SKG0201 Injection in SMA Type I: safety and early signals in infants

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a multicenter, open-label, dose-escalation Phase I/II trial to assess safety, preliminary efficacy, and immunogenicity of SKG0201 in infants with SMA Type I. Participants are enrolled across several large Chinese hospitals and receive escalating doses of SKG0201 to determine tolerability and potential early effectiveness. The study excludes patients with significant respiratory compromise, poor growth, active infections, or prior SMA gene therapy. Enrollment targets infants within 180 days of birth, with SMA confirmed by SMN1 mutations. Lead sponsor is Lanyue Biotech, with collaborations at three major institutions.

Why it matters

Who can join: the full rules

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.

Where it's running

• Peking University First Hospital
  Beijing, Beijing Municipality, China Recruiting
• Children's Hospital of Fudan University
  Shanghai, Shanghai Municipality, China Recruiting
• Children's Hospital, Zhejiang University School of Medicine
  Hangzhou, Zhejiang, China Recruiting

Study contacts

• Study coordinator: Mingxiu Li
• Email: mingxiu.li@skytx.com
• Phone: +86 19121738351

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.