A 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 two arms (groups): Active BMN 111 (experimental) and Placebo (placebo comparator).

Daily subcutaneous injection of placebo.

The registry classes the interventions as drug.

More on the Plan

This is a Phase 3 randomized, placebo-controlled, double-blind multicenter study with approximately 110 subjects, aged 5 to \< 18 years old.

Subjects with documented Achondroplasia confirmed by genetic testing will have been enrolled in Study 111-901 for at least a 6-month period immediately before entering into the 111-301 study.

The route of administration is subcutaneous injection and the frequency is daily.

Who Took Part

The registry lists 121 actual participants took part.

It was open to people of any sex aged from 5 years up to 18 years.

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

Examples of who could join: Parent(s) or guardian(s) consent. 5 to \< 18 years old. ACH, documented and confirmed by genetic testing. At least a 6-month period of pretreatment growth assessment in Study 111-901 before study entry. Ambulatory and able to stand without assistance.

The full record lists 6 inclusion and 28 exclusion criteria; among the exclusions: hypochondroplasia or short stature condition other than ach; have any of the following:; hypothyroidism or hyperthyroidism.

How the Study Was Run

The set-up was randomized, parallel-group and triple-blind.

The participants were allocated by chance, so the Vosoritide group and the comparison group should start out alike.

The Vosoritide group and the comparison group ran side by side rather than switching.

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

Those kept unaware of assignments: participant, care provider and investigator.

It started in 2016-12-12 with completion listed for 2019-10-30.

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

Researchers chose this as the main measure: Change From Baseline in Annualized Growth Velocity (AGV) at Week 52 (time frame: At Baseline and Week 52).

It also tracked 3 secondary outcomes, for example Change From Baseline in Height Z-score at Week 52, Change From Baseline in Upper to Lower Segment Body Ratio at Week 52 and Summary of Subjects Experiencing Adverse Events (AEs) During Treatment.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Vosoritide in 121 people.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

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