A Pilot Study of Thalidomide as an Inhibitor of Angiogenesis in the Treatment of Myelofibrosis With Myeloid Metaplasia (MMM)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 43
- 试验地点
- 1
- 主要终点
- Confirmed Response, i.e., an objective status of complete or partial response, recorded on 2 consecutive evaluations at least 4 weeks apart.
研究概览
简要总结
Phase II trial to study the effectiveness of thalidomide in treating patients who have myelofibrosis. Thalidomide may stop the growth of myelofibrosis by stopping blood flow to the cancer cells.
详细描述
PRIMARY OBJECTIVES:
I. To investigate whether thalidomide, a potent inhibitor of angiogenic and fibrogenic growth factors, is an effective therapeutic agent in patients with MMM. Specifically, to assess whether thalidomide improves anemia and/or organomegaly in patients with MMM.
II. To assess the effects of thalidomide on the myelofibrotic stroma with respect to microvascular architecture and angiogenesis, collagen and reticulin deposition, and the expression of the mediating growth factors bFGF, TGF-b, and PDGF, and their respective receptors.
OUTLINE: This is a multicenter study.
Patients receive oral thalidomide once daily for 1 year in the absence of disease progression or unacceptable toxicity. Patients with stable or responding disease may receive 1 additional year of therapy.
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
- •Histologically confirmed myelofibrosis with myeloid metaplasia
- •Agnogenic myeloid metaplasia
- •Post-polycythemic myeloid metaplasia
- •Post-thrombocythemic myeloid metaplasia
- •No metastatic carcinoma, lymphoma, myelodysplasia, hairy cell leukemia, mast cell disease, acute leukemia (including M7), or acute myelofibrosis
- •No chromosomal translocation t(9;22) or bcr/abl gene rearrangement
- •Presence of reticulin fibrosis in bone marrow and leukoerythroblastosis and dacrocytosis in peripheral blood
- •Presence of anemia (hemoglobin less than 10 g/dL), palpable splenomegaly, or hepatomegaly
- •Performance status - ECOG 0-2
- •Absolute neutrophil count greater than 750/mm^3
- •Platelet count less than 400,000/mm^3
- •WBC less than 50,000/mm^3
- •Bilirubin no greater than 2 mg/dL (if total bilirubin elevated, direct bilirubin must be normal)
- •AST no greater than 3 times upper limit of normal (ULN)
- •Alkaline phosphatase no greater than 3 times ULN
- •Creatinine no greater than 1.5 mg/dL
- •Creatinine clearance at least 60 mL/min
- •Not pregnant or nursing
- •Negative pregnancy test
- •Fertile women must use at least 1 highly active method AND 1 additional effective method of contraception for at least 4 weeks before study, during study, and for at least 4 weeks after study
- •Fertile men must use effective contraception during study and for at least 4 weeks after study
- •No uncontrolled infection
- •No concurrent condition that would preclude study
- •No peripheral neuropathy
- •At least 1 month since prior interferon, pirfenidone, anagrelide, or epoetin alfa
- •At least 1 month since prior hydroxyurea or other chemotherapy
- •At least 1 month since prior corticosteroids or androgen derivatives
排除标准
- 未提供
研究组 & 干预措施
Treatment (thalidomide)
Patients receive oral thalidomide once daily for 1 year in the absence of disease progression or unacceptable toxicity. Patients with stable or responding disease may receive 1 additional year of therapy.
干预措施: thalidomide (Drug)
Treatment (thalidomide)
Patients receive oral thalidomide once daily for 1 year in the absence of disease progression or unacceptable toxicity. Patients with stable or responding disease may receive 1 additional year of therapy.
干预措施: laboratory biomarker analysis (Other)
结局指标
主要结局
Confirmed Response, i.e., an objective status of complete or partial response, recorded on 2 consecutive evaluations at least 4 weeks apart.
时间窗: Up to 5 years
The proportion of successes will be estimated using the Binomial point estimator (number of successes divided by the total number of evaluable patients) and 95% confidence intervals calculated using the Duffy-Santner algorithm for multi-stage designs.
次要结局
- Survival(Number of days from registration date to the date of death or last follow-up, assessed up to 5 years)
- Time to progression(Number of days from registration date to the date of disease progression or last follow-up, assessed up to 5 years)
- Response duration(Number of days from the first date that objective status = complete or partial response was recorded to the date of disease progression or date of death, whichever comes first, assessed up to 5 years)