A Multicenter Multinational Observational Study of Children With Hypochondroplasia

Recruiting now

Conditions studied: Hypochondroplasia

In brief

This study will assess growth over time and the clinical course of HCH in children by collecting growth measurements and other variables of interest.

Key facts

Study ID

NCT06212947

Run by

BioMarin Pharmaceutical

People needed

400

Starts

2023-11-27

Expected to finish

2043-12-31

Last updated by the study team

2025-04-03

Who can join

Age: any, up to 15. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Participants must be ≤ 15 years old at the time of signing the informed consent
• Participants must have genetic confirmation of Hypochondroplasia diagnosis

You may not qualify if…

• Have a diagnosis of another genetic short stature condition other than Hypochondroplasia or a genetic variant known to cause another genetic syndrome associated with short stature
• Received an investigational product or medical device within 6 months before the Screening visit

Where it is running

• NHS Greater Glasgow and Clyde — Glasgow, Scottland, United Kingdom (enrolling)
• Istituto Giannina Gaslini — Genoa, Liguria, Italy (enrolling)
• Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico — Milan, Italy (enrolling)
• Fondazione Policlinico Universitario Agostino Gemelli IRCCS — Rome, Italy (enrolling)
• Kumamoto University Hospital — Kumamoto, Kumamoto, Japan (enrolling)
• Osaka University Hospital — Suita, Osaka, Japan (enrolling)
• Tottori University Hospital — Yonago, Tottori, Japan (enrolling)
• Institute of Science Tokyo Hospital — Tokyo, Japan (enrolling)
• Hospital Universitario La Paz — Madrid, Spain (enrolling)
• Vithas Hospital San Jose — Vitoria-Gasteiz, Spain (enrolling)
• Great Ormond Street Hospital for Children NHS Foundation Trust — London, England, United Kingdom (enrolling)
• Phoenix Children's Hospital — Phoenix, Arizona, United States (enrolling)
• UCSF Benioff Children's Hospital — Oakland, California, United States (enrolling)
• Nemours Alfred I. Dupont Hospital for Children — Wilmington, Delaware, United States (enrolling)
• Children's National Health System — Washington D.C., District of Columbia, United States (enrolling)
• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (enrolling)
• Boston Children's Hospital — Boston, Massachusetts, United States (enrolling)
• University of Minnesota Masonic Children's Hospital — Minneapolis, Minnesota, United States (enrolling)
• University of Missouri — Columbia, Missouri, United States (enrolling)
• Washington University School of Medicine in St. Louis — St Louis, Missouri, United States (enrolling)
• Mount Sinai Kravis Children's Hospital — New York, New York, United States (enrolling)
• Cook Children's Endocrinology — Fort Worth, Texas, United States (enrolling)
• Medical College of Wisconsin, Children's Hospital — Milwaukee, Wisconsin, United States (enrolling)
• Royal Children's Hospital Melbourne — Parkville, Victoria, Australia (enrolling)
• Stollery Children's Hospital — Edmonton, Alberta, Canada (enrolling)

Full record on ClinicalTrials.gov

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