A Multi-center, Open-label, Uncontrolled, Single-arm, Extension Study to Determine the Long-term Safety and Tolerability of Oral Lucerastat in Adult Subjects With Fabry Disease
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 107
- 试验地点
- 84
- 主要终点
- Treatment-emergent adverse events (AEs)
研究概览
简要总结
A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease. This study includes a sub-study evaluating kidney Gb3 inclusions (and other histologic lesions) in male participants with classic Fabry disease who have been treated for at least 2 years with lucerastat monotherapy in study ID-069A302.
详细描述
Study ID-069A302 will continue at each site until lucerastat is commercially available in the respective country, or until all subjects have (prematurely) discontinued the trial, or until the sponsor terminates the study, whichever is earliest.
Note that, in Europe (local protocol amendment), the maximum individual study participation is up to Month 96.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed ICF prior to any study-mandated procedure;
- •Subject completed the 6-month, double-blind treatment period in study ID 069A301
- •Woman of childbearing potential only if agreement 1) to follow a specified contraception scheme, 2) to undertake monthly urine pregnancy tests.
- •Fertile male only if agreement 1) to use a condom, 2) to not father a child.
排除标准
- •Pregnant / planning to be become pregnant or lactating subject;
- •Subject considered to be at high risk of developing clinical signs of organ involvement within the time period of the study, as per investigator judgment;
- •Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results as per investigator judgment.
- •In addition, the subject must not be enrolled in study ID-069A302 if at any time during study ID-069A301, one of the following criteria was met:
- •Subject's eGFR per the Chronic Kidney Disease Epidemiology Collaboration creatinine equation < 15 mL/min/1.73 m2;
- •Subject experienced an event of acute kidney injury Common Terminology Criteria for Adverse Event (CTCAE) grade 2 or above;
- •Subject experienced an event of stroke CTCAE grade 3 or above;
- •Subject experienced an event of heart failure leading to in-patient hospitalization or prolongation of ongoing hospitalization.
研究组 & 干预措施
Lucerastat
Dose will be based on subject's eGFR.
干预措施: Lucerastat (Drug)
结局指标
主要结局
Treatment-emergent adverse events (AEs)
时间窗: From enrollment to Follow-up 1 (FU1) visit; duration: for up to 10 years including 1 month Follow-up
次要结局
未报告次要终点