This is the BMN 111-206 study, a phase 2 study in Infants and Toddlers.
What happens in a Phase 2 study?
The participants have the disease or condition to be treated and can last up to 2 years. The purpose of this phase is to evaluate efficacy and side effects of the drug. On average, just approximately 33% of the drugs in evaluation in phase 2, move to the next phase. (3)
The 111-206 is a randomized, double-blind, placebo-controlled study of vosoritide in approximately 70 infants and young children with achondroplasia for 52 weeks or 1 year. This means that participants have an equal chance of receiving either placebo (an inactive treatment that looks the same as, and is given in the same way as, the investigational therapy but in just saline) or the investigational product BMN 111. It is not known whether placebo or investigational therapy is being administered to prevent bias. (2)
The study will be followed by a subsequent open-label extension. This means that at this time, both researchers and participants will know if they are getting vosoritide or placebo, and at what dose. (1)
Children in this study will have completed a minimum three-month baseline study to determine their respective baseline growth prior to entering the Phase 2 study. This differs from the phase 2 study, BMN 111-202, that requested 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. (4)
This trial is opening in Australia, Japan, the United Kingdom and the United States and participants
must remain a resident of the country they enrolled in throughout the trial period. (2)
Main goals of this 111-206 study
The main objectives of the study are to evaluate the safety, tolerability, and the effect of vosoritide on height Z-scores, which is the number of standard deviations in relation to the mean height of age-matched, average stature children.
The company also plans to augment the height Z-score data with assessments including proportionality, functionality, quality of life, sleep apnea, and foramen magnum dimension, as well as the advent of major illnesses and surgeries. (1)
One of the major goals to achieve in a treatment for achondroplasia is exactly what said in the previous paragraph, but rationally, this is a big challenge. Let’s wait for the next results and data publication on Vosoritide in children under 5 years-old, in whom effects of the drug are expected to be more evident.