U.S., Sept. 30 -- ClinicalTrials.gov registry received information related to the study (NCT07847411) titled 'Study of Velaglucerase Beta ERT in Children With Gaucher Disease' on Aug. 31.
Brief Summary: 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.
Study Start Date: Sept. 16
Study Type: INTERVENTIONAL
Condition:
Gaucher Disease
Gaucher Disease Type 1
Gaucher Disease Type 3
Intervention:
DRUG: Velaglucerase beta
All subjects will receive velaglucerase beta 60 U/kg IV Q2W for 37 weeks with two weeks of follow-up through Week 39 (9 months).
Recruitment Status: NOT_YET_RECRUITING
Sponsor...