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RECRUITING
Updated: Aug 26, 2026
A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I
Phase
N/A
Early 1
1
2
3
4
Brief Summary
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.
Key Information
Participation Criteria
Study Design
Locations & Contacts
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
- Known allergy to prednisolone, other glucocorticoids, or SKG0201's excipients.
- Clinically significant abnormal laboratory values prior to administration.
- Previously used Zolgensma or other SMA gene therapy drugs, or currently participating in other SMA clinical studies on therapeutic drugs.
- Having previously undergone major surgery or expected to undergo major surgery during the study assessment period.