A new open-label, dose-escalation, multicenter clinical trial is set to evaluate the safety and efficacy of GC101, a gene therapy drug, in patients with spinal muscular atrophy type 2 (SMA 2), a condition characterized by muscle weakness and motor function impairment.

GC101 will be administered intrathecally. The trial will consist of a short-term safety evaluation period lasting 52 weeks, followed by a long-term follow-up study lasting 5 years. Patients will undergo baseline assessments and will be followed up at various time points throughout the trial.

The trial will assess the efficacy of the drug using motor milestones. For patients aged between 6 and 24 months, the primary outcome will be the proportion of patients who can stand unassisted for at least 3 seconds at 12 months after treatment. For patients aged between 24 and 60 months, the primary outcome will be changed from baseline in Hammersmith Functional Motor Scale – Expanded (HFMSE) scores at 12 months.

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The secondary outcome measures to be evaluated in the trial include the proportion of patients achieving the motor milestone of walking alone for 5 steps at 12 months, changes in independent ventilatory support time, and improvement in HFMSE scores.

The study aims to enroll 33 participants and will follow a non-randomized, sequential assignment design. To be eligible for the trial, participants must be between 6 and 60 months of age and have a diagnosis of SMA type 2 based on gene mutation analysis.

Patients must be able to sit alone but never be able to stand or walk alone. Exclusion criteria include previous participation in gene therapy research trials, prior treatment with certain medications, and certain medical conditions.

The trial, officially titled “A Multicenter, 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,” is estimated to start in July 2023 and conclude in December 2028.

The results of this trial could potentially contribute to advancements in gene therapy approaches for SMA.


Evaluation of safety and efficacy of gene therapy drug in the treatment of Spinal Muscular Atrophy (SMA) type 2 patients. ClinicalTrials.gov. Updated June 18, 2023. Accessed July 3, 2023.