A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Vosoritide in Infants and Young Children With Hypochondroplasia, Aged 0 to < 36 Months
Status: Recruiting
Location: See all (26) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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.
Eligibility
Participation Requirements
Sex: All
Maximum Age: 3
Healthy Volunteers: f
View:
• Participants must be 0 to \< 36 months of age at randomization.
• Participants must have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated with HCH).
• Participants aged 0 to \< 12 months must have a height Z-score of ≤ -1.0 SDS andparticipants aged ≥ 12 to \< 36 months must have a height Z-score of ≤ -2.0 SDS in reference to the average stature of the same sex and age, as calculated using the Center for Disease Control and Prevention (CDC) growth charts.
• Participant's weight at the Day 1 visit (pre-treatment) must be ≥ 3 kg.
Locations
United States
Arizona
Phoenix Children's Hospital - Thomas Campus (Main)
RECRUITING
Phoenix
California
Cedars-Sinai Medical Center
NOT_YET_RECRUITING
Los Angeles
Benioff Children's Hospital - Oakland
RECRUITING
Oakland
Washington, D.c.
Children's National Medical Center
RECRUITING
Washington D.c.
Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITING
Chicago
Maryland
The Johns Hopkins University School of Medicine
RECRUITING
Baltimore
Minnesota
University of Minneasota Masonic Children's Hospital
RECRUITING
Minneapolis
Missouri
University of Missouri
RECRUITING
Columbia
Texas
Cook Children's Endocrinology
NOT_YET_RECRUITING
Fort Worth
Wisconsin
Children's Wisconsin - Fox Valley Hospital
NOT_YET_RECRUITING
Neenah
Other Locations
Australia
Royal Children's Hospital Melbourne
RECRUITING
Parkville
Children's Health Queensland Hospital and Health Service
NOT_YET_RECRUITING
South Brisbane
France
Hospices Civils de Lyon - Hôpital Femme Mère Enfant