Clinical Trials in China / NCT05901987
Active, not recruiting
Phase 1/2
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients
NCT05901987 · tracked via the Priya Life Science China tracker
Condition(s) studied
SMA II
Investigational drug(s) / intervention(s)
GC101
GC101: Self-complementary AAV9 carrying a codon-optimized SMN coding sequence(coSMN1) driven by CMV enhancer and chicken β-actin promoter
Study summary
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 2 (SMA 2) patients.
Eligibility
Inclusion Criteria:
* Between 6 months and 60 months of age on day of signing informed consent form;
* Patient with SMA Type 2 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 SMN
* Onset of disease between 6 and 18 months of age
* Patient who can sit alone but never be able to stand or walk alone ;
* 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 Criteria:
* Patient who has participated in any previous gene therapy research trials;
* Patient who has received Nusinersen within 4 months and Risdiplam within 15 days before treatment;
* Patient who has AAV9 neutralizing antibody titer ≥1:200;
* Patient with a point mutation in SMN2 (c.859G\>C);
* Patient who requires non-invasive ventilatory support averaging≥12 hours/day at screening, or use invasive ventilatory support or pulse oximetry \< 95% saturation while awake and calm at screening;
* Patient who is positive for human immunodeficiency virus (HIV) antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody;
* 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 \<lower limit of normal (LLN);Class IV patient based on Modified Ross Heart Failure Classification for Children;
* Patient with a history of glucocorticoid allergy;
* Contraindication that would interfere with the lumbar puncture procedures;
* Presence of an untreated active infection requiring systemic antiviral therapy at any time during the screening period;
* 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.
Primary outcome measure(s)
- Incidence of Treatment-Emergent Adverse Events — 52 weeks
Frequency of treatment-related adverse events (AEs), serious adverse events (SAEs), and changes from baseline in relevant clinical laboratory tests - Proportion of patients who can stand unassisted for at least 3 seconds at Month 12 — 52 weeks
- Change from baseline on Hammersmith Functional Motor Scale - Expanded (HFMSE) scores at Month 12 — 52 weeks
HFMSE consists of 33 activities that can be scored one of three ways: 0 for unable to perform, 1 for performs with modification/adaptation, and 2 for performs without modification.
Trial sites (5)
| Facility | City | Region | Status |
|---|---|---|---|
| Peking University， First Hospital, Department of Pediatrics | Beijing | China | |
| Bayi Children's Hospital, Seventh Medical Center, PLA general hospital | Beijing | China | |
| West China Second University Hospital, Sichuan University | Chengdu | China | |
| Children's Hospital of Chongqing Medical University | Chongqing | China | |
| Tongji Medical college of Huazhong University of Science&Technology, Affiliated Children's Hospital | Wuhan | China |
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Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05901987 on ClinicalTrials.gov ↗ ← All trials in China