BIIB067（Tofersen）の長期評価

基本情報

NCT ID

NCT03070119

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

139

治験依頼者名

Biogen

概要

The primary objective of the study is to evaluate the long-term safety and tolerability of BIIB067 (tofersen) in participants with amyotrophic lateral sclerosis (ALS) and confirmed superoxide dismutase 1 (SOD1) mutation. The secondary objectives are to evaluate the pharmacokinetic (PK), pharmacodynamic (PD), biomarker effects, and efficacy of BIIB067 administered to participants with ALS and a confirmed SOD1 mutation.

対象疾患

介入

依頼者（Sponsor）