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This Phase 1/2 study evaluates the safety and efficacy of the gene therapy drug GC101 in patients with type 3 spinal muscular atrophy (SMA). The drug is delivered intrathecally, into the fluid around the spinal cord. Eligible participants are at least 2 years old, have a genetic and clinical diagnosis of type 3 SMA with bi-allelic SMN1 deletion of 5qSMA, and an HFMSE score between 10 and 54 at screening.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * ≥2 years of age on the day of signing the informed consent form; * Genetic and clinical diagnosis of type 3 SMA with bi-allelic deletion of SMN1 of 5qSMA; * Hammersmith Functional Motor Scale - Expanded (HFMSE) score is between 10 and 54 at screening; * Female patients of childbearing age who are pregnant or lactating, as well as all enrolled patients （both male and female), should take effective contraceptive measures within 6 months after the treatment; * Patients or patient's legal guardian(s) must be able to understand the purpose and risks of the study and voluntarily provide signed and dated informed consent prior to any study-related procedures being performed. Exclusion Criteria: * Patient who has participated in any previous gene therapy research trials; * Patient who has AAV9 neutralizing antibody titer ≥1:200; * Patient who has received Nusinersen within 120 days and Risdiplam within 15 days before treatment; * Patient who requires invasive or non-invasive ventilatory support averaging≥16 hours/day at screening; * SMN2 copy numbers \>4； * Patient who needs nasal or gastric tube feeding for eating; * Patient who is positive for human immunodeficiency virus (HIV) antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody; * Known allergy or hypersensitivity to prednisolone or other glucocorticosteroids or their excipients * Severe contractures at screening that interfere with either the ability to attain/demonstrate functional measures or with the ability to receive intrathecal (IT) dosing; * Patient who has other serious diseases, such as severe cardiovascular and cerebrovascular diseases, digestive system diseases, urinary system diseases, endocrine system diseases, hematological diseases, immune system diseases, nervous system diseases (including but not limited to epilepsy, meningitis, history of convulsions or seizures, cerebrospinal fluid circulation disorders), and mental illnesses, etc.; * Patient with previous injuries (such as upper or lower limb fractures) or surgical operations that have not fully recovered or reached a stable state; * Vaccination no longer than 2 weeks before treatment; * Patient who has any other condition that, in the opinion of the investigator, makes the subject unsuitable for participation in the study.