A Multicenter Multinational Observational Study of Children With Hypochondroplasia

NCT ID: NCT06212947

Last Updated: 2025-04-03

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

400 participants

Study Classification

OBSERVATIONAL

Study Start Date

2023-11-27

Study Completion Date

2043-12-31

Brief Summary

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

Detailed Description

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Conditions

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Hypochondroplasia

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Children with Hypochondroplasia

Children with Hypochondroplasia

No interventions assigned to this group

Eligibility Criteria

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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
Maximum Eligible Age

15 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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BioMarin Pharmaceutical

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director, MD

Role: STUDY_DIRECTOR

BioMarin Pharmaceutical

Locations

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Phoenix Children's Hospital

Phoenix, Arizona, United States

Site Status RECRUITING

Cedars-Sinai Medical Center

Los Angeles, California, United States

Site Status NOT_YET_RECRUITING

UCSF Benioff Children's Hospital

Oakland, California, United States

Site Status RECRUITING

Nemours Alfred I. Dupont Hospital for Children

Wilmington, Delaware, United States

Site Status RECRUITING

Children's National Health System

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

Site Status RECRUITING

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

Chicago, Illinois, United States

Site Status RECRUITING

The Johns Hopkins University School of Medicine

Baltimore, Maryland, United States

Site Status NOT_YET_RECRUITING

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status RECRUITING

University of Minnesota Masonic Children's Hospital

Minneapolis, Minnesota, United States

Site Status RECRUITING

University of Missouri

Columbia, Missouri, United States

Site Status RECRUITING

Washington University School of Medicine in St. Louis

St Louis, Missouri, United States

Site Status RECRUITING

Mount Sinai Kravis Children's Hospital

New York, New York, United States

Site Status RECRUITING

Akron Children's Hospital

Akron, Ohio, United States

Site Status NOT_YET_RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status NOT_YET_RECRUITING

Cook Children's Endocrinology

Fort Worth, Texas, United States

Site Status RECRUITING

Medical College of Wisconsin, Children's Hospital

Milwaukee, Wisconsin, United States

Site Status RECRUITING

Hospital de Pediatria SAMIC Prof. Dr. Juan P Garrahan

Buenos Aires, , Argentina

Site Status NOT_YET_RECRUITING

Royal Children's Hospital Melbourne

Parkville, Victoria, Australia

Site Status RECRUITING

Hospital de Clinicas de Porto Algre

Porto Alegre, Rio Grande Do Sol, Brazil

Site Status NOT_YET_RECRUITING

Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto

Ribeirão Preto, São Paulo, Brazil

Site Status NOT_YET_RECRUITING

Stollery Children's Hospital

Edmonton, Alberta, Canada

Site Status RECRUITING

SickKids - The Hospital for Sick Children

Toronto, Ontario, Canada

Site Status RECRUITING

Centre Hospitalier Universitaire Sainte-Justine

Montreal, Quebec, Canada

Site Status NOT_YET_RECRUITING

Hospices Civils de Lyon - Hôpital Femme Mère Enfant

Lyon, , France

Site Status RECRUITING

Hopital de la Timone

Marseille, , France

Site Status RECRUITING

Hôpital Necker-Enfants Malades

Paris, , France

Site Status RECRUITING

CHU de Toulouse - Hopital des Efants

Toulouse, , France

Site Status RECRUITING

Uniklinik Koln

Cologne, , Germany

Site Status RECRUITING

Universitatskinderklinik Magdeburg

Magdeburg, , Germany

Site Status RECRUITING

Istituto Giannina Gaslini

Genoa, Liguria, Italy

Site Status RECRUITING

Ospedale Pediatrico Bambino Gesù

Roma, Rome, Italy

Site Status NOT_YET_RECRUITING

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, , Italy

Site Status RECRUITING

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Rome, , Italy

Site Status RECRUITING

Kumamoto University Hospital

Kumamoto, Kumamoto, Japan

Site Status RECRUITING

Osaka University Hospital

Suita, Osaka, Japan

Site Status RECRUITING

Tottori University Hospital

Yonago, Tottori, Japan

Site Status RECRUITING

Tokushima University Hospital

Tokushima, , Japan

Site Status NOT_YET_RECRUITING

Institute of Science Tokyo Hospital

Tokyo, , Japan

Site Status RECRUITING

Hospital Universitario La Paz

Madrid, , Spain

Site Status RECRUITING

Vithas Hospital San Jose

Vitoria-Gasteiz, , Spain

Site Status RECRUITING

Great Ormond Street Hospital for Children NHS Foundation Trust

London, England, United Kingdom

Site Status RECRUITING

Myriad Trials Ltd

London, England, United Kingdom

Site Status NOT_YET_RECRUITING

NHS Greater Glasgow and Clyde

Glasgow, Scottland, United Kingdom

Site Status RECRUITING

Countries

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United States Argentina Australia Brazil Canada France Germany Italy Japan Spain United Kingdom

Central Contacts

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Trial Specialist

Role: CONTACT

1-800-983-4587

Other Identifiers

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111-902

Identifier Type: -

Identifier Source: org_study_id

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