RecruitingPhase 2NCT06668805

A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment

A Phase 2, Randomized, Multicenter, Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment

Sponsor

BioMarin Pharmaceutical

Enrollment

30 participants

Start Date

Nov 22, 2024

Study Type

INTERVENTIONAL

Conditions

Noonan Syndrome

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

Min Age: 3 YearsMax Age: 11 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Vosoritide Injection for people with noonan syndrome. The study is currently recruiting participants at 36 locations. People eligible for this study include aged 3 Years to 11 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGVosoritide 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

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NCT06668805

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