Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI
Status:
Not yet recruiting
Trial end date:
2026-12-01
Target enrollment:
Participant gender:
Summary
This is a Phase I/II, single arm, open label study of vosoritide therapy provided
subcutaneously at 15 ug/kg/day for 48 weeks to 6 patients with MPS IVA or VI. Prior to
enrollment in the interventional arm of study, subjects will be followed for a minimum of 24
weeks to gather information on safety profiles and determine annualized growth velocity. The
primary study endpoint is the determination of safety and tolerability of daily vosoritide
treatment in MPS. Exploratory endpoints include changes in linear and segmental growth as
well as biomarkers of growth and bone metabolism.