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Updated: Feb 13, 2026

10:42
Oral Biofilm Sampling for Microbiome Analysis in Healthy Children
Published on: December 31, 2017
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トロフィネチドの生体同等性を評価するためのフェーズ1,ランダム化,オープンラベル試験は,健康な成人の口服用溶液および口服用溶液のための粉末として使用準備済みである
Mona Darwish1, Amy Yamamoto2, Yvonne Adegbenle2
1Acadia Pharmaceuticals Inc., 502 Carnegie Center, Suite 300, Princeton, NJ, 08540, USA. mdarwish@ACADIA-Pharm.com.
Advances in therapy
|February 12, 2026
まとめ
トロフィネチド (TPOS) の新しい粉末製剤は,レット症候群の治療のための口服溶液 (TOS) に生体同等である. このTPOS製剤は利点があり,生物学的利用可能性に影響を与えることなく,さまざまな水量で作ることができます.
科学分野:
- 薬物動態学と生物学的利用可能性に関する研究
- 薬剤の製造と開発について
- レット症候群の治療法 レット症候群の治療法
背景:
- トロフィネチドは,2歳以上の患者のレット症候群の治療に承認されており,現在,即用口服溶液 (TOS) として提供されています.
- 口服用溶液 (TPOS) のための新しいトロフィネチド粉は,より小さな包装,特定のエキシピエントを除外し,冷却の必要性を含む潜在的な利点を提示しています.
研究 の 目的:
- 新しいトロフィネチド粉末用口腔溶液 (TPOS) と,既存の使用済みの口腔溶液 (TOS) との比較において,その生物等価性を評価する.
- トロフィネチドの生物利用性にTPOSの異なる構成量による影響を評価する.
主な方法:
- 38人の健康な成人を対象とした3期間のランダム化,オープン・ラベル・クロスオーバー研究.
- 参加者は,TOS (60mL),TPOS (60mL水),TPOS (25mL水) の1回の12,000mgの口服投与を受けた.
- 生物同等性は,Cmax,AUC0-t,AUC0-∞を比較することによって決定され,幾何学平均比率の90%信頼区間は80-125%以内にする必要があります.
主要な成果:
- TPOS (60mL) とTOS.の間のすべての薬理学パラメータ (AUC0-t,AUC0-∞,Cmax) に対して,生物同等性基準が満たされた.
- 25mLの水で構成されたTPOSを含む他のすべての処置の比較も,生物同等性基準を満たしました.
- 最も頻繁に発生する有害事象は下痢 (15.8%) であったが,深刻なまたは予期せぬ有害事象は報告されなかった.
結論:
- この研究は,TPOSとTOSのバイオエキワレンスを実証し,それらの臨床的互換性を支持しています.
- TPOSは,トロフィネチドの生物利用性を損なうことなく,調整可能な水量で構成することができます.
- トロフィネチドのいずれの配方でも予期せぬ有害事象は観察されなかった.
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