NCT05901987View on ClinicalTrials.gov

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

PHASE1/PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

33

Participants

Timeline

Start Date

August 1, 2023

Primary Completion Date

April 22, 2025

Study Completion Date

December 31, 2028

Conditions
SMA II
Interventions
GENETIC

GC101

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

Trial Locations (5)

100034

Peking University， First Hospital, Department of Pediatrics, Beijing

100700

Bayi Children's Hospital, Seventh Medical Center, PLA general hospital, Beijing

Unknown

West China Second University Hospital, Sichuan University, Chengdu

Children's Hospital of Chongqing Medical University, Chongqing

Tongji Medical college of Huazhong University of Science&Technology, Affiliated Children's Hospital, Wuhan

All Listed Sponsors
lead

GeneCradle Inc

INDUSTRY