The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
140
Open-label administration of vosoritide using weight-band dosing
Evaluate the long-term efficacy of vosoritide treatment until final adult height (FAH)
As measured by change from baseline in height Z-score
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate the maintenance effect of vosoritide treatment on standing height
change from baseline in height at Week 52
Time frame: Baseline to week 52
Evaluate the maintenance effect of vosoritide treatment on standing height Z-score
Change from baseline in height Z-score at Week 52
Time frame: Baseline to week 52
Evaluate long-term effect of vosoritide treatment on growth velocity
Absolute 6- or 12-month interval AGV
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term effect of vosoritide treatment on body proportionality
Change from baseline in upper to lower body segment ratio
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term effect of vosoritide treatment on bone age versus chronological age
As measured by left-hand and wrist x-ray
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term effect of vosoritide treatment on bone metabolism biomarkers
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Phoenix Children's Hospital - Thomas Campus
Phoenix, Arizona, United States
Benioff Children's Hospital - Oakland
Oakland, California, United States
Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)
Wilmington, Delaware, United States
Children's National Medical Center
Washington D.C., 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
University of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, United States
University of Missouri
Columbia, Missouri, United States
Cook Children's Endocrinology
Fort Worth, Texas, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, United States
...and 20 more locations
Change from baseline in serum collagen X marker
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term changes in Health-Related Quality of Life (HRQoL)
Change from baseline as measured by Quality of Life In Short-Statured Youth (QoLISSY) total score
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term effect of vosoritide treatment on bone quality
Change from baseline in bone mineral density (BMD) as measured by Dual X-Ray Absorptiometry
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term effect of vosoritide treatment on pubertal growth as measured by Tanner Staging
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
To monitor the incidence of otitis media
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
To monitor the frequency of seizures
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males