New drug aims to boost growth in kids with noonan syndrome
NCT ID NCT06668805
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests vosoritide, a protein that stimulates bone growth, in 30 children with Noonan syndrome who are not growing well despite growth hormone treatment. The goal is to see if vosoritide can safely increase their growth rate over six months. Participants will receive one of three doses, and researchers will monitor height changes and any side effects.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Vosoritide (a modified protein that helps with bone growth)
What this could lead to
If successful, vosoritide could offer a new treatment option to help children with Noonan syndrome grow taller when growth hormone alone isn't enough.
What could go wrong
This is an early Phase 2 trial with only 30 participants, so results may not apply to everyone. The long-term safety and effectiveness are still unknown, and there may be side effects.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Angers University Hospital Center
RECRUITINGAngers, Maine-et-Loire, 49 933, France
Contact Email: •••••@•••••
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Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
Contact Phone: •••-•••-••••
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Atrium Health Carolinas Medical Center
RECRUITINGCharlotte, North Carolina, 28203, United States
Contact Email: •••••@•••••
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Azienda Ospedaliero Universitaria - Ospedale Pediatrico Meyer
RECRUITINGFlorence, Florence, Firenze, Italy
Contact Phone: •••-•••-•••• Email: •••••@•••••
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CHU de Toulouse - Hôpital des Enfants
RECRUITINGToulouse, Occitanie, France
Contact Email: •••••@•••••
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Central University Hospital of Asturias
NOT_YET_RECRUITINGOviedo, Principality of Asturias, 33011, Spain
Contact Email: •••••@•••••
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Children's Hospital Colorado
RECRUITINGAurora, Colorado, 80045, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Hospital at Montefiore
RECRUITINGThe Bronx, New York, 10467, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Hospital of Orange County Main Campus - Orange
RECRUITINGOrange, California, 92868, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Medical Center Dallas
RECRUITINGDallas, Texas, 75235, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's National Medical Center
RECRUITINGWashington D.C., District of Columbia, 20010, United States
Contact Email: •••••@•••••
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Childrens Health Queensland Hospital and Health Service
RECRUITINGSouth Brisbane, Queensland, 4101, Australia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Childrens Hospital Los Angeles
RECRUITINGLos Angeles, California, 90027, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Childrens Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Cincinnati Children's Hospital Medical Center
RECRUITINGCincinnati, Ohio, 45229-3026, United States
Contact Phone: •••-•••-••••
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Emory University School of Medicine
RECRUITINGAtlanta, Georgia, 30322, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Fondazione Policlinico Universitario Agostino Gemelli IRCCS
RECRUITINGRoma, 00 168, Italy
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Hôpital Robert-Debré
RECRUITINGParis, Paris, 75019, France
Contact Email: •••••@•••••
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Hôpital de la Timone
RECRUITINGMarseille, Bouches-du-Rhône, 13385, France
Contact Email: •••••@•••••
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IRCCS Istituto Giannina Gaslini
NOT_YET_RECRUITINGGenova, Genoa, 16147, Italy
Contact Email: •••••@•••••
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Icahn School of Medicine at Mount Sinai
RECRUITINGNew York, New York, 10029, United States
Contact Email: •••••@•••••
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Kentucky Children's Hospital
RECRUITINGLexington, Kentucky, 40506, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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M Health Fairview Pediatric Specialty Clinic - Explorer
RECRUITINGMinneapolis, Minnesota, 55454, United States
Contact Email: •••••@•••••
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M.A.G.I.C. Clinic
RECRUITINGCalgary, Alberta, T2E 7H7, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Murdoch Children's Research Institute
RECRUITINGParkville, Victoria, 3052, Australia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)
RECRUITINGWilmington, Delaware, 19803, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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New York Medical College
RECRUITINGBoston, Massachusetts, 02115, United States
Contact Email: •••••@•••••
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Nicklaus Children's Hospital
NOT_YET_RECRUITINGMiami, Florida, 33155, United States
Contact Email: •••••@•••••
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Riley Hospital for Children at Indiana University Health
RECRUITINGIndianapolis, Indiana, 46202, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Royal North Shore Hospital
RECRUITINGSaint Leonards, New South Wales, 2065, Australia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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South Paris University Hospitals - Bicetre Hospital
RECRUITINGLe Kremlin-Bicêtre, Paris, 94270, France
Contact Email: •••••@•••••
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St. Luke's Children's Endocrinology and Diabetes
RECRUITINGBoise, Idaho, 83712, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Iowa
RECRUITINGIowa City, Iowa, 52242, United States
Contact Phone: •••-•••-••••
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University of Texas Health Science Center at Houston (UT Health)
RECRUITINGHouston, Texas, 77030, United States
Contact Email: •••••@•••••
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Universitätsklinikum des Saarlandes
RECRUITINGHomburg, Saarland, 66421, Germany
Contact Phone: •••-•••-••••
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Vanderbilt University Medical Center
RECRUITINGNashville, Tennessee, 37232-2578, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••