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.
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
A genetically confirmed diagnosis of Turner syndrome, SHOX deficiency or Noonan syndrome.
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.
Tanner Stage 1, at time of signing the ICF.
Participants with Turner syndrome known to have Y-chromosome material unless they have undergone gonadectomy and have fully external female genitalia.
Diagnosis of systemic disease or condition that may cause short stature other than Turner syndrome, SHOX deficiency, or Noonan syndrome, eg, renal, neoplastic, pulmonary, cardiac, gastrointestinal, immunologic and metabolic disease.
Bone age advanced beyond chronological age by more than 2 years.
Uncorrected congenital heart disease which places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension,
Parallel
Vosoritide Injection
DrugModified recombinant human C-type natriuretic peptide Vosoritide
Change from baseline in Annualized Growth Velocity (AGV)
Incidence of treatment-emergent adverse events
Incidence of new diagnosis of hypertrophic cardiomyopathy in children with Noonan syndrome
Incidence of cardiac conditions requiring discontinuation of study treatment
Change from baseline in height
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