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.
調査の概要
詳細な説明
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.
研究の種類
介入
入学 (実際)
130
段階
- フェーズ 3
連絡先と場所
このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。
研究場所
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Athens、ギリシャ
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Goudi-Athens、ギリシャ
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Larisa、ギリシャ
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Patra、ギリシャ
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Thessalonikis、ギリシャ
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Berlin、ドイツ
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Dresden、ドイツ
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Duisburg、ドイツ
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Düsseldorf、ドイツ
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Dÿsseldorf、ドイツ
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München、ドイツ
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Oldenburg、ドイツ
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Würzburg、ドイツ
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Amiens、フランス
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Angers Cedex 9、フランス
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Bobigny、フランス
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Colmar、フランス
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Paris Cedex 10、フランス
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Pessac Cedex、フランス
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Pierre Benite Cedex、フランス
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Saint Priest En Jarez、フランス
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Tours Cedex 9、フランス
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Vandoeuvre Les Nancy、フランス
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Plovdiv、ブルガリア
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Sofia、ブルガリア
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Varna、ブルガリア
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Ekaterinburg、ロシア連邦
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St. Petersburg、ロシア連邦
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参加基準
研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。
適格基準
就学可能な年齢
18年歳以上 (大人、高齢者)
健康ボランティアの受け入れ
いいえ
受講資格のある性別
全て
説明
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
研究計画
このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。
研究はどのように設計されていますか？
デザインの詳細
- 主な目的：処理
- 割り当て：ランダム化
- 介入モデル：並列代入
- マスキング：4倍
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
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実験的：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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プラセボコンパレーター：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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この研究は何を測定していますか？
主要な結果の測定
結果測定 |
時間枠 |
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Erythroid response
時間枠：at week 24
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at week 24
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二次結果の測定
結果測定 |
時間枠 |
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Maintenance of Erythroid response
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：at baseline, week 24 and week 48
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at baseline, week 24 and week 48
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Drug consumption
時間枠：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
時間枠：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
時間枠：every 4 weeks from baseline to week 48
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every 4 weeks from baseline to week 48
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協力者と研究者
ここでは、この調査に関係する人々や組織を見つけることができます。
出版物と役立つリンク
研究に関する情報を入力する責任者は、自発的にこれらの出版物を提供します。これらは、研究に関連するあらゆるものに関するものである可能性があります。
研究記録日
これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。
主要日程の研究
研究開始
2011年10月1日
一次修了 (実際)
2015年1月1日
研究の完了 (実際)
2016年1月1日
試験登録日
最初に提出
2011年6月23日
QC基準を満たした最初の提出物
2011年6月23日
最初の投稿 (見積もり)
2011年6月27日
学習記録の更新
投稿された最後の更新 (見積もり)
2016年3月16日
QC基準を満たした最後の更新が送信されました
2016年3月14日
最終確認日
2016年3月1日
詳しくは
この情報は、Web サイト clinicaltrials.gov から変更なしで直接取得したものです。研究の詳細を変更、削除、または更新するリクエストがある場合は、register@clinicaltrials.gov。 までご連絡ください。 clinicaltrials.gov に変更が加えられるとすぐに、ウェブサイトでも自動的に更新されます。