A Multicenter Multinational Observational Study of Children With Hypochondroplasia

NCT06212947 · tracked via the Priya Life Science UK tracker

Sponsor

BioMarin Pharmaceutical

Phase

Observational

Started

2023-11-27

Last updated

2025-04-03

Condition(s) studied

Hypochondroplasia

Study summary

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

Eligibility

Sex

ALL

Min age

—

Max age

15 Years

Healthy volunteers

Inclusion Criteria: * Participants must be ≤ 15 years old at the time of signing the informed consent * Participants must have genetic confirmation of Hypochondroplasia diagnosis Exclusion Criteria: * 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

Primary outcome measure(s)

Change in annualized growth velocity (AGV) — Every 6 months through end of study, up to 15 years
Change in Height Z-score — Every 6 months through end of study, up to 15 years
Change in Height — Every 6 months through end of study, up to 15 years
Change in BMI — Every 6 months through end of study, up to 15 years
Change in BMI Z-score — Every 6 months through end of study, up to 15 years
Change in ratios of upper to lower body segments — Every 6 months through end of study, up to 15 years
Change in ratios of upper and lower leg length ratio — Every 6 months through end of study, up to 15 years
Change in ratio of arms span to standing height ratio — Every 6 months through end of study, up to 15 years

Trial sites (43)

Facility	City	Region	Status
Phoenix Children's Hospital	Phoenix	Arizona	Recruiting
Cedars-Sinai Medical Center	Los Angeles	California	Not Yet Recruiting
UCSF Benioff Children's Hospital	Oakland	California	Recruiting
Nemours Alfred I. Dupont Hospital for Children	Wilmington	Delaware	Recruiting
Children's National Health System	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	Not Yet Recruiting
Boston Children's Hospital	Boston	Massachusetts	Recruiting
University of Minnesota Masonic Children's Hospital	Minneapolis	Minnesota	Recruiting
University of Missouri	Columbia	Missouri	Recruiting
Washington University School of Medicine in St. Louis	St Louis	Missouri	Recruiting
Mount Sinai Kravis Children's Hospital	New York	New York	Recruiting
Akron Children's Hospital	Akron	Ohio	Not Yet Recruiting
Nationwide Children's Hospital	Columbus	Ohio	Not Yet Recruiting
Cook Children's Endocrinology	Fort Worth	Texas	Recruiting
Medical College of Wisconsin, Children's Hospital	Milwaukee	Wisconsin	Recruiting
Hospital de Pediatria SAMIC Prof. Dr. Juan P Garrahan	Buenos Aires	Argentina	Not Yet Recruiting
Royal Children's Hospital Melbourne	Parkville	Victoria	Recruiting
Hospital de Clinicas de Porto Algre	Porto Alegre	Rio Grande Do Sol	Not Yet Recruiting
Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto	Ribeirão Preto	São Paulo	Not Yet Recruiting
Stollery Children's Hospital	Edmonton	Alberta	Recruiting
SickKids - The Hospital for Sick Children	Toronto	Ontario	Recruiting
Centre Hospitalier Universitaire Sainte-Justine	Montreal	Quebec	Not Yet Recruiting
Hospices Civils de Lyon - Hôpital Femme Mère Enfant	Lyon	France	Recruiting
Hopital de la Timone	Marseille	France	Recruiting
Hôpital Necker-Enfants Malades	Paris	France	Recruiting
CHU de Toulouse - Hopital des Efants	Toulouse	France	Recruiting
Uniklinik Koln	Cologne	Germany	Recruiting
Universitatskinderklinik Magdeburg	Magdeburg	Germany	Recruiting
Istituto Giannina Gaslini	Genoa	Liguria	Recruiting
Ospedale Pediatrico Bambino Gesù	Roma	Rome	Not Yet Recruiting
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico	Milan	Italy	Recruiting
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy	Recruiting
Kumamoto University Hospital	Kumamoto	Kumamoto	Recruiting
Osaka University Hospital	Suita	Osaka	Recruiting
Tottori University Hospital	Yonago	Tottori	Recruiting
Tokushima University Hospital	Tokushima	Japan	Not Yet Recruiting
Institute of Science Tokyo Hospital	Tokyo	Japan	Recruiting
Hospital Universitario La Paz	Madrid	Spain	Recruiting
Vithas Hospital San Jose	Vitoria-Gasteiz	Spain	Recruiting

+ 3 more sites — see the full list on the official registry below.

Official registry record

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 NCT06212947 on ClinicalTrials.gov ↗ ← All trials in the UK