A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia

What Was Tested

This registered phase 2 trial tested Vosoritide.

The primary objective is to assess the safety and tolerability of daily BMN 111 administered to children with achondroplasia.

It had four arms (groups): Cohort 1 (experimental), Cohort 2 (experimental), Cohort 3 (experimental) and Cohort 4 (experimental).

The registry classes the interventions as drug.

Who Took Part

The registry lists 35 actual participants took part.

It was open to people of any sex aged from 5 years up to 14 years.

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

Examples of who could join: Parent(s) or guardian(s) are willing and able to provide written, signed informed consent. 5 to 14 years old at end of study. ACH, documented by clinical grounds, confirmed by genetic testing. At least 6-month of pretreatment growth assessment in Study 111-901 before study entry, and one standing height at least 6 months prior to screening for 111-202. Negative pregnancy test at the Screening Visit for females ≥ 10 years old or who have begun menses.

The full record lists 10 inclusion and 52 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

Design: non-randomized, sequential and open-label.

Everyone knew when Vosoritide was being given (open label), which is simpler to run but leaves more room for expectations to affect what is reported.

It started in 2014-01-13 with completion listed for 2017-10-02.

It ran at 9 sites in United States (6), Australia (1), France (1) and United Kingdom (1).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Overall Summary of Adverse Events During Initial 6-Month Period (time frame: Up to Month 6 ± 7 Days). Overall Summary of Adverse Events During Entire Study Period (time frame: Up to Month 25 ± 7 Days).

It also tracked 21 secondary outcomes, for example Change From Baseline in Annualized Growth Velocity (AGV) During Initial 6-Month, Change From Baseline in Annualized Growth Velocity (AGV) During Entire Study Period - Cohort 3 and 4, Change From Baseline in Annualized Growth Velocity (AGV) During Entire Study Period - Cohort 1 and 2 Switchers, Change From Baseline in Height Z-Scores Using CDC Reference Standard During Entire Study Period - Cohort 3 and 4, Change From Baseline in Height Z-Scores Using CDC Reference Standard During Entire Study Period - Cohort 1 and 2 Switchers and Change From Baseline in Upper to Lower Body Ratios During Initial 6-Months.

What This Means in Plain Language

Put simply, this is a phase 2, non-randomized trial of Vosoritide in 35 people.

Its size is small, so on its own it can only give an early look.

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.