U.S., Jan. 14 -- ClinicalTrials.gov registry received information related to the study (NCT06772402) titled 'GCB-001 in Treatment of Patients with Type II (SMA) Spinal Muscular Atrophy' on Jan. 03.
Brief 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 cohor...