A Study of Vosoritide for Children with Hypochondroplasia
This study is testing a drug called vosoritide against a placebo (an inactive substance) in children aged 0 to less than 36 months who have hypochondroplasia (HCH), a genetic bone growth disorder. The main goals are to see how safe vosoritide is by tracking any side effects and changes in lab test results over 52 weeks. You may be eligible if your child is between 0 and 36 months old, has a confirmed genetic diagnosis of HCH, and for those under 12 months, a specific height measurement. This study is currently recruiting 60 participants.
- Study design
- This is a Phase 2, randomized, double-blind study, meaning participants will be randomly assigned to receive either vosoritide or a placebo, and neither you nor your doctor will know which one your child is receiving. The study plans to enroll 60 participants.
- What's involved
- Participants will receive daily injections for 52 weeks. There will be follow-up visits to monitor safety and lab values at weeks 26 and 52.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will have a 2-week safety follow-up visit after the 52 weeks of treatment.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
At a glance
Conditions
Where it's being run
26 sites across 20 statesWho to contact
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What this trial measures
- Incidence of treatment-emergent adverse eventsFrom baseline to end of treatment at 52 weeks
- Incidence of serious adverse events versus placebo over the course of the studyFrom baseline to end of treatment at 52 weeks
- Changes from baseline in standard clinical laboratory values (hematology, urinalysis, and chemistry)At week 26, at week 52
- Changes from baseline in heart rateAt week 13, at week 26, at week 39, at week 52
Units of measure: bpm
- Change from baseline in height Z-scoreAt week 52
- Changes from baseline in respiratory rateAt week 13, at week 26, at week 39, at week 52
Units of measure: breaths/min
- Changes from baseline in temperatureAt week 13, at week 26, at week 39, at week 52
Units of measure: celsius
- Changes from baseline in blood pressureAt week 13, at week 26, at week 39, at week 52
Units of measure: mmHg