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.
The purpose of this trial is to evaluate safety and efficacy of gene therapy drug GC101 in SMA 2 patients. Open-label, dose-escalation clinical trial of GC101 will be conducted in multiple centers in China. GC101 will be administrated intrathecally. Short-term safety will be evaluated in 52 weeks and enter long-term follow-up study of 5 years at will. Patients will be tested at baseline and followed up on various time points. The primary analysis for efficacy will be assessed at 12 months after treatment with GC101 on the motor milestone of stand unassisted for at least 3 seconds for patients of age between 6 and 24 months, or changes from baseline HFMSE scores for patients of age between 24 and 60 months.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
33
Self-complementary AAV9 carrying a codon-optimized SMN coding sequence(coSMN1) driven by CMV enhancer and chicken β-actin promoter
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
Incidence of Treatment-Emergent Adverse Events
Frequency of treatment-related adverse events (AEs), serious adverse events (SAEs), and changes from baseline in relevant clinical laboratory tests
Time frame: 52 weeks
Proportion of patients who can stand unassisted for at least 3 seconds at Month 12
Time frame: 52 weeks
Change from baseline on Hammersmith Functional Motor Scale - Expanded (HFMSE) scores at Month 12
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.
Time frame: 52 weeks
Proportion of patients treated with GC101 who achieve motor milestone of walk alone for 5 steps at Month 12
Time frame: 52 weeks
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Tongji Medical college of Huazhong University of Science&Technology, Affiliated Children's Hospital
Wuhan, China