A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia

What Was Tested

This registered phase 2 trial tested Vosoritide.

Study 111-206 is a Phase 2 randomized, double-blind, placebo-controlled clinical trial of BMN 111 in infants and young children with a diagnosis of achondroplasia.

It had two arms (groups): Active BMN111 (experimental) and Placebo (placebo comparator).

Daily subcutaneous injection of placebo.

The registry classes the interventions as drug.

Who Took Part

75 actual participants took part.

It was open to people of any sex aged up to 59 months.

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

Examples of who could join: Diagnosis of achondroplasia (ACH), confirmed by genetic testing. If subjects had previous genetic testing, subjects must have a lab report from a certified laboratory with the study specific mutation documented. Age 0 to \< 60 months, at study entry (Day 1). Willing and able to perform all study procedures as physically possible. Parent(s) or caregiver(s) are willing to administer daily injections to the subjects and complete the required training.

The full record lists 6 inclusion and 75 exclusion criteria; among the exclusions: have hypochondroplasia or short-stature condition other than achondroplasia (e.g., trisomy 21, pseudoachondroplasia, etc.); subject weighs \< 5.0 kg (cohort 1 and 2) or \< 4.0 kg (cohort 3); have any of the following:.

How the Study Was Run

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

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.

Groups ran in parallel: each of the participants stayed in one arm throughout.

As a double-blind Vosoritide study, the assignments were hidden from both participants and investigators until the end.

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

It started in 2018-06-13 with completion listed for 2022-01-26.

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

The main thing measured (primary outcome): Number of Participants With Adverse Events (AEs) by Severity Grade and Study Drug Treatment-emergent Adverse Events (TEAEs) (time frame: Up to Week 56 (Safety Follow-Up +/-7d)). Change From Baseline in Height Z-score at Week 52 (time frame: Baseline to Week 52).

It also tracked 53 secondary outcomes, for example Change From Baseline in Height at Week 52, Change From Baseline in Annualized Growth Velocity (AGV) at Week 52, Change From Baseline in Upper to Lower Body Segment Ratio at Week 52, Change From Baseline in Body Mass Index (BMI) at Week 52, Change From Baseline in BMI Z-score at Week 52 and Change From Baseline in Weight Z-score at Week 52.

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of Vosoritide in 75 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.