A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
Condition(s) studied
Investigational drug(s) / intervention(s)
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
Primary outcome measure(s)
- Incidence of treatment-emergent adverse events — From baseline to end of treatment at 52 weeks
- Incidence of serious adverse events versus placebo over the course of the study — From 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 rate — At week 13, at week 26, at week 39, at week 52
Units of measure: bpm - Change from baseline in height Z-score — At week 52
- Changes from baseline in respiratory rate — At week 13, at week 26, at week 39, at week 52
Units of measure: breaths/min - Changes from baseline in temperature — At week 13, at week 26, at week 39, at week 52
Units of measure: celsius - Changes from baseline in blood pressure — At week 13, at week 26, at week 39, at week 52
Units of measure: mmHg
Trial sites (26)
| Facility | City | Region | Status |
|---|---|---|---|
| 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 |
More BioMarin Pharmaceutical trials in Italy
Other trials for the same condition
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 Italy