A Phase 2, Randomized, Multicenter, Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
Status: Active_not_recruiting
Location: See all (38) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY
The purpose of this study in children with Noonan syndrome is to evaluate the effect of 3 doses of vosoritide on growth as measured by AGV after 6 months of treatment. The long-term efficacy and safety of vosoritide at the therapeutic dose will be evaluated up to FAH.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 3
Maximum Age: 11
Healthy Volunteers: f
View:
• Participants must be ≥ 3 years old, and \< 11 years old (females) or \< 12 years old (males), at the time of signing the informed consent form
• A clinical diagnosis of Noonan syndrome.
• A height assessment corresponding to a height Z-score of ≤ -1.28 SDs (below the 10th percentile for height) in reference to the general population of the same age and sex.
• Tanner Stage 1, at time of signing the ICF.
• Previous or current hGH treatment for short stature associated with their condition.
• Inadequate growth confirmed with an AGV that is less than age- and sex-matched average stature AGV determined using median heights from CDC growth charts
Locations
United States
California
Childrens Hospital Los Angeles
Los Angeles
Children's Hospital of Orange County Main Campus - Orange
Orange
Colorado
Children's Hospital Colorado
Aurora
Washington, D.c.
Children's National Medical Center
Washington D.c.
Delaware
Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)
Wilmington
Florida
Nicklaus Children's Hospital
Miami
Georgia
Emory University School of Medicine
Atlanta
Iowa
University of Iowa
Iowa City
Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago
Indiana
Riley Hospital for Children at Indiana University Health
Indianapolis
Kentucky
Kentucky Children's Hospital
Lexington
Massachusetts
New York Medical College
Boston
North Carolina
Atrium Health Carolinas Medical Center
Charlotte
New York
Icahn School of Medicine at Mount Sinai
New York
Children's Hospital at Montefiore
The Bronx
Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati
Pennsylvania
Childrens Hospital of Philadelphia
Philadelphia
Tennessee
Vanderbilt University Medical Center
Nashville
Texas
Children's Medical Center Dallas
Dallas
University of Texas Health Science Center at Houston (UT Health)
Houston
Wisconsin
Froedtert & Medical College of Wisconsin
Milwaukee
Other Locations
Australia
Murdoch Children's Research Institute
Parkville
Royal North Shore Hospital
Saint Leonards
Childrens Health Queensland Hospital and Health Service
South Brisbane
Canada
M.A.G.I.C. Clinic
Calgary
Centre Hospitalier Universitaire Sainte-Justine
Montreal
SickKids - The Hospital for Sick Children
Toronto
France
Angers University Hospital Center
Angers
South Paris University Hospitals - Bicetre Hospital