Tests gene therapy GC101 in infants with spinal muscular atrophy type 1
Tests gene therapy GC101, given into the spinal fluid, in infants with SMA type 1.
Plain-language title generated from the registry record
Registry title: Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients
Key facts
- Where
- Beijing, Shenyang, and Suzhou, ChinaSee the 4 sites
- Phase
- Phase 1/2
Where in China
- Bayi Children's Hospital, Seventh Medical Center, PLA general hospitalBeijing, ChinaTaking people
- Peking University， First Hospital, Department of PediatricsBeijing, ChinaTaking people
- Shengjing Hospital Of China Medical UniversityShenyang, ChinaTaking people
- Children's Hospital of Soochow UniversitySuzhou, ChinaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- GeneCradle Inc
- Trial code
- NCT05824169
- Record updated
- 3 July 2025
- Original description
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 1 (SMA 1) patients.Read full original descriptionShow less
A Multi-center, 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 1 Patients
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 1 (SMA 1) patients.
Inclusion: Six months of age and younger on day of vector infusion with Type 1 SAM as defined by the following features:; Diagnosis of SMA based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and 2 copies of SMN2; Onset of disease before 6 months of age; The 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: Patient who has participated in a previous gene therapy research trials; Patient who has received Nusinersen and Risdiplam treatment; Patient who has AAV9 neutralizing antibody titer ≥1:200; Vaccination less than 2 weeks before infusion of vector; Patient who has any concurrent clinically significant major disease or any other condition that, in the opinion of the Investigator, makes the subject unsuitable for participation in the study; Note: Other protocol defined inclusion/exclusion criteria may apply; Patient who is positive for human immunodeficiency virus (HIV) antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody; Class IV patient based on Modified Ross Heart Failure Classification for Children; Patient with a history of glucocorticoid allergy; Presence of an untreated active infection requiring systemic antiviral therapy at any time during the screening period; Abnormal laboratory values considered clinically significant, including gamma-glutamyl transferase(GGT), Aspartate aminotransferase (AST), alanine aminotransferase (ALT), bilirubin > 3x upper limit of normal (ULN), Hemoglobin (Hgb)< 110 or >150 g/L, platelet <183x10\^9/L or 614x10\^9/L; Patient who requires non-invasive ventilatory support averaging≥16 hours/day; Patient with a point mutation in SMN2 (c.859G>C); Patient who requires non-invasive ventilatory support averaging≥16 hours/day at screening; Patient who use invasive ventilatory support or pulse oximetry < 95% saturation while awake and calm at screening; Contraindication that would interfere with the lumbar puncture procedures.