NCT05824169View on ClinicalTrials.gov

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

PHASE1/PHASE2RecruitingINTERVENTIONAL

Enrollment

Participants

Timeline

Start Date

February 25, 2023

Primary Completion Date

December 31, 2026

Study Completion Date

December 31, 2026

Conditions

Spinal Muscular Atrophy

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 (4)

100034

RECRUITING

Peking University， First Hospital, Department of Pediatrics, Beijing

100700

RECRUITING

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

110004

RECRUITING

Shengjing Hospital of China Medical University, Shenyang

215025

RECRUITING

Children's Hospital of Soochow University, Suzhou

All Listed Sponsors

lead

GeneCradle Inc

INDUSTRY

NCT05824169 - Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients | Biotech Hunter | Biotech Hunter