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
Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)
Wilmington, Delaware, United States
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
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
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
Medical College of Wisconsin
Milwaukee, Wisconsin, United States
Murdoch Children's Research Institute
Parkville, Victoria, Australia
University of Alberta Stollery Children's Hospital
Edmonton, Alberta, Canada
SickKids - The Hospital for Sick Children
Toronto, Ontario, Canada
Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, Canada
Hospices Civils de Lyon - Hopital Femme Mere Enfant
Lyon, France
Hopital de la Timone
Marseille, France
...and 13 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