Daily vosoritide versus placebo for infants and toddlers with hypochondroplasia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• Phoenix Children's Hospital - Thomas Campus (Main)
  Phoenix, Arizona, United States Recruiting
• Benioff Children's Hospital - Oakland
  Oakland, California, United States Recruiting
• Children's National Medical Center
  Washington D.C., District of Columbia, United States Recruiting
• Ann & Robert H. Lurie Children's Hospital of Chicago
  Chicago, Illinois, United States Recruiting
• The Johns Hopkins University School of Medicine
  Baltimore, Maryland, United States Recruiting
• University of Minneasota Masonic Children's Hospital
  Minneapolis, Minnesota, United States Recruiting

Study contacts

• Study coordinator: Trial Specialist
• Email: medinfo@bmrn.com
• Phone: 1-800-983-4587

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Registry for patients with achondroplasia and hypochondroplasia

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.