An Efficacy Study for Epoetin Alfa in Anemic Patients With Myelodysplastic Syndromes
2016年3月14日 更新者：Janssen-Cilag International NV
A Randomized, Double-Blind, Placebo-Controlled, Multicenter Study Evaluating Epoetin Alfa Versus Placebo in Anemic Patients With IPSS Low- or Intermediate-1-Risk Myelodysplastic Syndromes
The purpose of this study is to demonstrate that epoetin alfa works better than placebo in improving anemia in patients with lower-risk myelodysplastic syndromes (MDS).
The safety of epoetin alfa will also be evaluated.
研究概览
详细说明
This is a randomized (the treatment you receive will be assigned by chance), double-blind (neither physician nor patient knows the name of the assigned drug), placebo-controlled (comparison with patients that receive treatment without active ingredient), multicenter study of epoetin alfa in anemic patients who are diagnosed with myelodysplastic syndromes (MDS) according to protocol-specified criteria.
This study includes a 3-week prerandomization phase, a 24-week treatment phase and a 24-week treatment extension phase.
All patients enrolled in the study will complete an end-of-study visit 4 weeks after the last dose of study drug (Week 28 or Week 52), or 4 weeks after early withdrawal (unless the reason for early withdrawal is withdrawal of consent).
Between 125 and 159 patients will be enrolled in the treatment phase of the study.
During the screening phase, which will take place within 2 weeks before starting study drug, the study doctor will do tests to see if the patient is suitable for this study.
Patients meeting entry criteria for the study will then be randomly assigned to one of the 2 treatment groups.
This means that each patient who is allowed to join the study is put into a group by chance, like flipping a coin.
Group 1 patients will receive epoetin alfa 450 or increased up to 1050 International Units (IU) per kg body weight administered by subcutaneous injection (injection beneath the skin) using pre-filled syringes.
Injections will be done once every week at a weight-based dose regimen (the total weekly dose received will depend on your weight) with a possible total maximum dose of 40,000 IU once every week for the first 8 weeks of the treatment phase and 80,000 IU once every week at any other time during the study.
Group 2 patients will receive a matching volume of placebo administered once every week by subcutaneous injection.
The chance that the patient will get epoetin alfa is 2 to 1. Doses of study drug will be withheld, decreased, or increased on the basis of erythroid response, weekly hemoglobin concentrations monitored in patients and predefined dose adjustment guidelines.
Patients will see the study doctor every 4 weeks for a period of 24 weeks.
At each visit the patient will undergo a full hematologic evaluation, serum chemistry evaluation, measurement of blood pressure and pulse rate, recording of blood product transfusions and transfusion complications, adverse events, concomitant therapies and an evaluation for disease progression.
The patient's Erythroid response will be assessed at Week 8 and every 4 weeks thereafter, until Week 24.
Blinded study treatment will be administered to all patients at Week 24.
However, at the end of the treatment phase (after the Week 24 response assessment), only responders will enter the double-blind treatment extension phase to measure the duration of response.
Patients will continue to receive the same treatment, in the same blinded fashion, and at the same dose as received at Week 24, and will return to the study center every 4 weeks, until Week 48, for assessment of the Erythroid response and the evaluations as described above.
For all non-responders at Week 24 the treatment code will be broken after Week 28 assessments.
For responders at Week 48, the treatment code will be broken after the Week 48 visit, following completion of the response assessment.
The treatment code will not be broken for subjects who discontinue study treatment before Week 24, irrespective of whether they are responders or nonresponders.
For these subjects, the blind will not be broken until all subjects have completed the study and the database is final.
Once the patient stops receiving doses of study drug, he/she will be asked to see the study doctor for the safety follow-up visit, which is scheduled 4 weeks after the last dose of study drug.
Safety will be monitored throughout the study at predetermined intervals and as clinically indicated by physical examination, laboratory tests and evaluation of adverse events.
An Independent Data Monitoring Committee (IDMC) will periodically review study data and for the assessment of disease progression.
The total duration of study participation will be for about 30 or 54 weeks.
研究类型
介入性
注册 (实际的)
130
阶段
- 第三阶段
联系人和位置
本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。
学习地点
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Ekaterinburg、俄罗斯联邦
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St. Petersburg、俄罗斯联邦
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Plovdiv、保加利亚
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Sofia、保加利亚
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Varna、保加利亚
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Athens、希腊
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Goudi-Athens、希腊
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Larisa、希腊
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Patra、希腊
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Thessalonikis、希腊
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Berlin、德国
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Dresden、德国
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Duisburg、德国
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Düsseldorf、德国
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Dÿsseldorf、德国
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München、德国
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Oldenburg、德国
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Würzburg、德国
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Amiens、法国
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Angers Cedex 9、法国
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Bobigny、法国
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Colmar、法国
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Paris Cedex 10、法国
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Pessac Cedex、法国
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Pierre Benite Cedex、法国
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Saint Priest En Jarez、法国
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Tours Cedex 9、法国
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Vandoeuvre Les Nancy、法国
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参与标准
研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
资格标准
适合学习的年龄
18年 及以上 (成人、年长者)
接受健康志愿者
不
有资格学习的性别
全部
描述
Inclusion Criteria:
- Diagnosis of MDS according to World Health Organization or French-American-British pathologic classification (confirmed via bone marrow aspirate/biopsy) within 12 weeks prior to screening
- Documentation of an International Prognostic Scoring System score indicating Low- or Intermediate-1-risk disease within 12 weeks prior to screening
- Hemoglobin concentration at screening and baseline (before the first dose of study drug) of 10.0 g/dL or less
- Screening serum erythropoietin concentration of less than 500 mU/mL
- Red Blood Cell transfusion requirement of less than or equal to 4 red blood cell units over the last 8 weeks before randomization
Exclusion Criteria:
- Anemia attributed to factors other than MDS (including hemolysis, chronic renal failure, hepatitis, gastrointestinal bleeding)
- Secondary MDS (ie, MDS arising after chemotherapy, immunotherapy or radiation therapy/exposure)
- History of malignancy, except in situ skin basal cell carcinoma or carcinoma in situ of the cervix or breast curatively treated
- Prior therapy with any erythropoiesis-stimulating agent (ESA) (including innovative ESAs and biosimilar ESAs for approved indications or for investigational use) in the last 8 weeks before randomization
- Prior use of approved or experimental agents for the treatment of MDS
学习计划
本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。
研究是如何设计的？
设计细节
- 主要用途：治疗
- 分配：随机化
- 介入模型：并行分配
- 屏蔽：四人间
武器和干预
参与者组/臂 |
干预/治疗 |
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实验性的：Epoetin alfa
Group 1: Epoetin alfa type = range unit= IU/Kg number= 337.5 to 1050 IU/Kg form= solution for injection route= subcutaneous use weekly injections (max 40 000 IU per week for first 8 weeks of treatment max 80 000 IU per week later) using pre-filled 1mL 40 000 IU syringes for 24 to 48 weeks
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type = range, unit= IU/Kg, number= 337.5 to 1050 IU/Kg, form= solution for injection, route= subcutaneous use, weekly injections (max 40,000 IU per week for first 8 weeks of treatment, max 80,000 IU per week later) using pre-filled 1mL 40,000 IU syringes for 24 to 48 weeks
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安慰剂比较：No treatment
Group 2: Placebo form= solution for injection route= subcutaneous use weekly injections for 24 to 48 weeks
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form= solution for injection, route= subcutaneous use, weekly injections for 24 to 48 weeks
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研究衡量的是什么？
主要结果指标
结果测量 |
大体时间 |
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Erythroid response
大体时间：at week 24
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at week 24
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次要结果测量
结果测量 |
大体时间 |
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Maintenance of Erythroid response
大体时间：every 4 weeks from week 24 to week 48
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every 4 weeks from week 24 to week 48
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Duration of response
大体时间：every 4 weeks after week 24
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every 4 weeks after week 24
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Time to first Red Blood Cell transfusion
大体时间：from baseline to study end (week 28 for non responders, week 54 for responders or 4 weeks after early withdrawal)
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from baseline to study end (week 28 for non responders, week 54 for responders or 4 weeks after early withdrawal)
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Transfusion-free intervals
大体时间：from baseline to study end (week 28 for non responders, week 54 for responders or 4 weeks after early withdrawal)
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from baseline to study end (week 28 for non responders, week 54 for responders or 4 weeks after early withdrawal)
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Number of Red Blood Cell units transfused
大体时间：from baseline to study end (week 28 for non responders, week 54 for responders or 4 weeks after early withdrawal)
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from baseline to study end (week 28 for non responders, week 54 for responders or 4 weeks after early withdrawal)
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Quality of life as measured by Functional Assessment of Cancer Therapy-Anemia/Fatigue (FACT-An) questionnaire
大体时间：at baseline, week 24 and week 48
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at baseline, week 24 and week 48
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Quality of life as measured by EuroQol 5-dimension (EQ-5D) questionnaire
大体时间：at baseline, week 24 and week 48
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at baseline, week 24 and week 48
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Drug consumption
大体时间：every 4 weeks from baseline to week 48
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every 4 weeks from baseline to week 48
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Duration of hospitalization
大体时间：every 4 weeks from baseline to week 48
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every 4 weeks from baseline to week 48
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Number and duration of medical care encounters
大体时间：every 4 weeks from baseline to week 48
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every 4 weeks from baseline to week 48
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合作者和调查者
在这里您可以找到参与这项研究的人员和组织。
出版物和有用的链接
负责输入研究信息的人员自愿提供这些出版物。这些可能与研究有关。
研究记录日期
这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。
研究主要日期
学习开始
2011年10月1日
初级完成 (实际的)
2015年1月1日
研究完成 (实际的)
2016年1月1日
研究注册日期
首次提交
2011年6月23日
首先提交符合 QC 标准的
2011年6月23日
首次发布 (估计)
2011年6月27日
研究记录更新
最后更新发布 (估计)
2016年3月16日
上次提交的符合 QC 标准的更新
2016年3月14日
最后验证
2016年3月1日
更多信息
此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.