Status: Not yet recruiting
Study of Velaglucerase Beta ERT in Children With Gaucher Disease
This is a Phase 3, multicenter, open-label, single-arm study to evaluate the safety, efficacy, PK, PD, and immunogenicity of velaglucerase beta in untreated children with GD1 or GD3 for 39 weeks.
Participants needed: 10
Trial details
Phase: Phase 3Age: 2-12Biological sex: AllType: InterventionalSponsor: CANbridge Life Sciences Ltd.Updated: Sep 29, 2026Locations: 1
Eligibility criteria
Subject has a documented diagnosis of GD1 or GD3 according to the diagnostic cri... [+6]
Treatment with investigational drugs (including ambroxol) within 30 days or 5 ha... [+6]