The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH).
This is a Phase 3 randomized, stratified, placebo-controlled, double-blind multicenter study to evaluate the effect of 52 weeks of daily vosoritide administration on annualized growth velocity (AGV) in participants with HCH. Eligible participants with documented HCH confirmed by genetic testing will roll over from Study 111-902 and enter the 111-303 study. Participants will be randomly assigned to one of two treatment groups: Placebo or Vosoritide. The route of administration is subcutaneous injection, and the frequency of administration is daily.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
80
Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily.
Subcutaneous injection of recommended dose of placebo
Nemours Alfred I. DuPont Hospital for Children
Wilmington, Delaware, United States
Change from baseline in annualized growth velocity (AGV) at Week 52 versus placebo
Time frame: At week 52
Change from baseline in standing height at Week 52 versus placebo
Time frame: At week 52
Change from baseline in height Z-score at Week 52 versus placebo
Time frame: At week 52
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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
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Hospices Civils de Lyon - Hopital Femme Mere Enfant
Lyon, France
Hopital de la Timone
Marseille, France
...and 13 more locations