Evaluation of CEP-701, an Orally Available JAK2 Tyrosine Kinase Inhibitor, as a Therapy for Patients With Myelofibrosis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 27
- 试验地点
- 1
- 主要终点
- Number of Participants With Objective Response
研究概览
简要总结
The goal of this clinical research study is to find out if CEP-701 can help control myelofibrosis (MF). The safety of CEP-701 will also be studied.
详细描述
The Study Drug:
CEP-701 is designed to help prevent a certain type of molecule (called a mutated JAK2 receptor) that is found on myelofibrosis cells from sending continuous chemical signals that lead to the growth of cancer cells.
Study Treatment:
If you are found to be eligible to take part in this study, you will take CEP-701 by mouth (in liquid form) 2 times a day (once in the morning and once in the evening) every day in 30-day repeating cycles. You should take each dose about 12 hours apart.
The study doctor or nurse will teach you and/or a caregiver or family member how to prepare each dose of the study drug, as well as how much should be taken each time. At each study visit, you will be supplied with enough syringes, dosing cups, and study drug to last until your next study visit. For each dose, you will use the syringe to draw the proper amount of CEP-701. You will add the entire contents of the syringe to an approved juice in 1 of the provided dosing cups. You should also drink an additional dosing cup of juice after taking the drug dose. The following juices (100% juice only) are approved for use with CEP-701: grape, pineapple, apple, V8 vegetable juice, and orange juice.
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 Chronic Idiopathic Myelofibrosis (CIMF) requiring therapy, including those 1) previously treated by CIMF-directed therapy and relapsed, intolerant, or refractory to therapy; or 2) if newly diagnosed then with intermediate or high risk according to Lille scoring system (adverse prognostic factors are: Hb < 10 g/dl, WBC < 4 or > 30 x 10^9/L; risk group: 0 = low, 1 = intermediate, 2 = high), or with symptomatic spleen that is >/= 10cm below costal margin. However, patients with asymptomatic intermediate risk disease are not eligible.
- •JAK2 mutation positive test
- •Age of at least 18 years
- •Eastern Cooperative Oncology Group (ECOG) performance status 0-2
- •Adequate liver and renal function: total bilirubin </=2.0 mg/dL, alanine aminotransferase (ALT or SGPT) </=2.0 x institutional upper limit of normal (ULN), and creatinine </=2.0 mg/dl
- •Patients must be at least 2 weeks from prior chemotherapy, biological therapy, radiation therapy, major surgery, or other investigational anticancer therapy that is considered MF-directed, and have recovered from prior toxicities to Grade 0-
- •Concurrent therapy with supportive care medications (hydroxyurea, anagrelide) is allowed during the study.
- •All men of reproductive potential and women of child-bearing potential (WOCBP) must agree to practice effective contraception (iud, birth control pill, latex condoms, diaphragm) during the entire study period and for one month after the study ends, unless documentation of infertility exists. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately. WOCBP are women who are not menopausal for 12 months or no previous surgical sterilization."
- •Ability to understand and willingness to sign the informed consent form
- •Not willing to undergo, not a candidate for, or not having a donor for, a bone marrow transplant
排除标准
- •Pregnant or nursing women, due to the unknown effects of therapy on the developing fetus or newborn infant.
- •Patients diagnosed with another malignancy - unless following curative intent therapy the patient has been disease free for at least 3 years. Patients with early stage squamous cell carcinoma of the skin, basal cell carcinoma of the skin, or cervical intraepithelial neoplasia (CIN) are eligible for this study
- •Any condition, including serious medical condition, laboratory abnormality, or psychiatric illness, which places the subject at unacceptable risk as judged by the Principal Investigator, if he/she was to participate in the study
- •Known positive for Human immunodeficiency virus (HIV) or infectious hepatitis, type A, B or C
- •Presence of any gastrointestinal condition or concomitant medication use (e.g. coumadin) that would render a patient at high risk for gastrointestinal bleeding as judged by treating physician
- •History of any upper or lower gastrointestinal bleeding in the 6 months prior to enrollment
- •Elevated international normalized ratio (INR) or Partial thromboplastin time (PTT)
研究组 & 干预措施
CEP-701
80 mg orally twice a day for 30 days
干预措施: CEP-701 (Drug)
结局指标
主要结局
Number of Participants With Objective Response
时间窗: Response assessed after each 3 cycles (cycle = 30 days)
Objective response = Complete Response, absence sign/symptoms of disease (without use of growth factors, hydroxyurea, anagrelide, or transfusions for \> 1 month); Partial Response, absence of progressive disease (PD), and improvement in 2+ parameters (if abnormal): Absolute neutrophil count (ANC), hemoglobin, platelets, transfusions, splenomegaly, or bone marrow blasts; Clinical Improvement, absence of PD, and improvement in 1 parameter: ANC, hemoglobin, platelets, transfusions, splenomegaly, or bone marrow blasts). \[International Working Group on Myelofibrosis Research and Treatment\]
次要结局
未报告次要终点