This is a phase I/II clinical study to evaluate the safety, preliminary efficacy and immunogenicity of SKG0201 injection in subjects with Spinal Muscular Atrophy Type I.
This is a multicenter, open-label, dose-escalation phase I/II clinical study to evaluate the safety, preliminary efficacy and immunogenicity of SKG0201 injection in subjects with Spinal Muscular Atrophy Type I (SMA I).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
11
SKG0201 is a recombinant adeno-associated virus (rAAV) vector-based gene therapy product.
Peking University First Hospital
Beijing, Beijing Municipality, China
RECRUITINGChildren's Hospital of Fudan University
Shanghai, Shanghai Municipality, China
RECRUITINGChildren's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, China
RECRUITINGIncidence of AEs and SAEs
AEs: adverse events; SAEs: serious Adverse events
Time frame: to 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: to 18 months of age
The proportion of subjects whose CHOP-INTEND score reached 40 or above
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: to 18 months of age
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