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RECRUITING
NCT06421831
PHASE1/PHASE2

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients

Sponsor: GeneCradle Inc

View on ClinicalTrials.gov

Summary

The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 3 (SMA 3) patients.

Official title: 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 3 Patients

Key Details

Gender

All

Age Range

2 Years - Any

Study Type

INTERVENTIONAL

Enrollment

21

Start Date

2024-05-10

Completion Date

2028-12

Last Updated

2025-07-03

Healthy Volunteers

No

Interventions

GENETIC

GC101

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

Locations (1)

Beijing Tiantan Hospital, Capital Medical University

Beijing, Beijing Municipality, China