Trial · Interventional
RecruitingPhase 2Registry dataA Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
Overview
- Status
- Recruiting
- Phase
- Phase 2
- Enrollment
- 60 participants (estimated)
- Sponsor
- BioMarin Pharmaceutical · Industry
- Design
- Randomized · Quadruple masking
- Countries
7 countries
United States, Australia, France, Germany, Italy, Japan, United Kingdom
- Start
- 30 Jul 2025
- Primary completion
- 30 Jun 2028 (estimated)
- Completion
- 30 Jun 2028 (estimated)
- First posted
- 17 Aug 2025
- Last update posted
- 31 Mar 2026
- Conditions
- Hypochondroplasia
Timeline
1 trial · 1 ongoing
- Completed
- Ongoing
- Not yet recruiting
- Stopped
- Estimated dates (est.)
- Results posted
- Ph 2
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
NCT0712626230 Jul 2025 to est. 30 Jun 2028
Registry summary
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
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.
Interventions
- VosoritideDrug
- PlaceboDrug
Arms2
Experimental
Vosoritide injection with vial and syringe
Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily.
Placebo comparator
Placebo injection with vial and syringe
Subcutaneous injection of recommended dose of placebo
Primary outcomes8
Outcomes the sponsor listed as primary.
Incidence of treatment-emergent adverse events
Time frame · From baseline to end of treatment at 52 weeks
Incidence of serious adverse events versus placebo over the course of the study
Time frame · From baseline to end of treatment at 52 weeks
Changes from baseline in standard clinical laboratory values (hematology, urinalysis, and chemistry)
Time frame · At week 26, at week 52
Changes from baseline in heart rate
Time frame · At week 13, at week 26, at week 39, at week 52
Registry description
Units of measure: bpm
Change from baseline in height Z-score
Time frame · At week 52
Changes from baseline in respiratory rate
Time frame · At week 13, at week 26, at week 39, at week 52
Registry description
Units of measure: breaths/min
Changes from baseline in temperature
Time frame · At week 13, at week 26, at week 39, at week 52
Registry description
Units of measure: celsius
Changes from baseline in blood pressure
Time frame · At week 13, at week 26, at week 39, at week 52
Registry description
Units of measure: mmHg
Secondary outcomes(19)
Change in height
Time frame · At week 52
Cumulative annualized growth velocity (AGV)
Time frame · At week 52
6-month interval AGV
Time frame · At week 26, at week 52
Change from baseline in upper to lower body segment ratio
Time frame · At week 52
Change from baseline in arm span
Time frame · At week 52
Change from baseline in total body (less head) bone mineral density (BMD) Z-score
Time frame · At week 52
Change from baseline in lumbar spine BMD Z-score
Time frame · At week 52
Change from baseline in total body (less head) bone mineral content (BMC) as measured by DXA
Time frame · At week 52
Change from baseline in lumbar spine BMC as measured by DXA
Time frame · At week 52
Area under the plasma vosoritide concentration time-curve from time 0 to infinity (AUC0-∞)
Time frame · At week 26, at week 52
Area under the plasma vosoritide concentration time-curve from time 0 to the last measurable concentration (AUC0-t)
Time frame · At week 26, at week 52
Elimination half-life of vosoritide (t½)
Time frame · At week 26, at week 52
Apparent clearance of vosoritide
Time frame · At week 26, at week 52
Apparent volume of distribution of vosoritide based upon the terminal phase (Vz/F)
Time frame · At week 26, at week 52
Time vosoritide is present at maximum concentration (Tmax)
Time frame · At week 26, at week 52
Maximum concentration (Cmax) of vosoritide in plasma
Time frame · At week 26, at week 52
Change from pre-dose at pre-specified timepoints versus placebo in cyclic guanine monophosphate (cGMP)
Time frame · At week 26 and week 52
Incidence of otitis media
Time frame · From baseline to end of treatment at 52 weeks
Seizure frequency over the course of the study
Time frame · From baseline to end of treatment at 52 weeks
Posted results
Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.
No results are posted for this trial.
A trial that is still running, or that stopped early, may post results later.
Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works
Studies0
Studies that cite this trial.
No study cites this trial.
Eligibility
Who could join, as posted.
- Sex
- All
- Age
- from 0 Months up to 36 Months
- Healthy volunteers
- Not accepted
Inclusion and exclusion criteria, as posted
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