A Multicenter Multinational Observational Study of Children With Hypochondroplasia

Hypochondroplasia Clinical Trial

Official title:

A Multicenter Multinational Observational Study of Children With Hypochondroplasia

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06212947
• Other study ID #: 111-902
• Status: Recruiting
• Start date: November 27, 2023
• Est. completion date: December 31, 2043

Study information

• Verified date: June 2024
• Source: BioMarin Pharmaceutical
• Contact: Trial Specialist
• Phone: 1-800-938-4587
• Email: medinfo[@]bmrn.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 400
• Est. completion date: December 31, 2043
• Est. primary completion date: December 31, 2043
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 15 Years

Eligibility

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

Related Conditions & MeSH terms

• Dwarfism
• Hypochondroplasia
• Lordosis

Locations

Country	Name	City	State
Argentina	Hospital de Pediatria SAMIC Prof. Dr. Juan P Garrahan	Buenos Aires
Australia	Royal Children's Hospital Melbourne	Parkville	Victoria
Brazil	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	Sao Paulo
Canada	Stollery Children's Hospital	Edmonton	Alberta
Canada	Centre Hospitalier Universitaire Sainte-Justine	Montréal	Quebec
Canada	SickKids - The Hospital for Sick Children	Toronto	Ontario
France	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
Germany	Uniklinik Koln	Köln
Germany	Universitatskinderklinik Magdeburg	Magdeburg
Italy	Istituto Giannina Gaslini	Genoa	Liguria
Italy	Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico	Milan
Italy	Ospedale Pediatrico Bambino Gesù	Roma	Rome
Italy	Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome
Japan	Kumamoto University Hospital	Kumamoto
Japan	Osaka University Hospital	Suita	Osaka
Japan	Tokushima University Hospital	Tokushima
Japan	Tokyo Medical and Dental University Hospital	Tokyo
Japan	Tottori University Hospital	Yonago	Tottori
Spain	Hospital Universitario La Paz	Madrid
Spain	Vithas Hospital San Jose	Vitoria
United Kingdom	NHS Greater Glasgow and Clyde	Glasgow	Scottland
United Kingdom	Great Ormond Street Hospital for Children NHS Foundation Trust	London	England
United Kingdom	Myriad Trials Ltd	London	England
United States	Akron Children's Hospital	Akron	Ohio
United States	The Johns Hopkins University School of Medicine	Baltimore	Maryland
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	University of Missouri	Columbia	Missouri
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	Cook Children's Endocrinology	Fort Worth	Texas
United States	Cedars-Sinai Medical Center	Los Angeles	California
United States	Medical College of Wisconsin, Children's Hospital	Milwaukee	Wisconsin
United States	University of Minnesota Masonic Children's Hospital	Minneapolis	Minnesota
United States	Mount Sinai Kravis Children's Hospital	New York	New York
United States	UCSF Benioff Children's Hospital	Oakland	California
United States	Phoenix Children's Hospital	Phoenix	Arizona
United States	Washington University School of Medicine in St. Louis	Saint Louis	Missouri
United States	Seattle Children's Hospital	Seattle	Washington
United States	Children's National Health System	Washington	District of Columbia
United States	Nemours Alfred I. Dupont Hospital for Children	Wilmington	Delaware

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Countries where clinical trial is conducted

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in annualized growth velocity (AGV)		Every 6 months through end of study, up to 15 years
Primary	Change in Height Z-score		Every 6 months through end of study, up to 15 years
Primary	Change in Height		Every 6 months through end of study, up to 15 years
Primary	Change in BMI		Every 6 months through end of study, up to 15 years
Primary	Change in BMI Z-score		Every 6 months through end of study, up to 15 years
Primary	Change in ratios of upper to lower body segments		Every 6 months through end of study, up to 15 years
Primary	Change in ratios of upper and lower leg length ratio		Every 6 months through end of study, up to 15 years
Primary	Change in ratio of arms span to standing height ratio		Every 6 months through end of study, up to 15 years
Secondary	Change in Quality of Life in Short Statured Youth (QoLISSY) physical domain		Every 52 weeks through end of study, up to 15 years
Secondary	Change in Quality of Life in Short Statured Youth (QoLISSY) total score		Every 52 weeks through end of study, up to 15 years
Secondary	Change in patient global impression of severity (PGI-S)		Every 52 weeks through end of study, up to 15 years
Secondary	Change in caregiver global impression of severity (CaGI-S)		Every 52 weeks through end of study, up to 15 years
Secondary	Frequency of event rates of medical events of interest		Every 6 months through end of study, up to 15 years
Secondary	Proportion of children who report use of growth hormone, treatment patterns and impact on growth		Every 6 months through end of study, up to 15 years
Secondary	Proportion of children who report limb lengthening surgery		Every 6 months through end of study, up to 15 years