RecruitingNCT06212947

A Multicenter Multinational Observational Study of Children With Hypochondroplasia


Sponsor

BioMarin Pharmaceutical

Enrollment

400 participants

Start Date

Nov 27, 2023

Study Type

OBSERVATIONAL

Conditions

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

Max Age: 15 Years

Inclusion Criteria2

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

Exclusion Criteria2

  • 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

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Locations(43)

Phoenix Children's Hospital

Phoenix, Arizona, United States

Cedars-Sinai Medical Center

Los Angeles, California, United States

UCSF Benioff Children's Hospital

Oakland, California, United States

Nemours Alfred I. Dupont Hospital for Children

Wilmington, Delaware, United States

Children's National Health System

Washington D.C., District of Columbia, United States

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

Chicago, Illinois, United States

The Johns Hopkins University School of Medicine

Baltimore, Maryland, United States

Boston Children's Hospital

Boston, Massachusetts, United States

University of Minnesota Masonic Children's Hospital

Minneapolis, Minnesota, United States

University of Missouri

Columbia, Missouri, United States

Washington University School of Medicine in St. Louis

St Louis, Missouri, United States

Mount Sinai Kravis Children's Hospital

New York, New York, United States

Akron Children's Hospital

Akron, Ohio, United States

Nationwide Children's Hospital

Columbus, Ohio, United States

Cook Children's Endocrinology

Fort Worth, Texas, United States

Medical College of Wisconsin, Children's Hospital

Milwaukee, Wisconsin, United States

Hospital de Pediatria SAMIC Prof. Dr. Juan P Garrahan

Buenos Aires, Argentina

Royal Children's Hospital Melbourne

Parkville, Victoria, 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

View Full Details on ClinicalTrials.gov

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NCT06212947


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