Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial

What Was Tested

Researchers studied Vosoritide in this randomized controlled trial publication.

This phase 3 trial was designed to further assess these preliminary findings.

Who Took Part

The study group was 52 patients.

PubMed indexes the participants as young children, children and teenagers, both men and women.

This randomised, double-blind, phase 3, placebo-controlled, multicentre trial compared once-daily subcutaneous administration of vosoritide with placebo in children with achondroplasia.

Eligible patients had a clinical diagnosis of achondroplasia, were ambulatory, had participated for 6 months in a baseline growth study and were aged 5 to less than 18 years at enrolment.

How the Study Was Run

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

Blinding on both sides kept the patients and researchers unaware of who received Vosoritide.

Nothing was hidden in this open-label Vosoritide study: the patients and staff both knew the treatment.

Some patients received a placebo instead of Vosoritide, giving a reference point.

A time span of 6 months appears in the methods.

The trial was done in hospitals at 24 sites in seven countries (Australia, Germany, Japan, Spain, Turkey, the USA, and the UK).

Randomisation was done by means of a voice or web-response system, stratified according to sex and Tanner stage.

The trial is complete and is registered, with EudraCT, number, 2015-003836-11.

What Was Measured

This phase 3 trial was designed to further assess these preliminary findings.

The subject headings attached to it are Absorptiometry, Photon, Achondroplasia, Biomarkers, Body Height, Bone Density, Collagen Type X and Injection Site Reaction.

What This Means in Plain Language

The paper is a randomized, double-blind and open-label study of Vosoritide in 52 patients over 6 months.

Results are reported in around 7 sentences of the abstract; we leave them to the paper itself rather than summarise them.

The publication is from 2020 (Lancet (London, England)), a journal based in England.

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