A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
← catalyst calendarNCT07126262 · readout in 681 d
Sponsored by BioMarin Pharmaceutical (industry) · BMRN — their whole pipeline →. With ICON Clinical Research.
Phase
Phase 2
Status
Recruiting
Study type
Interventional
Design
Randomized · Quadruple · Treatment
Enrollment
60estimated
Sites
26
Countries
Australia, France, Germany +4
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2025-07-30 | actual | When the study began enrolling |
| Primary completion | 2028-06-30 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2028-06-30 | estimated | The whole study's end, after follow-up |
| First posted | 2025-08-17 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-03-31 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Hypochondroplasia
Interventions
- Drug: Vosoritide
- Drug: Placebo
Primary outcomes
what the primary-completion date above is the date OFIncidence of treatment-emergent adverse events
measured From baseline to end of treatment at 52 weeks
Incidence of serious adverse events versus placebo over the course of the study
measured From baseline to end of treatment at 52 weeks
Changes from baseline in standard clinical laboratory values (hematology, urinalysis, and chemistry)
measured At week 26, at week 52
Changes from baseline in heart rate
measured At week 13, at week 26, at week 39, at week 52
Change from baseline in height Z-score
measured At week 52
Changes from baseline in respiratory rate
measured At week 13, at week 26, at week 39, at week 52
Changes from baseline in temperature
measured At week 13, at week 26, at week 39, at week 52
Changes from baseline in blood pressure
measured At week 13, at week 26, at week 39, at week 52
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