Vosoritide for Selected Genetic Causes of Short Stature

PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

56

Participants

Timeline

Start Date

August 4, 2020

Primary Completion Date

October 15, 2025

Study Completion Date

June 1, 2035

Conditions
Short Stature
Interventions
DRUG

Vosoritide

After enrollment, subjects will be followed for a 6 month observation only period to establish a baseline height velocity as well as safety profile and quality of life assessment. Vosoritide will then be administered daily via subcutaneous injection at a dose of 15 µg/kg/day for 12 months.

Trial Locations (1)

20010

Children's National Hospital, Washington D.C.

All Listed Sponsors
lead

Andrew Dauber

OTHER