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Daily vosoritide versus placebo for infants and toddlers with hypochondroplasia

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Vosoritide in Infants and Young Children With Hypochondroplasia, Aged 0 to < 36 Months

Phase 2 Interventional BioMarin Pharmaceutical · NCT07126262

This trial will test whether daily vosoritide injections for 52 weeks help infants and toddlers under 36 months with genetically confirmed hypochondroplasia grow better and stay healthier.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment60 (estimated)
Ages0 Months to 36 Months
SexAll
SponsorBioMarin Pharmaceutical Industry-sponsored
Locations26 sites (Phoenix, Arizona and 25 other locations)
Trial IDNCT07126262 on ClinicalTrials.gov

What this trial studies

This Phase 2, randomized, double-blind, placebo-controlled, multicenter trial compares daily subcutaneous vosoritide to placebo in infants and young children with genetically confirmed hypochondroplasia. Eligible participants (0 to <36 months) are randomized 1:1 and receive weight-band dosed vosoritide or matching placebo once daily for 52 weeks, followed by a 2-week safety visit. Key entry criteria include a confirmed FGFR3 pathogenic variant, age-specific height Z-score cutoffs, and a minimum pre-treatment weight of 3 kg. The study is conducted at multiple U.S. pediatric centers with regular on-site visits for dosing, monitoring, and outcome measurements.

Who should consider this trial

Good fit: Ideal candidates are infants and toddlers under 36 months with a genetic diagnosis of hypochondroplasia who meet the study's height Z-score and weight requirements and can attend study visits.

Not a fit: Children whose short stature is caused by conditions other than hypochondroplasia, those with unstable medical conditions likely to need surgery during the study, or those unable to receive daily subcutaneous injections are unlikely to benefit from this trial.

Why it matters

Potential benefit: If successful, vosoritide could increase linear growth and improve early health outcomes for young children with hypochondroplasia.

How similar studies have performed: Vosoritide has shown growth benefits in children with achondroplasia in prior clinical trials, but its effects in hypochondroplasia are less well established and remain under study.

Eligibility criteria

Show full inclusion / exclusion criteria 
Key Inclusion Criteria:

1. Participants must be 0 to \< 36 months of age at randomization.
2. Participants must have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated with HCH).
3. Participants aged 0 to \< 12 months must have a height Z-score of ≤ -1.0 SDS andparticipants aged ≥ 12 to \< 36 months must have a height Z-score of ≤ -2.0 SDS in reference to the average stature of the same sex and age, as calculated using the Center for Disease Control and Prevention (CDC) growth charts.
4. Participant's weight at the Day 1 visit (pre-treatment) must be ≥ 3 kg.

Key Exclusion Criteria:

1. Short stature condition other than HCH (eg, ACH, trisomy 21, pseudoachondroplasia).
2. Have an unstable medical condition likely to require surgical intervention during the study period.
3. Taking any of the prohibited medications.
4. Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the 6 months prior to Screening, or long-term treatment (\> 3 months) at any time.
5. Require any investigational agent prior to completion of study period.
6. Have received another investigational product or investigational medical device within 30 days prior to the Screening visit.
7. Have used any other investigational product or investigational medical device for the treatment of HCH or short stature at any time.
8. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy.
9. Have known hypersensitivity to vosoritide or its excipients.
10. Have a condition or circumstance that, in the view of the investigator, places the participant at high risk for poor treatment compliance or for not completing the study.
11. Have any concurrent disease or condition that, in the view of the investigator, will interfere with study participation or safety evaluations, for any reason.

Where this trial is running

Phoenix, Arizona and 25 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Hypochondroplasia
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.