開かれた乳幼児の治験参加の展望 / Finally Opens for infants/toddlers to Get in Clinical Trial


間もなく、辿り着いたclinicaltrials.gov(study No:111-901)。当時、5-13.5歳が対象であったため、わが子は参加は見送ることとなった。あれから約3年経過した今、遂に5歳以下(メインは2-5)に年齢制限を引き下げて再募集となりました。アップデートされたプロトコルを見たとき、耳元が真空状態となったかのような感覚。。。なんというか、当時感じた感激、口惜しさ、再募集時への抱負など、いろんな感情が、無音の脳裏にこみ上げてきました。

If I say the starting point of this story of Eigo and me in relating achondroplasia is the day Eigo was diagnosed as Achondroplasia after his birth, it must be the 2nd origin to have known the clinical trials by Biomarin in that moment. As soon as I knew about it, I searched clinicaltrials.gov(study No:111-901). But its scope of enrollment was 5-13.5 years according to the study sites, so Eigo could not enroll at the moment.

Now it has passed around 3 years, finally the study re-opened for under 5 years. When I find the protocol updated recently, I felt…what can I say… no sounds…it is kind of like a feeling in the vacuum, and comes in my mind, with no sounds. Those memories that is delightful in finding, disappointment in that moment and aspiration next re-open.


当時と明らかに違うもの。それは、エイゴと過ごした3年間において、様々な人との関係・絆…積み上げた履歴が、エイゴの誕生後のあの頃とは比べてものにならないほど、多く積みあがってしまった。職場関係者、親友、そして、同士ともいうべきGLORY TO ACHONDROPLASIAに心を寄せて下さる家族の皆様。人との関係性、経済面、そして、家内や自分のキャリア、娘の教育を考えれば、容易に決断できるものではない。ただ、本プロトコルは募集から1か月を経過し、瞬く間に現地の病院では、Waiting Listに希望者の名前が刻まれていく。。。


-Hardly to Decide It Right Away

There is a one big thing which is different from that moment. That is personal history which I have made relationship, bonds of friendship and to be a big accumulation with for 3 years comparing with that moment Eigo’s birth. The history has made with colleague on business, close friends and families who have children with ACH and support “GLORY TO ACHONDROPLASIA”. It is hard to decide to go to the US for enrollment right way because of human relationship, expenditure, career of my daughter, my wife and me. Even though, in this moment, lots of applicants are trying to be in waiting list in study sites for the enrollment, because it has passed for 1 month since the protocol has updated.

What is the “GLORY” for us? What is the “Challenge” for us? I am in serious dilemma between the aspect of pure feeling I had three years ago and the other aspect of current reason  I face to reality. There is a thing I could say. My decision doesn’t matter, as long as the clinical trial have been proceeding. It must be true that many of families would get a hope for children and grandchildren future. It is just like our dream.


直接的に薬を投与するものではありません。BMN-111のフェーズ2に進むため、成長速度等のベースラインを無投薬状況で計測することが目的となります。従前、5-13.5歳を対象にしていたものが、このたび、更に乳幼児に対象が広がりました。2歳以上が主対象のようですが、状況によって0-2歳の参加も認められるかもしれないとの記載があります。日本では、参加可能な病院がないため、渡航・長期滞在に伴う経済的な負担はいうまでもありません。また、本治験に参加し、最低半年間の治験期間を経たからといって、フェーズ2に進める保障はどこにもありません。実際、5歳以上を対象としたstudy( No.111-202)においては、厳しい倍率が実績値として残っています。

Notes- about this trial

Multicenter, multinational study to collect consistent baseline growth measurements on pediatric patients with Achondroplasia being considered for subsequent enrollment in Study 111-202. No study drug is administered. At that moment, it was opened for 5-13.5 years but re-opened for under 5 years. Patients aged 2 to 13.5 years, inclusive, on the date of consent will be enrolled. Patients aged 0 to < 2 years on the date of consent may also be enrolled, the timing of which will be at the discretion of the sponsor. Since there are no study sites in Japan, needless to say, we would have lots of expenditure for flight and stay there during trial. Furthermore there are no guarantee to enroll drug study of phase 2 even if we are enrolled this measurement trial for at least half year. Actually according to the result over 5 years case(No.111-202) last year, it is easy to understand that proceeding phase 2 is very hard.