A Phase 2 Pilot Randomized Controlled Trial Assessing Feasibility of Thromboprophylaxis With Apixaban in JAK2-positive Myeloproliferative Neoplasm Patients
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 44
- 试验地点
- 1
- 主要终点
- Average monthly subject recruitment rate of all study sites during a 6-month recruitment period
研究概览
简要总结
Myeloproliferative neoplasms (MPNs) are blood disorders that occur when the body makes too many white or red blood cells, or platelets. This overproduction of blood cells in the bone marrow can create problems for blood flow and lead to various symptoms. One of the major problems is the formation of blood clots. These may form in the veins of a patient's legs or arms where they cause leg or arm pain, swelling or difficulty walking. These clots may travel to the lung and then cause chest pain, shortness of breath and sometimes death. Blood clots can also lead to poor or no blood flow to one's heart, brain, or other organs, causing damages that cannot be easily or ever repaired, such as stroke or heart attack.
Patients diagnosed with certain types of MPN are associated with a higher risk of developing blood clots and related complications. For this reason, MPN patients are usually treated with low-dose aspirin, a common drug used for blood clot prevention, on long-term basis to prevent the formation of blood clots and other complications. However, recent studies also show that the risk of blood clots remains elevated in MPN patients treated with aspirin, and there may not be improvement or reduction in fatal or other events that are associated with blood clots. In addition, since this medical condition is rare, so there's a lack of studies done with high quality results to help physicians decide the best treatment plan for these patients.
The study drug, apixaban, is a new type of orally-taken blood thinner that has been shown to be effective and safe for prevention and treatment of blood clots in various patient populations. The investigators will evaluate whether apixaban is safer and/or better at preventing blood clots and other complications in MPN patients compared to aspirin.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Prevention
- 盲法
- Single (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
- •Male or female subjects aged 18 years or older,
- •Confirmed diagnosis of PV, JAK2ET or JAK2 pre-fibrotic MF, per local clinical definitions
- •Able and willing to comply with study procedures and follow-up examinations contained within the written consent form
排除标准
- •Known allergy to apixaban or aspirin,
- •Another need for anticoagulation or specific anti-platelet therapy,
- •Contraindication to thromboprophylaxis (which would specifically include but not be limited to platelets less than 50x10^9/L and acquired Von Willebrand disease),
- •Current pregnancy or breast-feeding,
- •Renal dysfunction (Creatine Clearance <25 mL/min),
- •Known liver disease
- •Currently on any medication with a known interaction to apixaban
- •Unwilling to use an effective means of contraception for women of childbearing potential
- •Overtly fibrotic myelofibrosis
- •Myelodysplastic/myeloproliferative neoplasms
研究组 & 干预措施
Aspirin and cytoreductive therapy (if applicable)
Patients who are randomized to this group will take a low-dose aspirin 81mg pill once per day (standard-of-care) for at least 6 months along with cytoreductive therapy, if applicable. Patients will then be treated and followed up as per standard of care at the discretion of his or her treating physician after the completion of the study.
干预措施: Aspirin 81 mg (Drug)
Apixaban and cytoreductive therapy (if applicable)
Patients who are randomized to this group will receive apixaban 2.5mg twice daily for at least 6 months along with standard intervention, cytoreductive therapy, if applicable. Patients will then be treated and followed up as per standard of care at the discretion of their treating physician after the completion of the study.
干预措施: Apixaban 2.5 MG Oral Tablet [ELIQUIS] (Drug)
结局指标
主要结局
Average monthly subject recruitment rate of all study sites during a 6-month recruitment period
时间窗: For the duration of study enrollment period: 6 months
Study Feasibility 1: Feasibility of recruitment
时间窗: For the duration of study enrollment period: 6 months
Feasibility of recruitment efforts will be determined by the proportion of patients contacted for screening versus those who are consented
Number of JAK2MPN patients recruited in 6 months in comparison to a target recruitment total of 39 prevalent cases and 5 incident cases at minimum
时间窗: For the duration of study enrollment period: 6 months
Study Feasibility 2: Feasibility of enrollment
时间窗: For the duration of study enrollment period: 6 months
Feasibility of enrollment will be determined by the proportion of patients consented vs those were enrolled and randomized
Study Feasibility 3: Patient retention rate
时间窗: For the duration of the study follow-up period: 7 months
This will be defined as the proportion of patients who started study intervention versus those who completed each of the study follow-up visits.
Quality of life on apixaban and aspirin will be measured through the use of the RAND 36-Item Health Survey (SF-36), with scores being transformed into a 0-100 scale where the higher the score the less disability.
时间窗: For the duration of the study follow-up period: 7 months
次要结局
- Study visit compliance as assessed by the number of study visits (in person and/or phone call) completed(For the duration of the study follow-up period: 7 months)
- Study drug compliance as assessed by the proportion of study drug prescribed to the patient versus the actual amount study drug taken by the patient(For the duration of the study follow-up period: 7 months)
- Percentage of incident and prevalent cases included in the study(For the duration of study enrollment period: 6 months)
- Rate of combined arterial and venous thrombotic events (MI, stroke, transient ischemic attack, peripheral arterial thrombosis, VTE)(For the duration of the study follow-up period: 7 months)
- Rate of major bleeding as per the International Society of Thrombosis and Hemostasis definitions(For the duration of the study follow-up period: 7 months)
- Rate of all-cause mortality(For the duration of the study follow-up period: 7 months)
- Rate of non-major clinically relevant bleeding as per the International Society of Thrombosis and Hemostasis definitions(For the duration of the study follow-up period: 7 months)