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NCT05824169

A Multi-center, Open Label, Single-arm, Dose Ascending Clinical Trial for Evaluation of Safety and Efficacy of Gene Therapy Drug GC101 in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients

Study 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.

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Interventions

GC101GENETIC
Self-complementary AAV9 carrying a codon-optimized SMN coding sequence(coSMN1) driven by CMV enhancer and chicken β-actin promoter

Study Locations

FacilityCityStateCountry
Peking University， First Hospital, Department of PediatricsBeijingChina
Bayi Children's Hospital, Seventh Medical Center, PLA general hospitalBeijingChina
Shengjing Hospital of China Medical UniversityShenyangChina
Children's Hospital of Soochow UniversitySuzhouChina

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: May 29, 2026