日本人ゴーシェ病患者を対象としたベラグルセラーゼアルファの多施設共同延長試験

基本情報

NCT ID

NCT01842841

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

5

治験依頼者名

Takeda

概要

Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB) that leads to progressive accumulation of glucocerebroside within macrophages and subsequent tissue and organ damage; typically of the liver, spleen, bone marrow, and brain. Type 1 Gaucher disease affects an estimated 30,000 persons worldwide and is the most common. Type 1 Gaucher disease does not involve the central nervous system. Patients with Type 2 Gaucher disease present with acute neurological deterioration, which leads to early death. Those with Type 3 disease typically display a more sub-acute neurological course, with later onset and slower progression. The primary objective of this study is to evaluate the long-term safety of every other week (EOW) dosing of velaglucerase alfa in Japanese patients with Gaucher disease who completed study HGT-GCB-087 and elected to continue treatment with velaglucerase alfa. Velaglucerase alfa has been developed and approved as an enzyme replacement therapy for Type 1 Gaucher disease.

対象疾患

介入

依頼者（Sponsor）

実施施設 (5)