遺伝性筋萎縮性側索硬化症（ALS）の成人患者を対象としたBIIB067（トフェルセン）の有効性、安全性、忍容性、薬物動態および薬力学に関する研究

基本情報

NCT ID

NCT02623699

ステータス

完了

試験のフェーズ

第3相

試験タイプ

介入

目標被験者数

176

治験依頼者名

Biogen

概要

The primary objectives of Parts A and B of this study are to evaluate the safety, tolerability, and pharmacokinetics (PK) of ascending doses of tofersen in adults with ALS and a documented superoxide dismutase 1 (SOD1) mutation. The primary objective of Part C of this study is to evaluate the clinical efficacy of tofersen administered to adults with ALS and a confirmed SOD1 mutation. The secondary objective of Parts A and B of this study is to evaluate the effects of tofersen on levels of total SOD1 protein in the cerebrospinal fluid (CSF). The secondary objectives of Part C are to evaluate the safety, tolerability, pharmacodynamic (PD), and biomarker effects of tofersen.

対象疾患

介入

依頼者（Sponsor）

実施施設 (1)