A Multicenter Multinational Observational Study of Children With Hypochondroplasia
- Conditions
- Hypochondroplasia
- Registration Number
- NCT06212947
- Lead Sponsor
- BioMarin Pharmaceutical
- Brief 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 400
- Participants must be ≤ 15 years old at the time of signing the informed consent
- Participants must have genetic confirmation of Hypochondroplasia diagnosis
- 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
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Change in BMI Every 6 months through end of study, up to 15 years Change in ratios of upper to lower body segments Every 6 months through end of study, up to 15 years Change in Height Every 6 months through end of study, up to 15 years Change in Height Z-score Every 6 months through end of study, up to 15 years Change in BMI Z-score Every 6 months through end of study, up to 15 years Change in ratios of upper and lower leg length ratio Every 6 months through end of study, up to 15 years Change in annualized growth velocity (AGV) Every 6 months through end of study, up to 15 years Change in ratio of arms span to standing height ratio Every 6 months through end of study, up to 15 years
- Secondary Outcome Measures
Name Time Method Change in patient global impression of severity (PGI-S) Every 52 weeks through end of study, up to 15 years Change in caregiver global impression of severity (CaGI-S) Every 52 weeks through end of study, up to 15 years Change in Quality of Life in Short Statured Youth (QoLISSY) physical domain Every 52 weeks through end of study, up to 15 years 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 Frequency of event rates of medical events of interest Every 6 months through end of study, up to 15 years Proportion of children who report limb lengthening surgery Every 6 months through end of study, up to 15 years Change in Quality of Life in Short Statured Youth (QoLISSY) total score Every 52 weeks through end of study, up to 15 years
Trial Locations
- Locations (43)
Institute of Science Tokyo Hospital
🇯🇵Tokyo, Japan
Phoenix Children's Hospital
🇺🇸Phoenix, Arizona, United States
UCSF Benioff Children's Hospital
🇺🇸Oakland, California, United States
Uniklinik Koln
🇩🇪Köln, Germany
Cedars-Sinai Medical Center
🇺🇸Los Angeles, California, United States
Nemours Alfred I. Dupont Hospital for Children
🇺🇸Wilmington, Delaware, United States
Children's National Health System
🇺🇸Washington, 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
🇺🇸Saint 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, Sao Paulo, Brazil
Stollery Children's Hospital
🇨🇦Edmonton, Alberta, Canada
SickKids - The Hospital for Sick Children
🇨🇦Toronto, Ontario, Canada
Centre Hospitalier Universitaire Sainte-Justine
🇨🇦Montréal, 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
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
Osaka University Hospital
🇯🇵Suita, Osaka, Japan
Tottori University Hospital
🇯🇵Yonago, Tottori, Japan
Kumamoto University Hospital
🇯🇵Kumamoto, Japan
Tokushima University Hospital
🇯🇵Tokushima, Japan
Hospital Universitario La Paz
🇪🇸Madrid, Spain
Vithas Hospital San Jose
🇪🇸Vitoria, 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