A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
Start Date
7/30/2025
Completion Date
6/30/2028
Summary
The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with HCH aged 0 to \< 36 months over a 52-week period.
Detailed Description
Study 111-212 is a Phase 2, randomized, double-blind, placebo-controlled, multicenter study to assess the safety and efficacy of vosoritide versus placebo in infants and young children with HCH. Eligible participants with documented HCH confirmed by genetic testing will be randomized in a 1:1 ratio to receive vosoritide or placebo. Participants will receive study treatment daily for 52 weeks by subcutaneous (SC) injection, followed by a 2-week safety follow-up visit. Vosoritide dosing will follow a weight-band regimen.
Eligibility Criteria
Age Range: No minimum to No maximum
Interventions
Vosoritide
Placebo
Conditions
Locations
Phoenix Children's Hospital - Thomas Campus (Main)
Phoenix, Arizona 85016
United States
Cedars-Sinai Medical Center
Los Angeles, California 90048
United States
Benioff Children's Hospital - Oakland
Oakland, California 94609
United States
Children's National Medical Center
Washington D.C., District of Columbia 20010
United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois 60611
United States
The Johns Hopkins University School of Medicine
Baltimore, Maryland 21205
United States
University of Minneasota Masonic Children's Hospital
Minneapolis, Minnesota 55454
United States
University of Missouri
Columbia, Missouri 65211
United States
Cook Children's Endocrinology
Fort Worth, Texas 76104
United States
Children's Wisconsin - Fox Valley Hospital
Neenah, Wisconsin 54956
United States
Children's Health Queensland Hospital and Health Service
South Brisbane, Queensland 4101
Australia
Royal Children's Hospital Melbourne
Parkville, Victoria 3052
Australia
Hôpital Bicêtre
Le Kremlin-Bicêtre, Paris 94270
France
Hospices Civils de Lyon - Hôpital Femme Mère Enfant
Bron, Rhone-Alps 69500
France
Uniklinik Köln
Cologne, North Rhine-Westphalia 50937
Germany
Universitätsklinikum des Saarlandes
Homburg, Saarland 66421
Germany
Universitätskinderklinik Magdeburg
Magdeburg, Saxony-Anhalt 39120
Germany
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, Rome 00168
Italy
IRCCS Istituto Giannina Gaslini
Genova, 16147
Italy
Kumamoto University Hospital
Kumamoto, 860-8556
Japan
Osaka Women's and Children's Hospital
Osaka, 94-1101
Japan
Institute of Science Tokyo Hospital
Tokyo, 113-8519
Japan
Nihon University Itabashi Hospital
Tokyo, 173-8610
Japan
Tottori University Hospital
Tottori, 683-0854
Japan
Myriad Trials
London, England W1W 5DT
United Kingdom
Great Ormond Street Hospital
London, England WC1N3BH
United Kingdom