A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)

What Was Tested

This registered phase 2 trial tested Vosoritide.

The primary objective is to evaluate the long-term safety and tolerability of daily SC injections of BMN 111 in children with ACH who have completed two years of treatment in the 111-202 study.

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

The registry classes the interventions as drug.

Who Took Part

Enrollment: 30 actual participants took part.

It was open to people of any sex aged from 7 years with no upper age limit.

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

Examples of who could join: Have completed 24 months of BMN 111 treatment in Study 111-202. Females \>= 10 years old or who have started menses must have a negative pregnancy test at baseline and be willing to have additional pregnancy tests during the study. Willing and able to perform all study procedures as physically possible. Parents/caregivers willing to administer daily injections to the subjects and complete the required training.

The full record lists 6 inclusion and 6 exclusion criteria; among the exclusions: requires any investigational agent prior to completion of study period; concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations for any reason; permanently discontinued bmn 111 during the 111-202 study.

How the Study Was Run

Design: single-group 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.

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

It started in 2016-01-26 with completion listed for 2027-12.

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

Researchers chose this as the main measure: Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability].

It also tracked 4 secondary outcomes, for example Final Adult Height.

What This Means in Plain Language

Put simply, this is a phase 2 trial of Vosoritide in 30 people.

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

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.