An Open-label Mechanistic Study to Assess the Pharmacokinetics, Pharmacodynamics and Safety of Orally Administered Epeleuton in Patients With Sickle Cell Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 35
- 试验地点
- 36
- 主要终点
- Changes from baseline in Phosphatidylserine
研究概览
简要总结
To assess the pharmacokinetics, pharmacodynamics and safety of Epeleuton capsules in adult SCD patients who are aged ≥18 years.
详细描述
The trial will consist of a 28-day screening period, 16 weeks of active treatment and a 30-day post-treatment follow-up period.
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
- •Patients with sickle cell disease (SCD) including:
- •2 sickle hemoglobin genes [HbSS]
- •HbSβ0 thalassemia
- •HbSβ+ thalassemia
- •Heterozygous for hemoglobin S and hemoglobin C [HbSC]
- •Male or female patients aged 18 years and older on the day of signing the informed consent form (ICF)
- •Patients who have had between 2 and 15 episodes of vaso-occlusive crisis (VOC) in the past year (12 months)
- •For patients taking hydroxyurea (HU), the dose of HU must be stable for at least 3 months prior to signing the ICD and with no anticipated need for dose adjustment during the study.
- •Female patients and male patients with female partners of childbearing potential must use highly effective contraceptive methods for the duration of the study.
排除标准
- •Patients who are receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion), have received an RBC transfusion for any reason within three months of the baseline visit
- •Patients who have received a hematopoietic stem cell transplant.
- •Patients with inadequate venous access as determined by the Investigator
- •Patients who are pregnant, planning pregnancy, breastfeeding and/or are unwilling to use adequate contraception during the trial.
研究组 & 干预措施
Epeleuton 4g/day
干预措施: Epeleuton (Drug)
结局指标
主要结局
Changes from baseline in Phosphatidylserine
时间窗: 16 Weeks
Change in Phosphatidlyserine from baseline to week 16.
Changes from baseline in Vascular Cell Adhesion Molecule 1 (VCAM-1)
时间窗: 16 Weeks
Changes in VCAM-1 from baseline to Week 16
Change from baseline in PROMIS Pain Interference Short Form
时间窗: 16 Weeks
Change in PROMIS Pain Interference from baseline to Week 16
Determination of exploratory biomarkers from baseline
时间窗: 16 Weeks
Determination of exploratory biomarkers at baseline and Week 16
Changes from baseline in Dense Red Blood Cells
时间窗: 16 Weeks
Changes in Dense Red Blood Cells from baseline to Week 16
Changes from baseline in Osmoscan
时间窗: 16 Weeks
Changes in Osmoscan from baseline to Week 16
Changes from baseline in Oxygen Point of Sickling
时间窗: 16 Weeks
Changes in Oxygen Point of Sickling from baseline to Week 16
Changes from baseline in Leukocytes
时间窗: 16 Weeks
Changes in Leukocytes from baseline to Week 16
Change from baseline in PROMIS Physical Activity Short Form
时间窗: 16 Weeks
Change in PROMIS Physical Activity from baseline to Week 16
Changes from baseline in P-selectin
时间窗: 16 Weeks
Change in P-selectin from baseline at Week 16.
Changes from baseline in absolute reticulocyte count
时间窗: 16 Weeks
Change in absolute reticulocyte count from baseline at Week 16.
Changes from baseline in RBC Laminin Adhesion
时间窗: 16 Weeks
Changes in RBC Laminin Adhesion from baseline to week 16
Changes from baseline in D-dimer
时间窗: 16 Weeks
Changes in D-dimer from baseline to Week 16
Changes from baseline in Hemoglobin
时间窗: 16 Weeks
Change in hemoglobin from baseline at Week 16
Changes from baseline in E-selectin
时间窗: 16 Weeks
Change in E-selectin from baseline at Week 16.
Changes from baseline in annualized rate of VOCs leading to a healthcare visit, and VOCs that are treated at home
时间窗: 16 Weeks
Changes in annualized rate of VOCs leading to a healthcare visit, and VOCs that are treated at home from baseline to week 16
Trough plasma concentrations of total and unesterified 15 HEPE
时间窗: 16 Weeks
Trough plasma concentrations of total and unesterified 15 HEPE at baseline and Week 16
Change from baseline in annualized rate of VOCs leading to a healthcare visit, and VOCs that are treated at home
时间窗: 16 Weeks
Change in annualized rate of VOCs leading to a healthcare visit, and VOCs that are treated at home from baseline to week 16
次要结局
未报告次要终点