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Clinical Trials in the UK / NCT07126262
Recruiting Phase 2

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

NCT07126262 · tracked via the Priya Life Science UK tracker
Sponsor
BioMarin Pharmaceutical
Phase
Phase 2
Started
2025-07-30
Last updated
2026-03-31

Condition(s) studied

Hypochondroplasia

Investigational drug(s) / intervention(s)

VosoritidePlacebo

Vosoritide: The vosoritide dose administered will be based on the participant's weight and will follow the weight-band dosing regimen approved for ACH

Placebo: Subcutaneous injection of recommended dose of placebo

Study summary

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.

Eligibility

Sex
ALL
Min age
0 Months
Max age
36 Months
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (26)

FacilityCityRegionStatus
Phoenix Children's Hospital - Thomas Campus (Main) Phoenix Arizona Recruiting
Cedars-Sinai Medical Center Los Angeles California Not Yet Recruiting
Benioff Children's Hospital - Oakland Oakland California Recruiting
Children's National Medical Center Washington D.C. District of Columbia Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois Recruiting
The Johns Hopkins University School of Medicine Baltimore Maryland Recruiting
University of Minneasota Masonic Children's Hospital Minneapolis Minnesota Recruiting
University of Missouri Columbia Missouri Recruiting
Cook Children's Endocrinology Fort Worth Texas Not Yet Recruiting
Children's Wisconsin - Fox Valley Hospital Neenah Wisconsin Not Yet Recruiting
Children's Health Queensland Hospital and Health Service South Brisbane Queensland Not Yet Recruiting
Royal Children's Hospital Melbourne Parkville Victoria Recruiting
Hôpital Bicêtre Le Kremlin-Bicêtre Paris Recruiting
Hospices Civils de Lyon - Hôpital Femme Mère Enfant Bron Rhone-Alps Not Yet Recruiting
Uniklinik Köln Cologne North Rhine-Westphalia Recruiting
Universitätsklinikum des Saarlandes Homburg Saarland Recruiting
Universitätskinderklinik Magdeburg Magdeburg Saxony-Anhalt Not Yet Recruiting
Fondazione Policlinico Universitario Agostino Gemelli IRCCS Roma Rome Not Yet Recruiting
IRCCS Istituto Giannina Gaslini Genova Italy Recruiting
Kumamoto University Hospital Kumamoto Japan Recruiting
Osaka Women's and Children's Hospital Osaka Japan Recruiting
Institute of Science Tokyo Hospital Tokyo Japan Recruiting
Nihon University Itabashi Hospital Tokyo Japan Recruiting
Tottori University Hospital Tottori Japan Recruiting
Myriad Trials London England Not Yet Recruiting
Great Ormond Street Hospital London England Not Yet Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07126262 on ClinicalTrials.gov ↗ ← All trials in the UK