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Clinical Trials in France / NCT06212947
Recruiting Observational

A Multicenter Multinational Observational Study of Children With Hypochondroplasia

NCT06212947 · tracked via the Priya Life Science France 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
No
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)

FacilityCityRegionStatus
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.

More BioMarin Pharmaceutical trials in France

A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401) Enrolling by invitation
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months Recruiting
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia Active, not recruiting
Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia Enrolling by invitation
A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment Active, not recruiting

Other trials for the same condition

Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia Enrolling by invitation
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months Recruiting
An Interventional Study of Infigratinib in Children With Hypochondroplasia Enrolling by invitation
Prospective Clinical Assessment Study in Children With Hypochondroplasia Recruiting
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia Active, not recruiting
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 France