New drug aims to boost growth in kids with noonan syndrome

NCT ID NCT06668805

Recruiting now Disease control Sponsor: BioMarin Pharmaceutical Source: ClinicalTrials.gov ↗

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

bone disorder Dwarfism musculoskeletal system disorder Noonan syndrome SHOX-related short stature Turner syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

NOONAN SYNDROME SHORT STATURE HOMEOBOX- CONTAINING GENE SHOX DEFICIENCY TURNER SYNDROME

Contacts and locations

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Study contacts

Contact

Email: •••••@•••••
Contact

Email: •••••@•••••

Locations

Angers University Hospital Center
RECRUITING

Angers, Maine-et-Loire, 49 933, France
Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITING

Chicago, Illinois, 60611, United States
Atrium Health Carolinas Medical Center
RECRUITING

Charlotte, North Carolina, 28203, United States
Azienda Ospedaliero Universitaria - Ospedale Pediatrico Meyer
RECRUITING

Florence, Florence, Firenze, Italy
CHU de Toulouse - Hôpital des Enfants
RECRUITING

Toulouse, Occitanie, France
Central University Hospital of Asturias
NOT_YET_RECRUITING

Oviedo, Principality of Asturias, 33011, Spain
Children's Hospital Colorado
RECRUITING

Aurora, Colorado, 80045, United States
Children's Hospital at Montefiore
RECRUITING

The Bronx, New York, 10467, United States
Children's Hospital of Orange County Main Campus - Orange
RECRUITING

Orange, California, 92868, United States
Children's Medical Center Dallas
RECRUITING

Dallas, Texas, 75235, United States
Children's National Medical Center
RECRUITING

Washington D.C., District of Columbia, 20010, United States
Childrens Health Queensland Hospital and Health Service
RECRUITING

South Brisbane, Queensland, 4101, Australia
Childrens Hospital Los Angeles
RECRUITING

Los Angeles, California, 90027, United States
Childrens Hospital of Philadelphia
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
Cincinnati Children's Hospital Medical Center
RECRUITING

Cincinnati, Ohio, 45229-3026, United States
Emory University School of Medicine
RECRUITING

Atlanta, Georgia, 30322, United States
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
RECRUITING

Roma, 00 168, Italy
Hôpital Robert-Debré
RECRUITING

Paris, Paris, 75019, France
Hôpital de la Timone
RECRUITING

Marseille, Bouches-du-Rhône, 13385, France
IRCCS Istituto Giannina Gaslini
NOT_YET_RECRUITING

Genova, Genoa, 16147, Italy
Icahn School of Medicine at Mount Sinai
RECRUITING

New York, New York, 10029, United States
Kentucky Children's Hospital
RECRUITING

Lexington, Kentucky, 40506, United States
M Health Fairview Pediatric Specialty Clinic - Explorer
RECRUITING

Minneapolis, Minnesota, 55454, United States
M.A.G.I.C. Clinic
RECRUITING

Calgary, Alberta, T2E 7H7, Canada
Murdoch Children's Research Institute
RECRUITING

Parkville, Victoria, 3052, Australia
Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)
RECRUITING

Wilmington, Delaware, 19803, United States
New York Medical College
RECRUITING

Boston, Massachusetts, 02115, United States
Nicklaus Children's Hospital
NOT_YET_RECRUITING

Miami, Florida, 33155, United States
Riley Hospital for Children at Indiana University Health
RECRUITING

Indianapolis, Indiana, 46202, United States
Royal North Shore Hospital
RECRUITING

Saint Leonards, New South Wales, 2065, Australia
South Paris University Hospitals - Bicetre Hospital
RECRUITING

Le Kremlin-Bicêtre, Paris, 94270, France
St. Luke's Children's Endocrinology and Diabetes
RECRUITING

Boise, Idaho, 83712, United States
University of Iowa
RECRUITING

Iowa City, Iowa, 52242, United States
University of Texas Health Science Center at Houston (UT Health)
RECRUITING

Houston, Texas, 77030, United States
Universitätsklinikum des Saarlandes
RECRUITING

Homburg, Saarland, 66421, Germany
Vanderbilt University Medical Center
RECRUITING

Nashville, Tennessee, 37232-2578, United States

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