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

PHASE1/PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

6

Participants

Timeline

Start Date

September 25, 2023

Primary Completion Date

December 31, 2025

Study Completion Date

December 31, 2026

Conditions
MPS IVAMPS 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.

Trial Locations (1)

94609

UCSF Benioff Children's Hospital Oakland, Oakland

All Listed Sponsors
lead

University of California, San Francisco

OTHER

NCT05845749 - Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI | Biotech Hunter | Biotech Hunter