NCT00773890终止1 期
A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Activity of TRF-1101 on Microvascular Blood Flow, Vascular Endothelial Injury, and Vasoocclusive Pain in Patients With Sickle Cell Disease
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 80
- 试验地点
- 6
- 主要终点
- endothelial cell injury/inflammation
研究概览
简要总结
This study is designed to assess the safety, tolerability, and activity of TRF-1101 on microvascular blood flow, vascular endothelial injury, and vasoocclusive pain associated with sickle cell disease.
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
- •Be 18 years of age or older at the time of informed consent;
- •Have a diagnosis of homozygous sickle cell anemia or sickle cell-beta° thalassemia;
- •Have had 2 - 10 documented pain crises in the past year (pain crises are defined as visits to a medical clinic, Emergency Department or hospital, being bedridden and requiring constant analgesia at home for at least three days, or having a three-day interruption of life's activities [i.e., school, work, planned leisure activity] because of pain);
- •If female and of child bearing potential, have a negative serum or urine pregnancy test and be using an effective birth-control method with a history of reliability for the individual patient (use of mifepristone is not allowed);
- •Be properly informed of the nature and risks of the clinical investigation, be willing and able to comply with all clinical investigation-related procedures and assessments, and sign an Institutional Review Board (IRB) approved Informed Consent Form prior to entering the clinical investigation.
排除标准
- •Have a history of abnormal bleeding, stroke, moya moya vascular malformations, or any other contraindication to anticoagulation;
- •Be currently taking anticoagulant or thrombolytic medication;
- •Be currently taking an endothelin receptor antagonist, e.g., bosentan (Tracleer®);
- •Have a known sensitivity or allergy to heparin or related drugs;
- •Have a history of thrombocytopenia (platelet count < 100 x 103/mm3) induced by heparin or related drugs;
- •Have had fewer than 2 documented pain crises in the past year;
- •Have had a pain crisis within one month of screening or randomization;
- •If currently on or recently discontinued hydroxyurea treatment, have initiated or discontinued treatment or changed regimen within the past 6 months;
- •Have had a transfusion within last 120 days or have HbA% > 15% from prior transfusion;
- •Creatinine levels > 1.53 mg/dL (135 umol/L);
- •ALT levels ≥ 3 times normal;
- •Platelet count < 100 x 103/mm3;
- •INR > 2.0;
- •Be unable to tolerate oral medications;
- •Have unreliable venous access;
- •Be noncompliant with regular care;
- •Have a positive pregnancy test, be currently lactating, or be trying to become pregnant;
- •Have participation in an investigational drug or medical device study within previous 30 days;
- •Have any other condition or circumstance that in the opinion of the Investigator makes the patient a poor candidate for participation in the study.
研究组 & 干预措施
TRF-1101
Experimental
Daily treatment with TRF-1101
干预措施: TRF-1101 (Drug)
Placebo
Placebo Comparator
Daily treatment with placebo
干预措施: Placebo (Drug)
结局指标
主要结局
endothelial cell injury/inflammation
时间窗: Throughout trial
次要结局
- Microvascular blood flow and trends in frequency of vasoocclusive pain(throughout trial)
研究者
研究点 (6)
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