A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
A Phase 2, Randomized, Controlled, Multicenter Study of Vosoritide in Children With Idiopathic Short Stature
BioMarin Pharmaceutical
100 participants
Oct 21, 2024
INTERVENTIONAL
Conditions
Summary
The purpose of this study is to evaluate i) the effect of multiple doses of vosoritide and ii) the effect of the therapeutic dose of vosoritide compared to human growth hormone (hGH)(hGH; only in the United States), in children with idiopathic short stature (ISS).
Eligibility
Inclusion Criteria5
- Height assessment corresponding to a height Z-score of ≤ -2.25 SDs in reference to the general population of the same age and sex, as calculated using the Centers for Disease Control and Prevention (CDC) growth charts
- Tanner Stage 1, at time of signing the ICF (unless too young to stage).
- Key Exclusions:
- Known chromosomal imbalance or genetic variant causing short stature syndrome, including but not limited to Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, Turner syndrome, disproportionate skeletal dysplasias, abnormal SHOX gene analysis, or Rasopathy (including Noonan syndrome)
- Previous treatment with a growth promoting agent
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Interventions
Experimental Drug Lyophilized powder for reconstitution
Commercial product containing somatotropin
Lyophilized powder for reconstitution
Locations(47)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT06382155