NCT06971094 - Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

May 27, 2025

Primary Completion Date

December 31, 2026

Study Completion Date

December 31, 2026

Conditions

SMA - Spinal Muscular Atrophy

Interventions

GENETIC

GC101 adeno-associated virus injection

Self-complementary recombinant adeno-associated viral vector (scAAV) containing a single-stranded transgene encoding a codon-optimized human SMN1 gene

Trial Locations (7)

100700

RECRUITING

The Seventh Medical Center of Chinese PLA General Hospital, Beijing

102699

RECRUITING

Children's Medical Center of Peking University First Hospital, Beijing

200127

RECRUITING

National Children's Medical Center，Shanghai Jiaotong University, Shanghai

430015

RECRUITING

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

Unknown

RECRUITING

Beijing Children's Hospital, Capital Medical University, Beijing

RECRUITING

Shenzhen Children's Hospital, Shenzhen

RECRUITING

Children's Hospital of Soochow University, Suzhou

All Listed Sponsors

lead

GeneCradle Inc

INDUSTRY