A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I

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

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).

Inclusion Criteria:

1. Type I SMA, defined by bi-allelic mutations in the SMN1 gene.
2. Clinical history and signs are consistent with type I SMA, such as hypotonia, delayed motor function development, and poor head control.
3. On the day of administration, the age of the subjects do not exceed 180 days after birth.

Exclusion Criteria:

1. Pulse oximetry < 96% saturation at screening while the patient is awake or asleep without any supplemental oxygen or respiratory support.
2. Weight-for-age below the 3rd percentile for the same sex and age based on WHO Child Growth Standards.
3. Active viral infection.
4. In the presence of other severe infections or diseases that require systemic anti-infection treatment.
5. Known allergy to prednisolone, other glucocorticoids, or SKG0201's excipients.
6. Clinically significant abnormal laboratory values prior to administration.
7. Previously used Zolgensma or other SMA gene therapy drugs, or currently participating in other SMA clinical studies on therapeutic drugs.
8. Having previously undergone major surgery or expected to undergo major surgery during the study assessment period.