A Phase II, Prospective, Open Label Study (PO-MMM-PI-0011) to Determine the Safety and Efficacy of Pomalidomide (CC-4047) in Subjects With Primary, Post Polycythemia Vera, or Post Essential Thrombocythemia Myelofibrosis (PMF; Post-PV MF, or Post-ET MF)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 70
- 试验地点
- 1
- 主要终点
- Number of Participants With Best Overall Response
研究概览
简要总结
The goal of this clinical research study is to learn if CC-4047 (now called pomalidomide) and prednisone can help to control MMM. The safety of this therapy will also be studied.
详细描述
The Study Drug:
CC-4047 is a drug that may also affect the growth of blood vessels that support tumor growth. If these blood vessels stop growing, it may stop cancer cell growth.
Prednisone is a corticosteroid that is similar to a natural hormone made by your body. Prednisone is often given in combination with other chemotherapy to treat cancer.
Study Drug Administration:
On Days 1- 28 of every 28-day study "cycle," you will take CC-4047 capsule(s) by mouth. You should take the capsule(s), about the same time every day. The capsule(s) should not be opened, broken, or chewed. CC-4047 should be taken without food, at least 2 hours before or 2 hours after a meal. If a dose of CC-4047 is missed, it should be taken as soon as possible on the same day. If it is missed for the entire day, it should not be made up, rather it should be taken at the next scheduled time point.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 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
- •Must be >/= 18 years of age at the time of voluntarily signing an Institutional Review Board/Independent Ethics Committee (IRB/IEC) - approved informed consent form.
- •Must be diagnosed with myelofibrosis requiring therapy including myelofibrosis with myeloid metaplasia (MMM), de novo presentation (i.e. agnogenic myeloid metaplasia [AMMM], and developing after an antecedent history of Polycythemia vera (i.e., post-polycythemic myeloid metaplasia [PPMM]), or essential Polycythemia (i.e., post thrombocythemic myeloid metaplasia [PTMM]).
- •Screening total hemoglobin level < 10 g/dL or transfusion-dependent anemia defined as per International Working Group (IWG) criteria (transfusion dependency defined by a history of a least 2 units of red blood cell transfusions in the last 28 days for hemoglobin < 8.5 g/dL that was not associated with overt bleeding)
- •Must have adequate organ function as demonstrated by the following </= 14 days prior to starting study drug: ·Alanine transaminase (ALT) (SGOT) and Aspartate aminotransferase (AST) (SGPT) </= 3 x upper limit of normal (ULN), [unless upon judgment of the treating physician, it is believed to be due to extramedullary hematopoiesis (EMH)] ·Total bilirubin < 3 x ULN or Direct Bilirubin < 2 x ULN ·Serum creatinine </= 2.5 mg/dL ·Absolute neutrophil count >/= 1,000/µL (>/=1.0 x 10^9/L) ·Platelet count >/= 50,000/µL (>/=50 x 10^9/L)
- •Subjects must be willing to receive transfusion of blood products
- •Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0, 1, or 2 at screening.
- •Must be willing to adhere to the study visit schedule and other protocol requirements.
- •No active malignancies with the exception of basal cell or squamous cell carcinoma of the skin, or carcinoma (in situ) of the cervix or breast
- •All study participants must be registered into the mandatory POMALYST REMS™ program, and be willing and able to comply with the requirements of the POMALYST REMS™ program.
- •Females of reproductive potential (FCBP†) must adhere to the scheduled pregnancy testing as required in the POMALYST REMS™ program. Able to take aspirin (81 or 325 mg) daily as prophylactic anticoagulation (patients intolerant to ASA may use warfarin or low molecular weight heparin).
排除标准
- •Known positive status for HIV, hepatitis B carrier, or active hepatitis C infection.
- •The use of any growth factors, cytotoxic chemotherapeutic agents (e.g. hydroxyurea), corticosteroids, or experimental drug or therapy within 14 days of starting CC-4047 and/or lack of recovery from all toxicity from previous therapy to grade 1 or better.
- •Any serious medical condition or psychiatric illness that would prevent, (as judged by the treating physician) the subject from signing the informed consent form or any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
- •Pregnant or lactating females
- •Prior use of CC-4047
- •Currently enrolled on another clinical trial or receiving investigational agent
研究组 & 干预措施
Group 2 CC-4047
CC-4047 0.5 mg orally daily starting on day 1 through 28.
干预措施: CC-4047 (Drug)
Group 3 CC-4047 + Prednisone
CC-4047 0.5 mg orally daily starting on day 1 through 28. Prednisone given during first 3 cycles of therapy. It will be dosed orally at the dose of 30 mg/day during cycle 1, 15 mg/day during cycle 2, and 15 mg every other day during cycle 3, and then it will be discontinued.
干预措施: CC-4047 (Drug)
Group 3 CC-4047 + Prednisone
CC-4047 0.5 mg orally daily starting on day 1 through 28. Prednisone given during first 3 cycles of therapy. It will be dosed orally at the dose of 30 mg/day during cycle 1, 15 mg/day during cycle 2, and 15 mg every other day during cycle 3, and then it will be discontinued.
干预措施: Prednisone (Drug)
Group 1 CC-4047
CC-4047 3.0 mg orally daily starting on day 1 through 21.
干预措施: CC-4047 (Drug)
结局指标
主要结局
Number of Participants With Best Overall Response
时间窗: 6 months
Primary endpoint is best overall response. An evaluable subject classified as a treatment success for the primary endpoint if the subject's best overall response is clinical improvement (CI) as determined by International Working Group Criteria over the first 6 cycles of study treatment. International Working Group (IWG) consensus criteria for treatment response in myelofibrosis - Clinical improvement (CI) in anemia 1/ A minimum 20g/L increase in hemoglobin level or 2. becoming transfusion independent for at least 8 week duration.
次要结局
未报告次要终点