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

Spinal Muscular Atrophy Clinical Trial

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 1 Patients

Clinical Trial 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.

Clinical Trial Description

The purpose of this trial is to evaluate safety and efficacy of gene therapy drug GC101 in SMA 1 patients. Open-label, dose-escalation clinical trial of GC101 will be conducted in multiple centers in China. GC101 will be administrated intrathecally. Short-term safety will be evaluated in 52 weeks and enter long-term follow-up study of 5 years at will. Patients will be tested at baseline and followed up on various time points. The primary analysis for efficacy will be assessed when all patients reach 18 months of age on the motor milestone of sit unassisted for at least 10 seconds. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05824169
Study type Interventional
Source GeneCradle Inc
Contact GeneCradle, Inc. China
Phone 86-13501380583
Email ind@bj-genecradle.com
Status Recruiting
Phase Phase 1/Phase 2
Start date February 25, 2023
Completion date December 2025
View Details
See also
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