ベングルスタットがADPKD患者の将来的な治療法となり得るかどうかを判断するために設計された医学研究
基本情報
- NCT ID
- NCT03523728
- ステータス
- 中止
- 試験のフェーズ
- 第2/第3相
- 試験タイプ
- 介入
- 目標被験者数
- 478
- 治験依頼者名
- Sanofi
概要
Primary Objective: To determine the effect of venglustat on the rate of total kidney volume (TKV) growth (Stage 1) and estimated glomerular filtration rate (eGFR) decline in participants at risk of rapidly progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) (Stage 2). Secondary Objectives: * To determine the effect of venglustat on the rate of renal function decline (Stage 1) and on the rate of TKV growth (Stage 2). * To evaluate the pharmacokinetics (PK) of venglustat in ADPKD participants (Stages 1 and 2). * To determine the effect of venglustat on pain and fatigue, based on participant reported diary (Stages 1 and 2). * Safety/tolerability objectives: * To characterize the safety profile of venglustat (Stages 1 and 2). * To evaluate the effect of venglustat on mood using Beck Depression Inventory II (BDI-II) (Stages 1 and 2). * To evaluate the effect of venglustat on the lens by ophthalmological examination (Stages 1 and 2).