Tests SKG0201 gene therapy in infants with spinal muscular atrophy type 1
Tests SKG0201 gene injection in infants with spinal muscular atrophy type 1.
Plain-language title generated from the registry record
Registry title: A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I
Key facts
- Where
- Beijing, Hangzhou, and Shanghai, ChinaSee the 3 sites
- Phase
- Phase 1/2
Where in China
- Peking University First HospitalBeijing, ChinaTaking people
- Children's Hospital, Zhejiang University School of MedicineHangzhou, ChinaTaking people
- Children's Hospital Of Fudan UniversityShanghai, ChinaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Lanyue Biotech (Hangzhou) Co., Ltd.
- Trial code
- NCT07554924
- Record updated
- 14 October 2026
- Original description
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.Read full original descriptionShow less
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.
Inclusion: 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: 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; 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; Weight-for-age below the 3rd percentile for the same sex and age based on WHO Child Growth Standards; Pulse oximetry < 96% saturation at screening while the patient is awake or asleep without any supplemental oxygen or respiratory support.