Vosoritide 国内第3相試験開始へ / Vosoritide Phase 3 Study going forward

日本国内において遂にBMN-111(vosoritide)の第3相試験 (BMN111-301)が開始されることが、具体的な施設名を踏まえClinicalTrialsGovにて公表されました。
多くの方が既にご存じのとおり、試験薬の無投与状況での半年間の成長速度の計測(BMN111-901) を終えた5-17歳までの被験者が国内施設にて約1年間の日々の投与を踏まえて効果測定を行います。



Finally, it has been disclosed on ClinicalTrialsGov that phase 3 of vosoritide (study No. BMN-111-301) is going forward in Japan.
As many of Japanese parents have already known, the study sites are 3 and subjects who are 5-17 years and have finished half a year of measurement study (BMN111-901) and going to do daily dose. After the 1 year dosed we are going to find out the results of their growth velocity.

A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
Source: ClinicalTrialsGov


It is defenetily rare case that phase 3 study starts without Phase 1 & 2 study in Japan. That will be truly the result of patients hope for improvement of QOL for their children.
Although we know there are various opinions regarding its cost-effectiveness, as we were begging of patients group at this stage end of 2015, that was our brilliant goal. We really appreciate for all of your collaborations.