The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with HCH aged 0 to \< 36 months over a 52-week period.
Age range
0 Months – 36 Months
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Incidence of treatment-emergent adverse events
Timeframe: From baseline to end of treatment at 52 weeks
Incidence of serious adverse events versus placebo over the course of the study
Timeframe: From baseline to end of treatment at 52 weeks
Changes from baseline in standard clinical laboratory values (hematology, urinalysis, and chemistry)
Timeframe: At week 26, at week 52
Changes from baseline in heart rate
Timeframe: At week 13, at week 26, at week 39, at week 52
Change from baseline in height Z-score
Timeframe: At week 52
Changes from baseline in respiratory rate
Timeframe: At week 13, at week 26, at week 39, at week 52
Changes from baseline in temperature
Timeframe: At week 13, at week 26, at week 39, at week 52
Changes from baseline in blood pressure
Timeframe: At week 13, at week 26, at week 39, at week 52