A Multicenter Multinational Observational Study of Children With Hypochondroplasia

Study Purpose

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

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Observational
Eligible Ages N/A - 15 Years
Gender All
More Inclusion & Exclusion Criteria

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

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT06212947
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

BioMarin Pharmaceutical
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Medical Director, MD
Principal Investigator Affiliation BioMarin Pharmaceutical
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Industry
Overall Status Recruiting
Countries Argentina, Australia, Brazil, Canada, France, Germany, Italy, Japan, Spain, United Kingdom, United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Hypochondroplasia
Arms & Interventions

Arms

: Children with Hypochondroplasia

Children with Hypochondroplasia

Interventions

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Phoenix Children's Hospital, Phoenix, Arizona

Status

Not yet recruiting

Address

Phoenix Children's Hospital

Phoenix, Arizona, 85016

Cedars-Sinai Medical Center, Los Angeles, California

Status

Not yet recruiting

Address

Cedars-Sinai Medical Center

Los Angeles, California, 90048

UCSF Benioff Children's Hospital, Oakland, California

Status

Recruiting

Address

UCSF Benioff Children's Hospital

Oakland, California, 94609

Wilmington, Delaware

Status

Not yet recruiting

Address

Nemours Alfred I. Dupont Hospital for Children

Wilmington, Delaware, 19803

Children's National Health System, Washington, District of Columbia

Status

Recruiting

Address

Children's National Health System

Washington, District of Columbia, 20010

Chicago, Illinois

Status

Recruiting

Address

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

Chicago, Illinois, 60611

Baltimore, Maryland

Status

Not yet recruiting

Address

The Johns Hopkins University School of Medicine

Baltimore, Maryland, 21218

Boston Children's Hospital, Boston, Massachusetts

Status

Recruiting

Address

Boston Children's Hospital

Boston, Massachusetts, 02115

Minneapolis, Minnesota

Status

Not yet recruiting

Address

University of Minnesota Masonic Children's Hospital

Minneapolis, Minnesota, 55454

University of Missouri, Columbia, Missouri

Status

Recruiting

Address

University of Missouri

Columbia, Missouri, 65212

Saint Louis, Missouri

Status

Not yet recruiting

Address

Washington University School of Medicine in St. Louis

Saint Louis, Missouri, 63110

Mount Sinai Kravis Children's Hospital, New York, New York

Status

Not yet recruiting

Address

Mount Sinai Kravis Children's Hospital

New York, New York, 10029

Akron Children's Hospital, Akron, Ohio

Status

Not yet recruiting

Address

Akron Children's Hospital

Akron, Ohio, 44307

Nationwide Children's Hospital, Columbus, Ohio

Status

Not yet recruiting

Address

Nationwide Children's Hospital

Columbus, Ohio, 43205

Cook Children's Endocrinology, Fort Worth, Texas

Status

Not yet recruiting

Address

Cook Children's Endocrinology

Fort Worth, Texas, 76104

Seattle Children's Hospital, Seattle, Washington

Status

Not yet recruiting

Address

Seattle Children's Hospital

Seattle, Washington, 98105

Milwaukee, Wisconsin

Status

Recruiting

Address

Medical College of Wisconsin, Children's Hospital

Milwaukee, Wisconsin, 53226

International Sites

Buenos Aires, Argentina

Status

Not yet recruiting

Address

Hospital de Pediatria SAMIC Prof. Dr. Juan P Garrahan

Buenos Aires, ,

Royal Children's Hospital Melbourne, Parkville, Victoria, Australia

Status

Recruiting

Address

Royal Children's Hospital Melbourne

Parkville, Victoria, 3052

Hospital de Clinicas de Porto Algre, Porto Alegre, Rio Grande Do Sol, Brazil

Status

Not yet recruiting

Address

Hospital de Clinicas de Porto Algre

Porto Alegre, Rio Grande Do Sol,

Ribeirão Preto, Sao Paulo, Brazil

Status

Not yet recruiting

Address

Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto

Ribeirão Preto, Sao Paulo,

Stollery Children's Hospital, Edmonton, Alberta, Canada

Status

Recruiting

Address

Stollery Children's Hospital

Edmonton, Alberta,

Toronto, Ontario, Canada

Status

Not yet recruiting

Address

SickKids - The Hospital for Sick Children

Toronto, Ontario,

Montréal, Quebec, Canada

Status

Not yet recruiting

Address

Centre Hospitalier Universitaire Sainte-Justine

Montréal, Quebec,

Lyon, France

Status

Not yet recruiting

Address

Hospices Civils de Lyon - Hôpital Femme Mère Enfant

Lyon, ,

Hopital de la Timone, Marseille, France

Status

Not yet recruiting

Address

Hopital de la Timone

Marseille, ,

Hôpital Necker-Enfants Malades, Paris, France

Status

Recruiting

Address

Hôpital Necker-Enfants Malades

Paris, ,

CHU de Toulouse - Hopital des Efants, Toulouse, France

Status

Recruiting

Address

CHU de Toulouse - Hopital des Efants

Toulouse, ,

Uniklinik Koln, Köln, Germany

Status

Not yet recruiting

Address

Uniklinik Koln

Köln, ,

Universitatskinderklinik Magdeburg, Magdeburg, Germany

Status

Not yet recruiting

Address

Universitatskinderklinik Magdeburg

Magdeburg, ,

Istituto Giannina Gaslini, Genoa, Liguria, Italy

Status

Not yet recruiting

Address

Istituto Giannina Gaslini

Genoa, Liguria,

Ospedale Pediatrico Bambino Gesù, Roma, Rome, Italy

Status

Not yet recruiting

Address

Ospedale Pediatrico Bambino Gesù

Roma, Rome,

Milan, Italy

Status

Not yet recruiting

Address

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, ,

Rome, Italy

Status

Not yet recruiting

Address

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Rome, ,

Osaka University Hospital, Suita, Osaka, Japan

Status

Recruiting

Address

Osaka University Hospital

Suita, Osaka,

Tottori University Hospital, Yonago, Tottori, Japan

Status

Not yet recruiting

Address

Tottori University Hospital

Yonago, Tottori,

Kumamoto University Hospital, Kumamoto, Japan

Status

Not yet recruiting

Address

Kumamoto University Hospital

Kumamoto, ,

Tokushima University Hospital, Tokushima, Japan

Status

Not yet recruiting

Address

Tokushima University Hospital

Tokushima, ,

Tokyo, Japan

Status

Not yet recruiting

Address

Tokyo Medical and Dental University Hospital

Tokyo, ,

Hospital Universitario La Paz, Madrid, Spain

Status

Not yet recruiting

Address

Hospital Universitario La Paz

Madrid, ,

Vithas Hospital San Jose, Vitoria, Spain

Status

Not yet recruiting

Address

Vithas Hospital San Jose

Vitoria, ,

London, England, United Kingdom

Status

Not yet recruiting

Address

Great Ormond Street Hospital for Children NHS Foundation Trust

London, England,

Myriad Trials Ltd, London, England, United Kingdom

Status

Not yet recruiting

Address

Myriad Trials Ltd

London, England,

NHS Greater Glasgow and Clyde, Glasgow, Scottland, United Kingdom

Status

Not yet recruiting

Address

NHS Greater Glasgow and Clyde

Glasgow, Scottland,