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 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 Change in Height Z-score 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
Cedars-Sinai Medical Center
🇺🇸Los Angeles, California, United States
UCSF Benioff Children's Hospital
🇺🇸Oakland, 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
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