NCT06668805
Noonan Syndrome
The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.
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.
All
3 Years to 11 Years
No
Vosoritide Injection
Phase 2
Interventional
30
2024-11-22
2026-09-23
Los Angeles, California, United States
Orange, California, United States
Aurora, Colorado, United States
Wilmington, Delaware, United States
Washington D.C., District of Columbia, United States
Miami, Florida, United States
Atlanta, Georgia, United States
Chicago, Illinois, United States
Indianapolis, Indiana, United States
Iowa City, Iowa, United States
Lexington, Kentucky, United States
Boston, Massachusetts, United States
New York, New York, United States
The Bronx, New York, United States
Charlotte, North Carolina, United States
Cincinnati, Ohio, United States
Philadelphia, Pennsylvania, United States
Nashville, Tennessee, United States
Dallas, Texas, United States
Houston, Texas, United States
Milwaukee, Wisconsin, United States
Saint Leonards, New South Wales, Australia
South Brisbane, Queensland, Australia
Parkville, Victoria, Australia
Calgary, Alberta, Canada
Toronto, Ontario, Canada
Montreal, Quebec, Canada
Marseille, Bouches-du-Rhône, France
Angers, Maine-et-Loire, France
Toulouse, Occitanie, France
Le Kremlin-Bicêtre, Paris, France
Paris, Paris, France
Homburg, Saarland, Germany
Florence, Florence, Italy
Genova, Genoa, Italy
Roma, Italy
Santiago de Compostela, La Coruña, Spain
Oviedo, Principality of Asturias, Spain
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
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.
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