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.
Type I SMA, defined by bi-allelic mutations in the SMN1 gene.
Clinical history and signs are consistent with type I SMA, such as hypotonia, delayed motor function development, and poor head control.
On the day of administration, the age of the subjects do not exceed 180 days after birth.
Pulse oximetry < 96% saturation at screening while the patient is awake or asleep without any supplemental oxygen or respiratory support.
Weight-for-age below the 3rd percentile for the same sex and age based on WHO Child Growth Standards.
Active viral infection.
In the presence of other severe infections or diseases that require systemic anti-infection treatment.
Sequential
SKG0201 Injection
GeneticSKG0201 is a recombinant adeno-associated virus (rAAV) vector-based gene therapy product.
Incidence of AEs and SAEs
AEs: adverse events; SAEs: serious Adverse events
Incidence and characteristics of DLT
DLT: dose-limiting toxicity
Survival rate
Survival is defined as avoidance of either death or permanent ventilation.
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.
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.
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