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NCT06212947Recruiting

A Multicenter Multinational Observational Study of Children With Hypochondroplasia

BioMarin Pharmaceutical

Start Date

11/27/2023

Completion Date

12/31/2043

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 Criteria

Age Range: No minimum to 15 years

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

Interested in This Trial?

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Conditions

Hypochondroplasia

Locations

Phoenix Children's Hospital

Phoenix, Arizona 85016

United States

Cedars-Sinai Medical Center

Los Angeles, California 90048

United States

UCSF Benioff Children's Hospital

Oakland, California 94609

United States

Nemours Alfred I. Dupont Hospital for Children

Wilmington, Delaware 19803

United States

Children's National Health System

Washington D.C., District of Columbia 20010

United States

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

Chicago, Illinois 60611

United States

The Johns Hopkins University School of Medicine

Baltimore, Maryland 21218

United States

Boston Children's Hospital

Boston, Massachusetts 02115

United States

University of Minnesota Masonic Children's Hospital

Minneapolis, Minnesota 55454

United States

University of Missouri

Columbia, Missouri 65212

United States

Washington University School of Medicine in St. Louis

St Louis, Missouri 63110

United States

Mount Sinai Kravis Children's Hospital

New York, New York 10029

United States

Akron Children's Hospital

Akron, Ohio 44307

United States

Nationwide Children's Hospital

Columbus, Ohio 43205

United States

Cook Children's Endocrinology

Fort Worth, Texas 76104

United States

Medical College of Wisconsin, Children's Hospital

Milwaukee, Wisconsin 53226

United States

Hospital de Pediatria SAMIC Prof. Dr. Juan P Garrahan

Buenos Aires,

Argentina

Royal Children's Hospital Melbourne

Parkville, Victoria 3052

Australia

Hospital de Clinicas de Porto Algre

Porto Alegre, Rio Grande Do Sol

Brazil

Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto

Ribeirão Preto, São Paulo

Brazil

Stollery Children's Hospital

Edmonton, Alberta

Canada

SickKids - The Hospital for Sick Children

Toronto, Ontario

Canada

Centre Hospitalier Universitaire Sainte-Justine

Montreal, Quebec

Canada

Hospices Civils de Lyon - Hôpital Femme Mère Enfant

Lyon,

France

Hopital de la Timone

Marseille,

France

Hôpital Necker-Enfants Malades

Paris,

France

CHU de Toulouse - Hopital des Efants

Toulouse,

France

Uniklinik Koln

Cologne,

Germany

Universitatskinderklinik Magdeburg

Magdeburg,

Germany

Istituto Giannina Gaslini

Genoa, Liguria

Italy

Ospedale Pediatrico Bambino Gesù

Roma, Rome

Italy

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan,

Italy

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Rome,

Italy

Kumamoto University Hospital

Kumamoto, Kumamoto

Japan

Osaka University Hospital

Suita, Osaka

Japan

Tottori University Hospital

Yonago, Tottori

Japan

Tokushima University Hospital

Tokushima,

Japan

Institute of Science Tokyo Hospital

Tokyo,

Japan

Hospital Universitario La Paz

Madrid,

Spain

Vithas Hospital San Jose

Vitoria-Gasteiz,

Spain

Great Ormond Street Hospital for Children NHS Foundation Trust

London, England

United Kingdom

Myriad Trials Ltd

London, England

United Kingdom

NHS Greater Glasgow and Clyde

Glasgow, Scottland

United Kingdom