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 (19) 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)
NOT_YET_RECRUITING
Phoenix
California
Cedars-Sinai Medical Center
NOT_YET_RECRUITING
Los Angeles
Benioff Children's Hospital - Oakland
NOT_YET_RECRUITING
Oakland
Washington, D.c.
Children's National Medical Center
RECRUITING
Washington D.c.
Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
NOT_YET_RECRUITING
Chicago
Maryland
The Johns Hopkins University School of Medicine
NOT_YET_RECRUITING
Baltimore
Minnesota
University of Minneasota Masonic Children's Hospital
NOT_YET_RECRUITING
Minneapolis
Missouri
University of Missouri
NOT_YET_RECRUITING
Columbia
Wisconsin
Children's Wisconsin - Fox Valley Hospital
NOT_YET_RECRUITING
Neenah
Other Locations
Australia
Royal Children's Hospital Melbourne
NOT_YET_RECRUITING
Parkville
France
Hospices Civils de Lyon - Hôpital Femme Mère Enfant