New hope for babies with rare dwarfism: daily shot may boost growth

NCT ID NCT07126262

Recruiting now Disease control Sponsor: BioMarin Pharmaceutical Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a daily injection called vosoritide in infants and toddlers (0 to 3 years old) with hypochondroplasia, a genetic condition that causes short stature. The goal is to see if the drug safely improves growth over 52 weeks. About 60 children will receive either vosoritide or a placebo, and their height, bone density, and side effects will be tracked.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

hypochondroplasia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HYPOCHONDROPLASIA

Contacts and locations

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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

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

Chicago, Illinois, 60611, United States
Benioff Children's Hospital - Oakland
RECRUITING

Oakland, California, 94609, United States
Cedars-Sinai Medical Center
NOT_YET_RECRUITING

Los Angeles, California, 90048, United States
Children's Health Queensland Hospital and Health Service
NOT_YET_RECRUITING

South Brisbane, Queensland, 4101, Australia
Children's National Medical Center
RECRUITING

Washington D.C., District of Columbia, 20010, United States
Children's Wisconsin - Fox Valley Hospital
NOT_YET_RECRUITING

Neenah, Wisconsin, 54956, United States
Cook Children's Endocrinology
NOT_YET_RECRUITING

Fort Worth, Texas, 76104, United States
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
NOT_YET_RECRUITING

Roma, Rome, 00168, Italy
Great Ormond Street Hospital
NOT_YET_RECRUITING

London, England, WC1N3BH, United Kingdom
Hospices Civils de Lyon - Hôpital Femme Mère Enfant
NOT_YET_RECRUITING

Bron, Rhone-Alps, 69500, France
Hôpital Bicêtre
RECRUITING

Le Kremlin-Bicêtre, Paris, 94270, France
IRCCS Istituto Giannina Gaslini
RECRUITING

Genova, 16147, Italy
Institute of Science Tokyo Hospital
RECRUITING

Tokyo, 113-8519, Japan
Kumamoto University Hospital
RECRUITING

Kumamoto, 860-8556, Japan
Myriad Trials
NOT_YET_RECRUITING

London, England, W1W 5DT, United Kingdom
Nihon University Itabashi Hospital
RECRUITING

Tokyo, 173-8610, Japan
Osaka Women's and Children's Hospital
RECRUITING

Osaka, 94-1101, Japan
Phoenix Children's Hospital - Thomas Campus (Main)
RECRUITING

Phoenix, Arizona, 85016, United States
Royal Children's Hospital Melbourne
RECRUITING

Parkville, Victoria, 3052, Australia
The Johns Hopkins University School of Medicine
RECRUITING

Baltimore, Maryland, 21205, United States
Tottori University Hospital
RECRUITING

Tottori, 683-0854, Japan
Uniklinik Köln
RECRUITING

Cologne, North Rhine-Westphalia, 50937, Germany
University of Minneasota Masonic Children's Hospital
RECRUITING

Minneapolis, Minnesota, 55454, United States
University of Missouri
RECRUITING

Columbia, Missouri, 65211, United States
Universitätskinderklinik Magdeburg
NOT_YET_RECRUITING

Magdeburg, Saxony-Anhalt, 39120, Germany
Universitätsklinikum des Saarlandes
RECRUITING

Homburg, Saarland, 66421, Germany