2016年3月末、BMN-111フェーズ2に関する新しいプロ
Phase 2 において、子どもの軟骨無形成症患者へ2年間の長期投与を行った
本件は、1月11日の同社会合にて発表された内容の一部がプロトコルとして具体的に文書化されています。
(the 34th Annual J.P. Morgan Healthcare Conference on Monday, January 11, 2016 at 8:30 am PT, or 11:30 am ET, in San Francisco）
また前回プロトコルとの比較に関しては、
- 対象年齢は、7歳から16歳。
- 半年間投与結果で効果が優れなかった5 µg/kg、10 µg/kgの各投与群は、良好な結果が得られた投与群15 µg/kgに統廃合。（30 µg/kgは勿論、最大60µg/kgに関する記載も従来どおり
です） - 評価項目は、有害事象（2区分）の発生件数、成長速度(cm/
yr)および成長パラメータ(Z-score)、 体のプロポーション（Upper-to-lower body segment ratio）。 - 参加・除外基準に関する記述がシンプルに。（invitatio
n only から察するに、 既存被験者を継続することで新規参加者を募る必要性が生じないか らかも（？）） - 各位の関心事のひとつである当該study の完了時期は、2022年12月。
実用化が見込まれる時期は果たして。。。
フェーズ3のプロセスを加味しても、
A new protocol about Phase 2 study of BMN 111 in Children with ACH carried end of March below. I think that is the case of the 2 years extension in phase 2 announced in JP Morgan conference in 11th January. (the 34th Annual J.P. Morgan Healthcare Conference on Monday, January 11, 2016 at 8:30 am PT, or 11:30 am ET, in San Francisco)
I am worried about the Estimated Study Completion Date. What’s your anticipation of the practical use of vosoritide? I wish not so far including P3 process.
English speakers may check it original document below.
==============================
Last updated: March 25, 2016
Title:
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
https://clinicaltrials.gov/
■Purpose
This is a Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia. 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.
■Study number: 111-205
■Estimated Enrollment: 46
■Study Start Date: January 2016
■Estimated Study Completion Date: December 2022
■Ages Eligible for Study: 7 Years to 16 Years
■Experimental: BMN 111 – Subcutaneous Injection
111-205 is an open-label, extension study. Subjects receive the same stable dose of BMN 111 received upon completion of the 111-202 study. BMN 111 will be administered in one of the following daily dosing regimens: 15 µg/kg, 30 µg/kg, up to 60 µg/kg daily.
■Further study details as provided by BioMarin Pharmaceutical:
○Primary Outcome Measures:
•Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: 260 Weeks ] [ Designated as safety issue: Yes ]
•Number of study participants with treatment-emergent adverse events.
•Number of study participants with treatment-emergent serious adverse events
○Secondary Outcome Measures:
•Growth Velocity [Efficacy] [ Time Frame: 260 Weeks ] [ Designated as safety issue: No ]Annualized growth velocity (cm/yr)
•Growth Parameters (Efficacy) [ Time Frame: 260 weeks ] [ Designated as safety issue: No ]Height standard score (Z-score)
•Body Proportions (Efficacy) [ Time Frame: 260 weeks ] [ Designated as safety issue: No ]Upper-to-lower body segment ratio
■Criteria
○Inclusion Criteria:
•Have completed 24 months of BMN 111 treatment in Study 111-202.
•Parent(s) or guardian(s) are willing and able to provide written, signed informed consent.
•If sexually active, willing to use a highly effective method of contraception while participating in 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.
○Exclusion Criteria:
•Requires any investigational agent prior to completion of study period.
•Have a condition or circumstance that, in the view of the Investigator, places the subject at high risk for poor treatment compliance or for not completing the study.
•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.
•Subject is pregnant.
•Current chronic therapy with restricted medications.
==============================