Mucopolysaccharidosis Type IVA clinical trials at University of California Health
2 in progress, 1 open to eligible people
In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases
open to eligible females ages 18-50
The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.
Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI
Sorry, not yet accepting patients
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.
Our lead scientists for Mucopolysaccharidosis Type IVA research studies include Paul Harmatz, MD Tippi MacKenzie, MD.