A Multicenter, dOuble-blind, ranDomized, Placebo-controlled, Parallel-group Study to Determine the effIcacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With FabrY Disease
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 118
- 试验地点
- 49
- 主要终点
- Neuropathic Pain Monthly Score: Change From Baseline to Month 6
研究概览
简要总结
This study aimed to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease.
详细描述
The primary objective of this prospective, multicenter, double-blind, randomized, placebo-controlled, parallel group, Phase 3 study is to determine the effect of oral lucerastat monotherapy on neuropathic pain in subjects with Fabry disease (FD) through daily collection of patient-reported outcomes with an electronic diary.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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 and dated ICF prior to any study-mandated procedure;
- •Male or female adult subjects;
- •FD diagnosis confirmed with local genetic test results;
- •Fabry-associated neuropathic pain, as defined by the subject, in the last 3 months prior to screening;
- •Enzyme replacement therapy (ERT) status:
- •Subject never treated with ERT; or
- •Subject has not received ERT for at least 6 months prior to screening; or
- •Subject treated with ERT since at least 12 months at the time of the screening visit, and agreeing to stop ERT for approximately 8 months.
- •A woman of childbearing potential is eligible only under certain conditions, e.g. taking contraceptive measures.
- •Subjects with moderate or severe neuropathic pain during the screening period.
排除标准
- •Pregnant, planning to be become pregnant, or lactating subject.
- •Severe renal insufficiency (eGFR < 30 mL/min/1.73 m2) at screening.
- •Subject on regular dialysis for the treatment of chronic kidney disease.
- •Known and documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within 6 months prior to screening.
- •Clinically significant unstable cardiac disease (e.g. uncontrolled symptomatic arrhythmia, congestive heart failure NYHA class III or IV).
- •Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results.
研究组 & 干预措施
Lucerastat
干预措施: Lucerastat (Drug)
Placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Neuropathic Pain Monthly Score: Change From Baseline to Month 6
时间窗: From baseline to Month 6 (duration: 6 months)
Neuropathic pain on the modified BPI-SF3: subjects rated their neuropathic pain intensity ("neuropathic pain at its worst in the last 24 hours") on an 11-point scale, from 0 (no neuropathic pain) to 10 (worst imaginable neuropathic pain).
次要结局
- Plasma Globotriaosylceramide (Gb3; in ng/ml): Change From Baseline to Month 6(From baseline to Month 6 (duration: 6 months))
- Abdominal Pain Monthly Score: Change From Baseline to Month 6(From baseline to Month 6 (duration: 6 months))
- Number of Days With Diarrhea: Change From Baseline to Month 6(From baseline to Month 6 (duration: 6 months))