A Multicenter Multinational Observational Study of Children with Hypochondroplasia

Last updated: February 6, 2025
Sponsor: BioMarin Pharmaceutical
Overall Status: Active - Recruiting

Phase

N/A

Condition

Severe Short Stature

Treatment

N/A

Clinical Study ID

NCT06212947
111-902
  • Ages < 15
  • All Genders

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 Criteria

Inclusion

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

Exclusion Criteria:

  • Have a diagnosis of another genetic short stature condition other thanHypochondroplasia or a genetic variant known to cause another genetic syndromeassociated with short stature

  • Received an investigational product or medical device within 6 months before theScreening visit

Study Design

Total Participants: 400
Study Start date:
November 27, 2023
Estimated Completion Date:
December 31, 2043

Connect with a study center

  • Hospital de Pediatria SAMIC Prof. Dr. Juan P Garrahan

    Buenos Aires,
    Argentina

    Site Not Available

  • Royal Children's Hospital Melbourne

    Parkville, Victoria 3052
    Australia

    Active - Recruiting

  • Hospital de Clinicas de Porto Algre

    Porto Alegre, Rio Grande do Sol
    Brazil

    Site Not Available

  • Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto

    Ribeirão Preto, Sao Paulo
    Brazil

    Site Not Available

  • Stollery Children's Hospital

    Edmonton, Alberta
    Canada

    Active - Recruiting

  • SickKids - The Hospital for Sick Children

    Toronto, Ontario
    Canada

    Active - Recruiting

  • Centre Hospitalier Universitaire Sainte-Justine

    Montréal, Quebec
    Canada

    Site Not Available

  • Hospices Civils de Lyon - Hôpital Femme Mère Enfant

    Lyon,
    France

    Active - Recruiting

  • Hopital de la Timone

    Marseille,
    France

    Active - Recruiting

  • Hôpital Necker-Enfants Malades

    Paris,
    France

    Active - Recruiting

  • CHU de Toulouse - Hopital des Efants

    Toulouse,
    France

    Active - Recruiting

  • Uniklinik Koln

    Köln,
    Germany

    Active - Recruiting

  • Universitatskinderklinik Magdeburg

    Magdeburg,
    Germany

    Active - Recruiting

  • Istituto Giannina Gaslini

    Genoa, Liguria
    Italy

    Active - Recruiting

  • Ospedale Pediatrico Bambino Gesù

    Roma, Rome
    Italy

    Site Not Available

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

    Milan,
    Italy

    Active - Recruiting

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    Rome,
    Italy

    Active - Recruiting

  • Osaka University Hospital

    Suita, Osaka
    Japan

    Active - Recruiting

  • Tottori University Hospital

    Yonago, Tottori
    Japan

    Active - Recruiting

  • Kumamoto University Hospital

    Kumamoto,
    Japan

    Active - Recruiting

  • Tokushima University Hospital

    Tokushima,
    Japan

    Site Not Available

  • Institute of Science Tokyo Hospital

    Tokyo,
    Japan

    Active - Recruiting

  • Tokyo Medical and Dental University Hospital

    Tokyo,
    Japan

    Active - Recruiting

  • Hospital Universitario La Paz

    Madrid,
    Spain

    Active - Recruiting

  • Vithas Hospital San Jose

    Vitoria,
    Spain

    Active - Recruiting

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    London, England
    United Kingdom

    Active - Recruiting

  • Myriad Trials Ltd

    London, England
    United Kingdom

    Site Not Available

  • NHS Greater Glasgow and Clyde

    Glasgow, Scottland
    United Kingdom

    Active - Recruiting

  • Phoenix Children's Hospital

    Phoenix, Arizona 85016
    United States

    Active - Recruiting

  • Cedars-Sinai Medical Center

    Los Angeles, California 90048
    United States

    Site Not Available

  • UCSF Benioff Children's Hospital

    Oakland, California 94609
    United States

    Active - Recruiting

  • Nemours Alfred I. Dupont Hospital for Children

    Wilmington, Delaware 19803
    United States

    Active - Recruiting

  • Children's National Health System

    Washington, District of Columbia 20010
    United States

    Active - Recruiting

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

    Chicago, Illinois 60611
    United States

    Active - Recruiting

  • The Johns Hopkins University School of Medicine

    Baltimore, Maryland 21218
    United States

    Site Not Available

  • Boston Children's Hospital

    Boston, Massachusetts 02115
    United States

    Active - Recruiting

  • University of Minnesota Masonic Children's Hospital

    Minneapolis, Minnesota 55454
    United States

    Active - Recruiting

  • University of Missouri

    Columbia, Missouri 65212
    United States

    Active - Recruiting

  • Washington University School of Medicine in St. Louis

    Saint Louis, Missouri 63110
    United States

    Active - Recruiting

  • Mount Sinai Kravis Children's Hospital

    New York, New York 10029
    United States

    Active - Recruiting

  • Akron Children's Hospital

    Akron, Ohio 44307
    United States

    Site Not Available

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio 45229
    United States

    Site Not Available

  • Nationwide Children's Hospital

    Columbus, Ohio 43205
    United States

    Site Not Available

  • Cook Children's Endocrinology

    Fort Worth, Texas 76104
    United States

    Active - Recruiting

  • Seattle Children's Hospital

    Seattle, Washington 98105
    United States

    Site Not Available

  • Medical College of Wisconsin, Children's Hospital

    Milwaukee, Wisconsin 53226
    United States

    Active - Recruiting

  • Children's Wisconsin - Fox Valley Hospital

    Neenah, Wisconsin 54956
    United States

    Site Not Available

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