Study of GC101 Gene Therapy Delivered into the Spine for Treating Spinal Muscular Atrophy Type 1 in Babies Up to 6 Months Old

What this Trial Is About

Researchers are evaluating the safety and effectiveness of the gene therapy drug GC101 for treating patients with Spinal Muscular Atrophy Type 1 SMA 1. This open-label, dose-escalation clinical trial is conducted at multiple centers in China and focuses on children diagnosed with SMA 1, a condition caused by specific genetic mutations. The study aims to understand how well the treatment works in helping patients reach motor milestones and to monitor the safety of the therapy over time. Participants receive a one-time dose of GC101 delivered through an intrathecal injection, which means the drug is administered directly into the spinal fluid. There are two dosage groups a low dose group receiving 2.4x1014 viral genomes per person and a high dose group receiving 4.8x1014 viral genomes per person, each with three patients. The study includes a 52-week short-term safety evaluation followed by an optional long-term follow-up lasting five years. During the trial, patients will have assessments at the start and at various points afterward to monitor their progress. Researchers will track the occurrence of any treatment-related side effects and evaluate if patients can sit without support for at least 10 seconds by 18 months of age. Other measures include survival without major events, ability to thrive, and changes in neuromuscular function scores. The total participation time may extend up to five years for those continuing in the long-term follow-up phase.

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