Pediatric endocrinology

Effect of Vosoritide Therapy on IGF-I and Endogenous C-Type Natriuretic Peptide in Hypochondroplasia

J Clin Endocrinol MetabPublished Secondary analysis of a phase 2 single-arm clinical trial

Authors' conclusion

In children with HCH, IGF-1 levels are reduced and NTproCNP levels are elevated at baseline. A correlation between changes in these levels during treatment suggests a possible interaction between IGF-1 and CNP signaling.

Study context

Population
Prepubertal children aged 3 to 11 years with hypochondroplasia and height less than −2.25 SD
Primary endpoint
Longitudinal changes in IGF-1 and NTproCNP and their correlations with changes in height SD and AGV
Follow-up
12 months
Methodological detailsunblinded, 6-month baseline observation period
  • Unblinded
  • 6-month baseline observation period

Primary result

As reported in the abstract

Change in IGF-1 at 12 months positively correlated with change in NTproCNP (ρ)

ρ = 0.57

P = .024

Original publication

Effect of Vosoritide Therapy on IGF-I and Endogenous C-Type Natriuretic Peptide in Hypochondroplasia. J Clin Endocrinol Metab. Published 29 October 2025. doi:10.1210/clinem/dgaf591

Composed by AI from the published abstract and checked against it. How Briefs are made →

Get them every week

New studies on this topic in My Week and in your Monday e-mail.

We e-mail you a sign-in link and a code. No password, no spam.