Hypochondroplasia: Vosoritide Treatment Study

We are testing a new medication, vosoritide, to see if it helps children with hypochondroplasia grow taller compared to a placebo. The study aims to evaluate its effects on growth velocity and height.

1-2 yearsConfirmation phase (III)Post-Trial Drug AccessCost ReimbursementPartially RemoteStandard MedicinesInjectionPediatrics

What diseases are being studied?

Health conditions and diseases that the clinical trial is designed to study and treat.

Who can participate in the clinical trial?

These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..

What medicines are being studied?

Medicines that are officially approved for use in the European Union and considered safe by EMA. These medicines are already on the market and may be tested for new conditions or populations.

Voxzogo
Voxzogo is a medicine used to help children with achondroplasia grow taller.
Water For Injections Kabi
Water For Injections Kabi is sterile water used to dissolve or dilute medicines for injections, including those given into a vein.

What active substances are being studied?

Active substances that are officially approved for use in the European Union and considered safe by EMA. These substances are already on the market and may be tested for new conditions or populations.

Vosoritide
Vosoritide is a substance that stimulates bone growth to increase height in children with achondroplasia.
Water For Injection
Water for Injection is sterile purified water used to dilute and prepare substances for safe injection into the body.

What similar medicines might you know?

Don't worry if you don't know the medicines tested in the trial. There is a chance you have heard about the similar medicines. Check the list below to see if you are familiar with any of them.

Acetilcolina Cusí
Acqua Per Preparazioni Iniettabil Baxter
Acqua Per Preparazioni Iniettabili B. Braun

Where is the clinical trial being conducted?

Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.

Loading map...
Centre Hospitalier Universitaire De Toulouse
Endocrinologie, Gynécologie et Génétique Médicale
Blagnac, France
Hospices Civils De Lyon
Service de Génétique, Centre de Référence Maladies Osseuses Constitutionelles
Bron, France
Centre Hospitalier Regional De Marseille
Pediatric Multidisciplinary unit
Marseille, France
Sponsor: Biomarin Pharmaceutical Inc.
Last updated: Nov 26, 2025

Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.