Vosoritide for Selected Genetic Causes of Short Stature

  • STATUS
    Recruiting
  • End date
    Jun 1, 2023
  • participants needed
    35
  • sponsor
    Andrew Dauber
Updated on 10 May 2021
growth hormone
KRAS
stimulation test
malnutrition
GH deficiency
igf-i
testicular
FGFR3
hormone deficiency
cardiofaciocutaneous syndrome
mras
saddan syndrome
hormones levels
somatotropin
vosoritide
shox deficiency
regulatory region
costello syndrome

Summary

Short stature can be caused by a number of genetic etiologies, many of which directly affect the growth plate. The FGFR3/CNP pathway is central to growth of the chondrocyte. The study team hypothesizes that patients with selected genetic causes of short stature that interact with this pathway will benefit from treatment with vosoritide, a CNP analog, a selective NPR-B agonist which directly targets the growth plate. This study will enroll patients with short stature in selected genetic categories and will follow them for a 6 month observation period to obtain a baseline growth velocity, safety profile and quality of life assessment. Patients will then be treated with vosoritide for 12 months and will be assessed for safety monitoring and improvement in height outcomes.

Details
Condition Dwarfism, Idiopathic Short Stature, Severe Short Stature, short stature
Treatment Vosoritide
Clinical Study IdentifierNCT04219007
SponsorAndrew Dauber
Last Modified on10 May 2021

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