RecruitingPhase 2NCT07126262

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

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

Sponsor

BioMarin Pharmaceutical

Enrollment

60 participants

Start Date

Jul 30, 2025

Study Type

INTERVENTIONAL

Conditions

Hypochondroplasia

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

Min Age: 0 MonthsMax Age: 36 Months

Plain Language Summary

Simplified for easier understanding

This study tests vosoritide — a daily injection medication already approved for achondroplasia — in infants and very young children (under 3 years old) with hypochondroplasia (HCH), a related but milder genetic form of short stature caused by a different mutation in the same gene. The goal is to find out whether early treatment can improve growth and reduce height deficits during a critical developmental window. Infants and children from birth to under 36 months with a genetic diagnosis of HCH and a height significantly below average for their age may be eligible, while those with other causes of short stature, unstable medical conditions, or prior growth hormone treatment are excluded. Participation involves daily subcutaneous (under-the-skin) injections of either vosoritide or placebo for 52 weeks, with regular monitoring of growth, safety, and development. This summary was generated with AI assistance to help patients understand the study in plain language.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGVosoritide

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

DRUGPlacebo

Subcutaneous injection of recommended dose of placebo

Locations(26)

Phoenix Children's Hospital - Thomas Campus (Main)

Phoenix, Arizona, United States

Cedars-Sinai Medical Center

Los Angeles, California, United States

Benioff Children's Hospital - Oakland

Oakland, California, United States

Children's National Medical Center

Washington D.C., District of Columbia, United States

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

The Johns Hopkins University School of Medicine

Baltimore, Maryland, United States

University of Minneasota Masonic Children's Hospital

Minneapolis, Minnesota, United States

University of Missouri

Columbia, Missouri, United States

Cook Children's Endocrinology

Fort Worth, Texas, United States

Children's Wisconsin - Fox Valley Hospital

Neenah, Wisconsin, United States

Children's Health Queensland Hospital and Health Service

South Brisbane, Queensland, Australia

Royal Children's Hospital Melbourne

Parkville, Victoria, Australia

Hôpital Bicêtre

Le Kremlin-Bicêtre, Paris, France

Hospices Civils de Lyon - Hôpital Femme Mère Enfant

Bron, Rhone-Alps, France

Uniklinik Köln

Cologne, North Rhine-Westphalia, Germany

Universitätsklinikum des Saarlandes

Homburg, Saarland, Germany

Universitätskinderklinik Magdeburg

Magdeburg, Saxony-Anhalt, Germany

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Roma, Rome, Italy

IRCCS Istituto Giannina Gaslini

Genova, Italy

Kumamoto University Hospital

Kumamoto, Japan

Osaka Women's and Children's Hospital

Osaka, Japan

Institute of Science Tokyo Hospital

Tokyo, Japan

Nihon University Itabashi Hospital

Tokyo, Japan

Tottori University Hospital

Tottori, Japan

Myriad Trials

London, England, United Kingdom

Great Ormond Street Hospital

London, England, United Kingdom

View Full Details on ClinicalTrials.gov

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NCT07126262

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