Status: Not yet recruiting
A Phase I/II Clinical Study to Evaluate 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.
Participants needed: 11
Trial details
Phase: Phase 1, Phase 2Age: Up to 180Biological sex: AllType: InterventionalSponsor: Lanyue Biotech (Hangzhou) Co., Ltd.Updated: Jun 8, 2026Locations: 3
Eligibility criteria
Type I SMA, defined by bi-allelic mutations in the SMN1 gene. [+2]
Pulse oximetry < 96% saturation at screening while the patient is awake or aslee... [+7]