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Clinical Trials in the USA / NCT06668805
Recruiting Phase 2

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

NCT06668805 · tracked via the Priya Life Science USA tracker
Sponsor
BioMarin Pharmaceutical
Phase
Phase 2
Started
2024-11-22
Last updated
2026-08-24

Condition(s) studied

Noonan Syndrome

Investigational drug(s) / intervention(s)

Vosoritide Injection

Vosoritide Injection: Modified recombinant human C-type natriuretic peptide Vosoritide

Study 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

Sex
ALL
Min age
3 Years
Max age
11 Years
Healthy volunteers
No
Inclusion Criteria: 1. Participants must be ≥ 3 years old, and \< 11 years old (females) or \< 12 years old (males), at the time of signing the informed consent form 2. A clinical diagnosis of Noonan syndrome. 3. A height assessment corresponding to a height Z-score of ≤ -1.28 SDs (below the 10th percentile for height) in reference to the general population of the same age and sex. 4. Tanner Stage 1, at time of signing the ICF. 5. Previous or current hGH treatment for short stature associated with their condition. 6. Inadequate growth confirmed with an AGV that is less than age- and sex-matched average stature AGV determined using median heights from CDC growth charts Exclusion Criteria: 1. Diagnosis of systemic disease or condition that may cause short stature other than Noonan syndrome, eg, renal, neoplastic, pulmonary, cardiac, gastrointestinal, immunologic and metabolic disease. 2. Bone age advanced beyond chronological age by more than 2 years. 3. Uncorrected congenital heart disease which places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension, 4. Have an unstable condition likely to require surgical intervention during the study. 5. Evidence of decreased growth velocity (AGV \< 1.5 cm/year) as assessed over a period of at least 6 months and growth plate closure assessed using bilateral lower extremity X-rays. 6. Previous limb-lengthening surgery, or planned or expected to have limb lengthening surgery during the study period. 7. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period.

Primary outcome measure(s)

Trial sites (41)

FacilityCityRegionStatus
Childrens Hospital Los Angeles Los Angeles California Recruiting
Children's Hospital of Orange County Main Campus - Orange Orange California Recruiting
Children's Hospital Colorado Aurora Colorado Recruiting
Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children) Wilmington Delaware Recruiting
Children's National Medical Center Washington D.C. District of Columbia Recruiting
Nicklaus Children's Hospital Miami Florida Recruiting
Emory University School of Medicine Atlanta Georgia Recruiting
St. Luke's Children's Endocrinology and Diabetes Boise Idaho Withdrawn
Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois Recruiting
Riley Hospital for Children at Indiana University Health Indianapolis Indiana Recruiting
University of Iowa Iowa City Iowa Recruiting
Kentucky Children's Hospital Lexington Kentucky Recruiting
New York Medical College Boston Massachusetts Recruiting
M Health Fairview Pediatric Specialty Clinic - Explorer Minneapolis Minnesota Withdrawn
Icahn School of Medicine at Mount Sinai New York New York Recruiting
Children's Hospital at Montefiore The Bronx New York Recruiting
Atrium Health Carolinas Medical Center Charlotte North Carolina Recruiting
Cincinnati Children's Hospital Medical Center Cincinnati Ohio Recruiting
Childrens Hospital of Philadelphia Philadelphia Pennsylvania Recruiting
Vanderbilt University Medical Center Nashville Tennessee Recruiting
Children's Medical Center Dallas Dallas Texas Recruiting
University of Texas Health Science Center at Houston (UT Health) Houston Texas Recruiting
Froedtert & Medical College of Wisconsin Milwaukee Wisconsin Active Not Recruiting
Royal North Shore Hospital Saint Leonards New South Wales Recruiting
Childrens Health Queensland Hospital and Health Service South Brisbane Queensland Recruiting
Murdoch Children's Research Institute Parkville Victoria Recruiting
M.A.G.I.C. Clinic Calgary Alberta Not Yet Recruiting
SickKids - The Hospital for Sick Children Toronto Ontario Active Not Recruiting
Centre Hospitalier Universitaire Sainte-Justine Montreal Quebec Recruiting
Hôpital de la Timone Marseille Bouches-du-Rhône Recruiting
Angers University Hospital Center Angers Maine-et-Loire Recruiting
CHU de Toulouse - Hôpital des Enfants Toulouse Occitanie Recruiting
South Paris University Hospitals - Bicetre Hospital Le Kremlin-Bicêtre Paris Recruiting
Hôpital Robert-Debré Paris Paris Recruiting
Universitätsklinikum des Saarlandes Homburg Saarland Recruiting
Azienda Ospedaliero Universitaria - Ospedale Pediatrico Meyer Florence Florence Recruiting
IRCCS Istituto Giannina Gaslini Genova Genoa Recruiting
Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Ospedale San Raffaele Milan Milan Withdrawn
Fondazione Policlinico Universitario Agostino Gemelli IRCCS Roma Italy Recruiting
Hospital Clínico Universitario de Santiago Santiago de Compostela La Coruña Not Yet Recruiting

+ 1 more sites — see the full list on the official registry below.

More BioMarin Pharmaceutical trials in the USA

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06668805 on ClinicalTrials.gov ↗ ← All trials in the USA