This is a clinical study to evaluate the safety and efficacy of gene therapy drug SKG0201 Injection in patients with spinal muscular atrophy Type 1 (SMA 1).
This is a multicenter, open, dose-escalation clinical study to evaluate the safety, initial efficacy, and immunogenicity of SKG0201 injection in patients with spinal muscular atrophy type 1 (SMA 1).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
SKG0201 is a recombinant adeno-associated virus (rAAV) vector-based in vivo gene therapeutic product.
West China Sencond Hospital, Sichuan University / West China women's and children's Hospital
Chengdu, Sichuan, China
Xinhua Hospital Affiliated To Shanghai Jiao Tong University School Of Medicine
Shanghai, China
National Children's Medical Center, Children's Hospital of Fudan University
Shanghai, China
Incidence of AEs and SAEs
AEs: adverse events; SAEs: serious Adverse events
Time frame: 18 months of age
Incidence and characteristics of DLT
DLT: dose-limiting toxicity
Time frame: 4 weeks
Survival rate
Survival is defined as avoidance of either death or permanent ventilation.
Time frame: 14 months of age
CHOP-INTEND score changes from baseline
CHOP-INTEND (Children's hospital of Philadelphia Infant Test of Neuromuscular Disorders) score ranges from 0 to 64 with higher scores indicating higher motor function.
Time frame: 24 weeks
Proportion of subjects who achieve developmental milestones with improvement in exercise intensity and function assessed according to BSID-III
Developmental milestones are defined according to BSID-III (Bayley Scales of Infant and Toddler Development Third Edition) criteria.
Time frame: 24 weeks
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