What is vosoritide studying for hypochondroplasia?

Vosoritide is being studied to assess its safety and effects on growth in infants and young children with hypochondroplasia. Researchers want to understand how daily vosoritide injections compare to placebo over a one-year period. This study is sponsored by BioMarin Pharmaceutical and focuses on children aged 0 to less than 36 months with confirmed genetic diagnosis.

Who may be eligible for this hypochondroplasia clinical trial?

Children from birth up to less than 36 months old with genetically confirmed hypochondroplasia may be eligible. They must meet certain growth measurements based on age and sex and weigh at least 3 kilograms. Children with other short stature conditions or recent treatments like growth hormone may not qualify. Please review eligibility criteria for more details.

Is there any cost to participate in this hypochondroplasia clinical trial?

Study-related care, including the study drug or procedure and all trial visits, is provided at no cost. Personal travel expenses may vary and are not guaranteed to be covered. Participants can expect no charges for the treatment and assessments during the study.

Actively Recruiting

Phase 2

Age: 0Months - 36Months

All Genders

ID07126262

Phase 2 Study of Daily Vosoritide Injections in Infants and Young Children With Hypochondroplasia Aged 0 to Under 3 Years Evaluating Safety and Effectiveness Compared to Placebo

Led by BioMarin Pharmaceutical · Updated on 2026-03-31

Participants Needed

Research Sites

N/A

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating the safety and effects of daily vosoritide treatment compared to placebo in infants and young children with hypochondroplasia HCH, aged from birth up to less than 36 months. This Phase 2, randomized, double-blind, placebo-controlled, multicenter study aims to better understand vosoritides impact on growth and health over one year in this specific young population with genetically confirmed HCH. Participants will be randomly assigned to receive either vosoritide or a placebo through daily subcutaneous injections for 52 weeks. The vosoritide dose will be adjusted based on weight bands approved for a related condition. After treatment, there will be a 2-week safety follow-up to monitor any adverse effects or changes. During the study, participants will have regular evaluations including clinical lab tests, heart rate, respiratory rate, blood pressure, and body temperature measures at several time points. Researchers will assess growth changes such as height, body segment ratios, and bone density using scans, along with monitoring adverse events and vosoritide blood levels. The total participation duration is about 54 weeks including treatment and safety follow-up.

CONDITIONS

Brief Title

A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months

Who Can Participate

Age: 0Months - 36Months

All Genders

Eligibility Criteria

You may qualify if you...

Participants must be 0 to less than 36 months of age at randomization.
Confirmed genetic diagnosis of hypochondroplasia by whole genome sequencing showing a FGFR3 variant.
Participants aged 0 to less than 12 months must have a height Z-score of ≤ -1.0 SDS; those aged 12 to less than 36 months must have a height Z-score of ≤ -2.0 SDS based on CDC growth charts.
Participant's weight at Day 1 visit must be at least 3 kg.

You will not qualify if you...

Presence of short stature condition other than hypochondroplasia (e.g., achondroplasia, trisomy 21, pseudoachondroplasia).
Unstable medical condition likely to require surgery during the study period.
Use of prohibited medications.
Treatment with growth hormone, IGF-1, or anabolic steroids in past 6 months or long-term treatment over 3 months at any time.
Need for investigational agents before study completion.
Receipt of other investigational products or devices within 30 days before screening.
Previous use of investigational treatments for hypochondroplasia or short stature.
Current or history of malignancy or under evaluation for suspected malignancy.
Known hypersensitivity to vosoritide or its ingredients.
Conditions that may risk poor compliance or study completion.
Any concurrent disease or condition that may interfere with participation or safety assessments.

Research Team

Trial Specialist

Not the Right Trial for You?

Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.

Already have an account? Log in here

Phase 2 Study of Daily Vosoritide Injections in Infants and Young Children With Hypochondroplasia Aged 0 to Under 3 Years Evaluating Safety and Effectiveness Compared to Placebo

On this page

AI-Summary

What this Trial Is About

Brief Title

Who Can Participate

Eligibility Criteria

Research Team

Not the Right Trial for You?

On this page

Who Can Participate

Eligibility Criteria