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

NCT05845749 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2026-03-10

No results posted yet for this study

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.

Conditions

  • MPS IVA
  • MPS VI

Interventions

DRUG

Vosoritide Injection [Voxzogo]

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.

Sponsors & Collaborators

Principal Investigators

  • Ellen B Fung, PhD · University of California, San Francisco

Study Design

Allocation
NA
Purpose
OTHER
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
5 Years
Max Age
10 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-09-25
Primary Completion
2028-12-31
Completion
2029-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05845749 on ClinicalTrials.gov