NCT06772402

GCB-001 in Treatment of Patients With Type II (SMA) Spinal Muscular Atrophy

An Open Label, Single Arm IIT Clinical Study Evaluating the Safety, Tolerability, and Preliminary Efficacy of GCB-001 in the Treatment of Patients With Delayed Onset Type 2 SMA Who Can Sit Alone But Cannot Walk.

• Status: Enrolling By Invitation
• Phase: N/A
• Study type: Interventional
• Enrollment: 6 (estimated)
• Sponsor: Genecombio Ltd. · Academic / Other
• Sex: All
• Age: 2 Years – 12 Years
• Healthy volunteers: Not accepted

Summary

This study explored dose escalation of single-arm, open, single intrathecal injection in patients with delayed onset type 2 SMA. The investigator plans to conduct 2 cohorts. It is expected that each dose will be enrolled 3 subjects, with a total of 6 subjects aged from 2-12 years old. For safety reasons, first subject of each dose cohort needs to complete a 30-day safety observation. After the researcher determines that the dosing is safe and tolerable, the next two subjects can be enrolled in the cohort; The follow-up dose cohort adopts a sentinel test design, with the first subject of each dose group being a sentinel. During the DLT observation period, if the subject does not observe DLT and the researcher believes that continuing treatment can bring clinical benefits to the subject, the subject will continue to receive treatment; During the DLT observation period, if there is no occurrence of DLT or ≥ grade 2 adverse events related to the investigational drug, it will be escalated to the next dose. If the subject experiences grade ≥ 2 adverse events related to the study drug, the dose will be expanded to 3 subjects for further safety observation. Each subject in each dose cohort will be enrolled on a case by case basis.

Conditions

• Spinal Muscular Atrophy Type 2

Interventions

Type	Name	Description
GENETIC	GCB-001	GCB-001 is a self-complementary AAV9 carrying a full length human SMN transgenetic product.

Timeline

• Start date: 2025-01-15
• Primary completion: 2026-12-01
• Completion: 2030-12-01
• First posted: 2025-01-13
• Last updated: 2025-03-07

Locations

1 site across 1 country: China