SKG0201 Injection: SKG0201 is a recombinant adeno-associated virus (rAAV) vector-based gene therapy product.
Study 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.
Eligibility
Sex
ALL
Min age
—
Max age
180 Days
Healthy volunteers
No
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.
Primary outcome measure(s)
Incidence of AEs and SAEs — to 18 months of age AEs: adverse events; SAEs: serious Adverse events
Incidence and characteristics of DLT — 4 weeks DLT: dose-limiting toxicity
Trial sites (3)
Facility
City
Region
Status
Peking University First Hospital
Beijing
Beijing Municipality
Recruiting
Children's Hospital of Fudan University
Shanghai
Shanghai Municipality
Recruiting
Children's Hospital, Zhejiang University School of Medicine
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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