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

Part of paid clinical trials in Oakland, California.

Sponsor
University of California, San Francisco
Study ID
NCT05845749
Phase
PHASE1/PHASE2
Status
Active Not Recruiting

Conditions

  • MPS IVA
  • MPS VI

Eligibility Criteria

Sex
ALL
Age
5 Years - 10 Years
Healthy Volunteers
Not accepted

Interventions

  • Vosoritide Injection [Voxzogo] — DRUG
    Vosoritide will be given via a once daily subcutaneous injection at a dose of 15 ug/kg/day, at approximately the same time each day when feasible. Vosoritide will be supplied to the subject as 0.4 mg vial, 0.56 mg vial or 1.2 mg vials to be reconstituted with sterile water up to 0.8 mg/mL or 2 mg/mL concentrations for injection. The volume to be administered (injection volume) will be based on the subject's body weight and the concentration of vosoritide. All supplies will be provided to the subject for home based administration after training at the study site.

Study Details

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.

Key Dates

Start date
Sep 25, 2023
Status verified
Mar 2026
Primary completion
Dec 31, 2028
Completion
Dec 31, 2029

Study Design

Enrollment
6 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
OTHER

Arms

  • Other: Vosoritide
    This is a single arm open label study of daily SQ dose of vosoritide

Primary Outcome Measure

Safety and tolerability: Incidence of adverse events while treated with vosoritide [ Time Frame: 48 weeks ]

Locations (1)

FacilityCityStateZIPSite coordinators
UCSF Benioff Children's Hospital OaklandOaklandCalifornia94609-

Find similar trials in Oakland, CA

By research site
UCSF Benioff Children's Hospital Oakland· Oakland, CA

Related Studies