A Multicenter, Single-arm Study to Assess the Safety, Pharmacokinetics and Efficacy of Lenalidomide in Japanese Subjects With Low- or Intern=Mediate-1-risk Myelodysplastic Syndromes (MDS) Associated With a Deletion 5 (q31-33) Abnormality and Symptomatic Anemia
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Celgene
- 入组人数
- 11
- 试验地点
- 1
- 主要终点
- Number of Participants With Adverse Events (AE)
研究概览
简要总结
The purpose of this clinical experience study is to determine whether CC-5013 is safe and effective (to include studying the process by which a drug is absorbed, distributed, metabolized, and eliminated by the body [pharmacokinetics]) in Japanese subjects with low- or intermediate-1-risk MDS (IPSS risk categories) associated with a deletion 5(q31-33) abnormality and symptomatic anemia.
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
- 20 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Must understand and voluntarily sign an informed consent form.
- •Age ≥ 20 years at the time of signing the informed consent form.
- •Must be able to adhere to the study visit schedule and other protocol requirements.
- •Diagnosis of Myelodysplastic Syndrome (MDS) that meets International Prognostic Scoring System (IPSS) criteria for low- or intermediate-1-risk disease associated with a deletion 5(q31-33) abnormality
- •Symptomatic anemia secondary to MDS defined as:Untransfused Hb level < 10.0 g/dL and a Functional Assessment of Cancer Therapy (FACT)-anemia subscale score of ≤ 74 or Transfusion dependent anemia
排除标准
- •Pregnant or lactating females.
- •Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study.
- •Prior therapy with lenalidomide.
- •Patients with any of the following laboratory abnormalities within 14 days of starting study drug: Absolute Neutrophil Count (ANC) < 750 cells/μL (0.75 x 10^9/L) Platelet count < 50,000/μL (50x10^9/L) Serum creatinine > 2.5 mg/dL Aspartate Aminotransferase (AST) or Alanine Aminotransferase (ALT) > 3.0 x Upper Limit of Normal (ULN)
研究组 & 干预措施
Lenalidomide
Oral 10mg daily on Days 1-21 days every 28 days until disease progression/relapse or CC-5013 is permanently discontinued for any reason for up to 156 weeks (3 years).
干预措施: Lenalidomide (Drug)
结局指标
主要结局
Number of Participants With Adverse Events (AE)
时间窗: After the first study dose until 28 days after completion of/discontinuation from the study (maximum time on study was 155 weeks).
An AE that resulted in any of the following outcomes was defined as a serious adverse event (SAE): * Death; * Life-threatening event; * Any inpatient hospitalization or prolongation of existing hospitalization; * Persistent or significant disability or incapacity; * Congenital anomaly or birth defect; * Any other important medical event. The investigator determined the relationship of an AE to study drug based on the timing of the AE relative to drug administration and whether or not other drugs, therapeutic interventions, or underlying conditions could provide a sufficient explanation for the event. The severity of an AE was evaluated by the investigator according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) (Version 3.0) where Grade 1 = Mild, Grade 2 = Moderate, Grade 3 = Severe, Grade 4 = Life-threatening and Grade 5 = Death.
次要结局
- Maximum Observed Plasma Concentration (Cmax) of Lenalidomide(Days 1 and 4 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, and 12 hours post-dose.)
- Terminal Half-life (T1/2) of Lenalidomide(Days 1 and 4 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, and 12 hours post-dose.)
- Apparent Total Plasma Clearance (CL/F) of Lenalidomide(Days 1 and 4 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, and 12 hours post-dose.)
- Apparent Terminal Elimination Rate Constant of Lenalidomide(Days 1 and 4 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, and 12 hours post-dose.)
- Number of Participants With a Erythroid Response(Response was assessed every 28 days through Week 156.)
- Duration of Erythroid Response(From the first dose of study drug through Week 156)
- Area Under the Plasma Concentration-time Curve From Time Zero to the Last Measurable Concentration (AUCt) of Lenalidomide(Days 1 and 4 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, and 12 hours post-dose.)
- Time to Maximum Plasma Concentration (Tmax) of Lenalidomide(Days 1 and 4 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, and 12 hours post-dose.)
- Area Under the Plasma Concentration-time Curve From Time Zero to Infinity (AUC∞) of Lenalidomide(Day 1 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, 12 and 24 hours post-dose.)
- Time to Erythroid Response(From the first dose of study drug through Week 156)
- Area Under the Plasma Concentration-time Curve Over the Dosing Interval (AUCτ) of Lenalidomide(Days 1 and 4 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, 12 and 24 hours post-dose.)
- Apparent Volume of Distribution (VzF) of Lenalidomide(Days 1 and 4 at predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, and 12 hours post-dose.)
- Number of Participants With a Neutrophil Response(Response was assessed every 28 days through Week 156)
- Number of Participants With a Cytogenetic Response(Response was assessed every 12 weeks through Week 156)
- Change From Baseline in Percentage of Bone Marrow Erythroblasts(Baseline, at the end of Cycle 3 (Day 85) and Cycle 6 (Day 169).)
- Percentage of Bone Marrow Myeloblasts(Baseline, at the end of Cycle 3 (Day 85) and Cycle 6 (Day 169).)
- Percentage of Bone Marrow Promyelocytes(Baseline, at the end of Cycle 3 (Day 85) and Cycle 6 (Day 169).)
- Change From Baseline in Hemoglobin Concentration(Baseline and from Day1 until the maximum observed value (up to 155 weeks))
- Number of Participants With a Platelet Response(Response was assessed every 28 days through Week 156)