An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

What Was Tested

In this phase 3 trial, researchers set out to test Vosoritide.

The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia.

It had 1 arm (groups): BMN 111 (experimental).

The registry classes the interventions as drug.

Who Took Part

The registry lists 119 actual participants took part.

It was open to people of any sex aged from 6 years with no upper age limit.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Must have completed Study 111-301. Are willing and able to perform all study procedures.

The full record lists 5 inclusion and 8 exclusion criteria; among the exclusions: permanently discontinued bmn 111 or placebo prior to completion of the 111-301 study; require any investigational agent prior to completion of study period; current therapy with medications known to alter renal function.

How the Study Was Run

Design: single-group and open-label.

Being open-label, the Vosoritide study did not mask who received what.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2017-12-12 with completion listed for 2031-06.

It ran at 24 sites in United States (11), Japan (3), Spain (3), Australia (2), Germany (2) and United Kingdom (2).

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Change from baselines in mean annualized growth velocity (time frame: Through study completion, an average of 1 year).

It also tracked 13 secondary outcomes, for example Changes in health-related quality of life as measured by the Quality of Life in Short-Statured Youth questionnaire, Potential changes in daily activity performance as measured by Activities of Daily Living questionnaire, Characterize maximum concentration (Cmax) of BMN 111 in plasma, Characterize the area under the plasma concentration time-curve from time 0 to infinity (AUC0-∞), Characterize the area under the plasma concentration time-curve from time 0 to the last measurable concentration (AUC0-t) and Characterize the elimination half-life of BMN 111 (t1⁄2).

What This Means in Plain Language

Put simply, this is a phase 3 trial of Vosoritide in 119 people.

Its status is active not recruiting; no results are posted to the registry yet, so nothing here describes outcomes.

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