Summary

Eligibility
for people ages 5-10 (full criteria)
Location
at UCSF
Dates
study started
completion around
Principal Investigator
by Paul Harmatz, MD (ucsf)
Headshot of Paul Harmatz
Paul Harmatz

Description

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.

Official Title

A Proof of Concept Study to Evaluate the Safety and Efficacy of Voxzogo (Vosoritide) for the Treatment of Growth Deficits in MPS IVA and VI

Details

Keywords

MPS IVA, MPS VI, Growth, Vosoritide, Children, Height, Voxzogo, Mucopolysaccharidosis IV, Vosoritide Injection [Voxzogo]

Eligibility

Location

  • UCSF Benioff Children's Hospital Oakland
    Oakland California 94609 United States

Lead Scientist at University of California Health

  • Paul Harmatz, MD (ucsf)
    Dr. Paul Harmatz is a gastroenterologist who specializes in mucopolysaccharidoses (MPS) and other lysosomal storage diseases (genetic disorders in which a lack of certain enzymes results in progressive damage to cells and organ systems). He leads a team of specialists who diagnose and care for patients with these rare diseases, offering therapies such as weekly enzyme infusion.

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
University of California, San Francisco
ID
NCT05845749
Phase
Phase 1/2 research study
Study Type
Interventional
Participants
About 6 people participating
Last Updated