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A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
Sponsor: BioMarin Pharmaceutical
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.
Official title: A Phase 2, Randomized, Multicenter, Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
Key Details
Gender
All
Age Range
3 Years - 11 Years
Study Type
INTERVENTIONAL
Enrollment
30
Start Date
2024-11-22
Completion Date
2041-09
Last Updated
2026-05-11
Healthy Volunteers
No
Conditions
Interventions
Vosoritide Injection
Modified recombinant human C-type natriuretic peptide Vosoritide
Locations (36)
Childrens Hospital Los Angeles
Los Angeles, California, United States
Children's Hospital of Orange County Main Campus - Orange
Orange, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)
Wilmington, Delaware, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
Nicklaus Children's Hospital
Miami, Florida, United States
Emory University School of Medicine
Atlanta, Georgia, United States
St. Luke's Children's Endocrinology and Diabetes
Boise, Idaho, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, United States
University of Iowa
Iowa City, Iowa, United States
Kentucky Children's Hospital
Lexington, Kentucky, United States
New York Medical College
Boston, Massachusetts, United States
M Health Fairview Pediatric Specialty Clinic - Explorer
Minneapolis, Minnesota, United States
Icahn School of Medicine at Mount Sinai
New York, New York, United States
Children's Hospital at Montefiore
The Bronx, New York, United States
Atrium Health Carolinas Medical Center
Charlotte, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
Children's Medical Center Dallas
Dallas, Texas, United States
University of Texas Health Science Center at Houston (UT Health)
Houston, Texas, United States
Royal North Shore Hospital
Saint Leonards, New South Wales, Australia
Childrens Health Queensland Hospital and Health Service
South Brisbane, Queensland, Australia
Murdoch Children's Research Institute
Parkville, Victoria, Australia
M.A.G.I.C. Clinic
Calgary, Alberta, Canada
Hôpital de la Timone
Marseille, Bouches-du-Rhône, France
Angers University Hospital Center
Angers, Maine-et-Loire, France
CHU de Toulouse - Hôpital des Enfants
Toulouse, Occitanie, France
South Paris University Hospitals - Bicetre Hospital
Le Kremlin-Bicêtre, Paris, France
Hôpital Robert-Debré
Paris, Paris, France
Universitätsklinikum des Saarlandes
Homburg, Saarland, Germany
Azienda Ospedaliero Universitaria - Ospedale Pediatrico Meyer
Florence, Florence, Italy
IRCCS Istituto Giannina Gaslini
Genova, Genoa, Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, Italy
Central University Hospital of Asturias
Oviedo, Principality of Asturias, Spain