This is a long-term, multicenter, non-interventional study of children ages 2.5 to \<17 years with hypochondroplasia (HCH).
The objective is to evaluate growth, HCH-related medical complications, health-related quality of life, functional abilities and cognitive functions of study participants. Data collected will contribute to the characterization of the natural history of children with HCH. No study medication will be administered.
Study Type
OBSERVATIONAL
Enrollment
150
Annualized height velocity
Time frame: up to 3 years
Change over time in height Z-score, upper arm to forearm ratio, and upper leg to lower leg ratio
Time frame: up to 3 years
Cognitive functions
Time frame: up to 3 years
HCH-related medical events reported as medical history or NT-AEs
Time frame: up to 3 years
HCH-related surgical procedures
Time frame: up to 3 years
Change from BL in the Physical Functioning dimension of the Pediatric Quality of Life Generic Core Scale Short Form.
Scale scores 0-100. Higher score=better Health-Related Quality of Llife
Time frame: up to 3 years
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
UCSF Benioff Children's Hospital
Oakland, California, United States
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Aurora, Colorado, United States
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Washington D.C., District of Columbia, United States
RECRUITINGJohns Hopkins School of Medicine
Baltimore, Maryland, United States
RECRUITINGUniversity of Missouri
Columbia, Missouri, United States
RECRUITINGCincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
RECRUITINGVanderbilt University Medical Center
Nashville, Tennessee, United States
RECRUITINGUniversity of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
Madison, Wisconsin, United States
RECRUITINGMurdoch Children's Research Institute
Parkville, Victoria, Australia
RECRUITINGLondon Health Sciences Centre - Children's Hospital of Western Ontario
London, Ontario, Canada
RECRUITING...and 15 more locations