Vosoritide therapy in children with achondroplasia aged 3-59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 trial

What Was Tested

Researchers studied Vosoritide in this randomized controlled trial publication.

Who Took Part

By PubMed's age tags, the people studied were infants and young children, both men and women.

Children younger than 60 months with a clinical diagnosis of achondroplasia confirmed by genetic testing and who had completed a baseline growth study or observation period were enrolled into one of three sequential cohorts based on age at screening: 24-59 months (cohort 1); 6-23 months (cohort 2); and 0-5 months (cohort 3).

The second primary outcome was change in height Z score at 52 weeks from baseline, analysed in all randomly assigned participants.

How the Study Was Run

The researchers set it up as a randomized, double-blind and placebo-controlled study.

Because people in this Vosoritide study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.

Neither the participants nor the staff measuring them knew who was on Vosoritide, so expectations could not easily colour the readings.

A placebo (an inactive look-alike) gave the Vosoritide group a baseline to be compared against.

This double-blind, randomised, placebo-controlled, phase 2 trial was done in 16 hospitals across Australia, Japan, the UK, and the USA.

This trial is registered with EudraCT, 2016-003826-18, and ClinicalTrials.gov, NCT03583697.

What Was Measured

PubMed files the paper under topics including Achondroplasia, Gastroenteritis and Natriuretic Peptide, C-Type.

What This Means in Plain Language

The paper is a randomized, double-blind and placebo-controlled study of Vosoritide.

Its findings take up roughly 6 sentences of the abstract and are left out here on purpose; the source paper is the place to read them.

The publication is from 2024 (The Lancet. Child & adolescent health), a journal based in England.

More context: the Vosoritide compound page, all human trials.