Not Yet RecruitingPhase 1/2

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

China11 participantsStarted 2026-05-15

Plain-language 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.

Who can participate

Age range180 Days

SexALL

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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.

What they're measuring

Incidence of AEs and SAEs

Timeframe: to 18 months of age

Incidence and characteristics of DLT

Timeframe: 4 weeks

Trial details

NCT IDNCT07554924

SponsorLanyue Biotech (Hangzhou) Co., Ltd.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2029-05-25

Contact for this trial

Yongqin Wang

+86 18616737445 yongqin.wang@skytx.com

View on ClinicalTrials.gov More Spinal Muscular Atrophy 1 trials