A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
Start Date
11/22/2024
Completion Date
9/1/2041
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.
Detailed Description
This is a Phase 2, randomized, multicenter, study of vosoritide in children with Noonan syndrome who have inadequate growth during or after human growth hormone (hGH) treatment. The study is intended to characterize the short-term efficacy and safety of 3 dosing regimens of vosoritide. The efficacy and safety of the vosoritide therapeutic dose will be further evaluated, and an analysis of the impact of vosoritide on final adult height (FAH).
Eligibility Criteria
Age Range: 3 years to 11 years
Interventions
Vosoritide Injection
Conditions
Locations
Childrens Hospital Los Angeles
Los Angeles, California 90027
United States
Children's Hospital of Orange County Main Campus - Orange
Orange, California 92868
United States
Children's Hospital Colorado
Aurora, Colorado 80045
United States
Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)
Wilmington, Delaware 19803
United States
Children's National Medical Center
Washington D.C., District of Columbia 20010
United States
Nicklaus Children's Hospital
Miami, Florida 33155
United States
Emory University School of Medicine
Atlanta, Georgia 30322
United States
St. Luke's Children's Endocrinology and Diabetes
Boise, Idaho 83712
United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois 60611
United States
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana 46202
United States
University of Iowa
Iowa City, Iowa 52242
United States
Kentucky Children's Hospital
Lexington, Kentucky 40506
United States
New York Medical College
Boston, Massachusetts 02115
United States
M Health Fairview Pediatric Specialty Clinic - Explorer
Minneapolis, Minnesota 55454
United States
Icahn School of Medicine at Mount Sinai
New York, New York 10029
United States
Children's Hospital at Montefiore
The Bronx, New York 10467
United States
Atrium Health Carolinas Medical Center
Charlotte, North Carolina 28203
United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio 45229-3026
United States
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United States
Vanderbilt University Medical Center
Nashville, Tennessee 37232-2578
United States
Children's Medical Center Dallas
Dallas, Texas 75235
United States
University of Texas Health Science Center at Houston (UT Health)
Houston, Texas 77030
United States
Royal North Shore Hospital
Saint Leonards, New South Wales 2065
Australia
Childrens Health Queensland Hospital and Health Service
South Brisbane, Queensland 4101
Australia
Murdoch Children's Research Institute
Parkville, Victoria 3052
Australia
M.A.G.I.C. Clinic
Calgary, Alberta T2E 7H7
Canada
Hôpital de la Timone
Marseille, Bouches-du-Rhône 13385
France
Angers University Hospital Center
Angers, Maine-et-Loire 49 933
France
CHU de Toulouse - Hôpital des Enfants
Toulouse, Occitanie
France
South Paris University Hospitals - Bicetre Hospital
Le Kremlin-Bicêtre, Paris 94270
France
Hôpital Robert-Debré
Paris, Paris 75019
France
Universitätsklinikum des Saarlandes
Homburg, Saarland 66421
Germany
Azienda Ospedaliero Universitaria - Ospedale Pediatrico Meyer
Florence, Florence Firenze
Italy
IRCCS Istituto Giannina Gaslini
Genova, Genoa 16147
Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, 00 168
Italy
Central University Hospital of Asturias
Oviedo, Principality of Asturias 33011
Spain