GeneCradle Inc
Non-randomizedSequentialOpen-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 1 (SMA 1) patients.

Timeline

Start
2023-02-25
Primary completion
2026-12
Completion
2026-12

Drugs

EvaluationDrugModalityDoseRoute
Subject GC101 Gene therapy (AAV / viral vector) 2.4e+11 vg Intrathecal
Subject GC101 Gene therapy (AAV / viral vector) 4.8e+11 vg Intrathecal

Indications

spinal muscular atrophy