drugset
drugset / Trial / NCT05901987

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients

NCT05901987 ↗

Phase 1/2 Active not recruiting 33 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 2 (SMA 2) patients.

Timeline

Start
2023-08-01
Primary completion
2025-04-22
Completion
2028-12

Drugs

EvaluationDrugModalityDoseRoute
Subject GC101 Gene therapy (AAV / viral vector) 1.2e+14 vg Intrathecal
Subject GC101 Gene therapy (AAV / viral vector) 2.4e+14 vg Intrathecal
Subject GC101 Gene therapy (AAV / viral vector) 4.8e+14 vg Intrathecal

Indications

spinal muscular atrophy, type II