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

What Was Tested

This registered phase 2 trial tested Vosoritide.

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

The registry classes the interventions as drug.

Who Took Part

73 actual participants took part.

It was open to people of any sex aged from 15 months 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 111-206 on investigational treatment (BMN 111 or placebo). Are willing and able to perform all study procedures.

The full record lists 3 inclusion and 7 exclusion criteria; among the exclusions: permanently discontinued bmn 111 or placebo prior to completion of study 111-206; require any investigational agent (except bmn 111) prior to completion of study period; pregnant or planning to become pregnant (self or partner) at any time during the study.

How the Study Was Run

Design: single-group and open-label.

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

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

It started in 2019-06-12 with completion listed for 2038-05.

It ran at 16 sites in United States (9), Japan (3), Australia (2) and United Kingdom (2).

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Evaluate the incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] (time frame: "Through study completion, an average of 5 years"). Evaluate change in height/length z-score in children with ACH treated with BMN 111 (time frame: "Through study completion, an average of 5 years").

It also tracked 15 secondary outcomes, for example Evaluate the change from baseline of mean annualized growth velocity (AGV), 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 elimination half-life of BMN 111 (t½), Characterize the apparent clearance of drug and Characterize the apparent volume of distribution based upon the terminal phase (Vz/F).

What This Means in Plain Language

Put simply, this is a phase 2 trial of Vosoritide in 73 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.