NCT01873703已完成2 期
A Phase 2 Randomized Double-Blind Placebo-Controlled Study of Pracinostat in Combination With Azacitidine in Patients With Previously Untreated International Prognostic Scoring System (IPSS) Intermediate Risk-2 or High-Risk Myelodysplastic Syndrome (MDS)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 102
- 试验地点
- 24
- 主要终点
- Estimate efficacy
研究概览
简要总结
The purpose of this randomized, double-blind, placebo-controlled study is to determine the safety and efficacy of pracinostat compared to placebo when combined with azacitidine, and FDA approved treatment for Myelodysplastic Syndrome (MDS).
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
- Triple (Participant, Care Provider, Investigator)
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
- •Voluntary written informed consent
- •Histologically or cytologically documented diagnosis of MDS (any French-American-British [FAB] classification subtype; that is classified as intermediate 2 (1.5 to 2.0 points) or high risk (≥2.5 points) according to the International Prognostic Scoring System risk category, with >5% and <30% blasts, and a peripheral blast count of <20,000
- •Bone marrow aspirate smears and bone marrow biopsies within 28 days of first study treatment
- •There must be a clinical indication for treatment with azacitidine.
- •Previously untreated with hypomethylating agents (prior therapy with transfusions, hematopoietic growth factors, or immunosuppressive therapy is allowed)
- •Eastern Cooperative Oncology Group performance status of 0, 1, or 2
- •Adequate organ function as evidenced by:
- •Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤2.5 x the upper limit of normal (ULN) (≤5 x ULN for patients with hepatic metastases
- •Total bilirubin ≤1.5 x ULN or total bilirubin of 2, whichever is higher
- •Serum creatinine <2 mg/dL, or creatinine clearance ≤1.5 x ULN
- •QTcF interval ≤470 msec
- •Female or male patients ≥18 years-of-age
- •Male patients who are surgically sterile or willing to use adequate contraceptive measures or abstain from heterosexual intercourse during the entire study treatment period
- •Female patients who are surgically sterile or post menopausal or female patients who are not of child-bearing potential and female patients of child-bearing potential who agree to use adequate contraceptive measures or abstain from intercourse during the study treatment period, who are not breastfeeding, and who have had a negative serum pregnancy test ≤7 days prior to first study treatment.
- •Willingness and ability to comply with the trial and follow-up procedures
排除标准
- •Received any of the following within the specified time frame prior to administration of study medication:
- •Any investigational agent within 14 days or 5 half-lives prior to first study treatment, whichever is longer
- •Previous therapy for malignancy within 21 days prior to first study treatment, including any chemotherapy, immunotherapy, biological or hormonal therapy (6 weeks for nitrosoureas or mitomycin C)
- •Hydroxyurea within 48 hours prior to first study treatment
- •Hematopoietic growth factors: erythropoietin, granulocyte colony stimulating factor (G-CSF), granulocyte macrophage colony stimulating factor (GM-CSF), or thrombopoietin receptor agonists at least 7 days (14 days for Aranesp) prior to study enrollment
- •Major surgery within 4 weeks prior to first study treatment
- •Patients that have not recovered from side effects of previous therapy
- •Cardiopulmonary function exclusion:
- •Current unstable arrhythmia requiring treatment
- •History of symptomatic congestive heart failure (New York Heart Association Classes III or IV)
- •History of myocardial infarction within 6 months of enrollment
- •Current unstable angina
- •Concomitant treatment with histone deacetylase (HDAC) inhibitors or drugs with significant action as HDAC inhibitors, such as valproic acid, is not permitted
- •Clinical evidence of central nervous system involvement
- •Patients with gastrointestinal (GI) tract disease, causing the inability to take oral medication, malabsorption syndrome, a requirement for IV alimentation, prior surgical procedures affecting absorption, uncontrolled inflammatory GI disease (e.g., Crohn's disease, ulcerative colitis).
- •Active infection with HIV or chronic hepatitis B or C
- •Life-threatening illness unrelated to cancer, or any serious medical or psychiatric illness that could, in the investigator's opinion, potentially interfere with participation in this study
- •Presence of a malignant disease within the last 12 months, with the exception of adequately treated in-situ carcinomas, basal or squamous cell carcinoma, or non-melanomatous skin cancer
- •Inability (including psychological, familial, sociological, or geographical conditions) to comply with trial and/or follow-up procedures
研究组 & 干预措施
pracinostat plus azacitadine
Experimental
60 mg of pracinostat by mouth 3 times a week for 3 weeks followed by 1 week of rest repeated every 28 days.
75 mg/m2 Azacitidine for 7 days of each 28 day cycle, via subcutaneous (SC) injection or intravenous infusion if SC injections are intolerable
干预措施: pracinostat (Drug)
pracinostat plus azacitadine
Experimental
60 mg of pracinostat by mouth 3 times a week for 3 weeks followed by 1 week of rest repeated every 28 days.
75 mg/m2 Azacitidine for 7 days of each 28 day cycle, via subcutaneous (SC) injection or intravenous infusion if SC injections are intolerable
干预措施: Azacitidine (Drug)
Placebo with Azacitadine
Placebo Comparator
Placebo by mouth 3 times a week for 3 weeks followed by 1 week of rest repeated every 28 days.
75 mg/m2 Azacitidine for 7 days of each 28 day cycle, via subcutaneous (SC) injection or intravenous infusion if SC injections are intolerable
干预措施: Placebo (Drug)
Placebo with Azacitadine
Placebo Comparator
Placebo by mouth 3 times a week for 3 weeks followed by 1 week of rest repeated every 28 days.
75 mg/m2 Azacitidine for 7 days of each 28 day cycle, via subcutaneous (SC) injection or intravenous infusion if SC injections are intolerable
干预措施: Azacitidine (Drug)
结局指标
主要结局
Estimate efficacy
时间窗: 6 months
Estimate the relative efficacy, measured by complete remission rate of treatment wiht pracinostat plus azacitidine versus placebo plus azacitidine
次要结局
- Overall response rate(6 months)
- Hematologic Improvement(6 months)
- Duration of response(6 months)
- Progression free survival(12 months)
- Rate of leukemic transformation(6 - 24 months)
- Overall survival(6-24 months)
- AE profile(12 months)
研究者
研究点 (24)
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