Active — Not RecruitingPhase 1/2

Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI

United States6 participantsStarted 2023-09-25

Plain-language summary

This is a Phase I/II, single arm, open label study of vosoritide therapy provided subcutaneously at 15 ug/kg/day for 96 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.

Who can participate

Age range5 Years – 10 Years

SexALL

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Inclusion criteria

✓. Demonstration of 2 pathogenic or likely pathogen mutations (or homozygous for single mutation) and elevated GAG (either before or during ERT treatment), OR
✓. Demonstration of diagnostic enzyme deficiency, elevated GAG (either before or during ERT treatment), and a normal second sulfatase

What they're measuring

Safety and tolerability: Incidence of adverse events while treated with vosoritide

Timeframe: 48 weeks

Trial details

NCT IDNCT05845749

SponsorUniversity of California, San Francisco

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2028-12-31

View on ClinicalTrials.gov More MPS IVA trials