SMA II 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 2 Patients
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 2 (SMA 2) patients.
The purpose of this trial is to evaluate safety and efficacy of gene therapy drug GC101 in SMA 2 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 at 12 months after treatment with GC101 on the motor milestone of stand unassisted for at least 3 seconds for patients of age between 6 and 24 months, or changes from baseline HFMSE scores for patients of age between 24 and 60 months. ;
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