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血小板輸血依存症の低リスクMDS患者におけるエルトロンボパグの有効性と安全性の研究

2026年10月16日 更新者：Novartis Pharmaceuticals

血小板輸血依存症の成人低リスク骨髄異形成症候群（MDS）患者におけるエルトロンボパグ単剤療法とプラセボを比較する無作為化二重盲検プラセボ対照日本国内第II相臨床試験

This study is designed to evaluate the efficacy and safety of eltrombopag monotherapy in Japanese adult patients with platelet transfusion-dependent lower-risk Myelodysplastic syndromes (LR-MDS).

調査の概要

状態

積極的、募集していない

条件

介入・治療

詳細な説明

This is a randomized, double-blind, placebo-controlled, Japanese local phase II study to evaluate the efficacy and safety of eltrombopag monotherapy in Japanese adult patients with platelet transfusion-dependent lower-risk MDS (IPSS-R very low, low, intermediate risk with bone marrow blast count < 5% and cytogenetic very good, good or intermediate risk). Platelet transfusion dependence at baseline is defined as receiving platelet transfusion regularly with a frequency of 2 or more times within 4 weeks prior to randomization. Platelet transfusion should be performed for a patient with platelet counts < 20 X 10^9/L, or with hemorrhagic symptoms and platelet counts < 30 X 10^9/L.

The primary objective is to demonstrate superiority of eltrombopag versus placebo in terms of the proportion of participants who achieve platelet transfusion independence at Week 24.

研究の種類

介入

入学 (実際)

36

段階

  • フェーズ2

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

  • 日本
      • Aomori、日本、0308553
        • Novartis Investigative Site
      • Chiba、日本、2600852
        • Novartis Investigative Site
      • Fukushima、日本、9601295
        • Novartis Investigative Site
      • Gifu、日本、5008513
        • Novartis Investigative Site
      • Kumamoto、日本、8600008
        • Novartis Investigative Site
      • Osaka、日本、5340021
        • Novartis Investigative Site
      • Yamagata、日本、9909585
        • Novartis Investigative Site
    • Chiba
      • Narita、Chiba、日本、2868523
        • Novartis Investigative Site
    • Fukuoka
      • Kurume、Fukuoka、日本、8308543
        • Novartis Investigative Site
    • Hiroshima
      • Ohtake、Hiroshima、日本、7390696
        • Novartis Investigative Site
    • Hyōgo
      • Nishinomiya、Hyōgo、日本、6638501
        • Novartis Investigative Site
    • Ishikawa-ken
      • Kanazawa、Ishikawa-ken、日本、9208530
        • Novartis Investigative Site
    • Kanagawa
      • Isehara、Kanagawa、日本、2591193
        • Novartis Investigative Site
      • Yokohama、Kanagawa、日本、2210855
        • Novartis Investigative Site
    • Miyagi
      • Sendai、Miyagi、日本、9808574
        • Novartis Investigative Site
    • Nagasaki
      • Nagasaki、Nagasaki、日本、8528501
        • Novartis Investigative Site
    • Osaka
      • Sakai、Osaka、日本、5900197
        • Novartis Investigative Site
    • Tokyo
      • Bunkyo-ku、Tokyo、日本、1138603
        • Novartis Investigative Site
      • tabashi City、Tokyo、日本、1738606
        • Novartis Investigative Site
    • Yamaguchi
      • Shimonoseki、Yamaguchi、日本、7500061
        • Novartis Investigative Site

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

20年～100年 (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:

  • Patients diagnosed with MDS according to the WHO classification revised 4th edition by investigator assessment with one of the following prognostic risk categories, based on the International

Prognostic Scoring System (IPSS-R):

  • very low (0-1.5)

  • low (2-3)

  • intermediate risks (3.5-4.5) All following criteria for prognostic variables per IPSS-R should be met.

  • Bone marrow blast < 5% (per both investigator's assessment and central review)

  • Cytogenetic very good, good or intermediate risk corresponding to IPSS-R

    • Platelet transfusion dependence
    • Refractory, intolerant to, or ineligible for MDS treatments
    • Eastern Cooperative Oncology Group (ECOG) Performance status (PS) 0 or 1

Exclusion Criteria:

  • Patients with a history of prior administration of eltrombopag, romiplostim, or other TPO-RA
  • Therapy-related MDS per WHO classification revised 4th edition
  • MDS/myeloproliferative neoplasms including chronic myelomonocytic leukaemia per the WHO classification revised 4th edition
  • MDS with excess blasts (EB) per WHO classification revised 4th edition
  • Known history of IPSS-R high or very high risk MDS
  • Currently receiving treatments for MDS (e.g., HMA, cyclosporine A (CsA) or lenalidomide). Supportive treatment with erythropoiesis-stimulating agents (ESAs) or erythroid mutation agent in anemic patients or granulocyte-colony stimulating factor (G-CSF) in patients with severe neutropenia and recurrent infections is allowed if at stable dosage for 3 months prior to screening and continued at the same dosing/schedule until the optimal dose of eltrombopag has been established.
  • Patients scheduled for hematopoietic stem cell transplantation
  • Bone marrow fibrosis that leads to an inability to aspirate adequate bone marrow sample
  • Known thrombophilic risk factors (except in cases where potential benefits of participating in the study outweighed potential risks of thromboembolic events (TEE), as determined by the investigator)

Other protocol-defined inclusion/exclusion criteria may apply.

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：なし（オープンラベル）

武器と介入

参加者グループ / アーム
介入・治療
実験的：Eltrombopag Arm

Participants randomized to a 1: 1 ratio will take eltrombopag.
薬：Eltrombopag

Eltrombopag comes in 12.5 mg & 25 mg tablets and is taken orally once per day (QD)

他の名前：
  • ETB115
プラセボコンパレーター：Placebo Arm

Participants randomized to a 1: 1 ratio will take Placebo.
薬：Placebo

Placebo comes in 12.5 mg & 25 mg tablets and is taken orally once per day (QD)

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
メジャーの説明
時間枠
Percentage of participants who achieve platelet transfusion independence at Week 24
時間枠：Week 24

Platelet transfusion independence is defined as the absence of platelet transfusion for at least 8 weeks. Platelet count should be higher than the baseline count.
Week 24

二次結果の測定

結果測定
メジャーの説明
時間枠
Time to platelet transfusion independence
時間枠：Year 1, Year 2

This is defined as time from the date of randomization to the first day of a platelet transfusion-free period lasting at least 8 weeks.
Year 1, Year 2
Duration of platelet transfusion independence
時間枠：Year 1, Year 2

This is defined for participants who have achieved platelet transfusion independence only and will be calculated from the first date of platelet transfusion-free resulting in achievement of transfusion independence to the date before becoming platelet transfusion dependent.
Year 1, Year 2
Percentage of participants with platelet transfusion independence
時間枠：Year 1, Year 2

Platelet transfusion independence is defined as the absence of platelet transfusion for at least 8 weeks. Platelet count should be higher than the baseline count.
Year 1, Year 2
Percentage of participants with platelet transfusion frequency reduction at Week 24
時間枠：Week 24

This is defined as a reduction by at least 50 percent during the 4 prior weeks as compared to the 4 weeks prior to randomization.
Week 24
Percentage of participants with platelet response (Hematologic improvement (HI) - platelet))
時間枠：Week 24, Year 1, Year 2

Platelet response is defined as HI-platelet per International Working Group criteria.
Week 24, Year 1, Year 2
Time to platelet response
時間枠：Week 24, Year 1, Year 2

This is defined as time from the date of randomization to the first date of achieving the platelet response criteria per modified IWG criteria.
Week 24, Year 1, Year 2
Duration of platelet response
時間枠：Week 24, Year 1, Year 2

This is defined for participants who have achieved platelet response and will be calculated from the first date of achieving the platelet response criteria to the date before loss of platelet response per modified IWG criteria.
Week 24, Year 1, Year 2
Percentage of hematologic improvement-erythroid and -neutrophil
時間枠：Week 24, Year 1, Year 2

per modified IWG criteria at 24 weeks, Year 1 and 2
Week 24, Year 1, Year 2
Percentage of participants with disease progression excluding relapse after HI
時間枠：Week 24, Year 1, Year 2

This is with regards to blast counts for both investigator assessment and central review per modified IWG criteria.
Week 24, Year 1, Year 2
Percentage of participants with relapse after HI and transfusion independence
時間枠：Week 24, Year 1, Year 2

The percentage of participants with relapse after HI and transfusion independence at Week 24, Year 1 and 2.
Week 24, Year 1, Year 2
Percentage of participants with progression to Acute myeloid leukemia (AML)
時間枠：Week 24, Year 1, Year 2

This is defined as ≥ 20% blasts at Week 24, Year 1 and 2 for both investigator assessment and central review per WHO classification revised 4th edition.
Week 24, Year 1, Year 2
Leukemia free survival (LFS)
時間枠：Week 24, Year 1, Year 2

This is defined as time from the date of randomization to progression to AML per WHO classification revised 4th edition of ≥ 20 percent blasts or death due to any cause for both investigator assessment and central review.
Week 24, Year 1, Year 2
Clinically significant bleeding events
時間枠：Week 24, Year 1, Year 2

This is defined as ≥ grade 2 events as per WHO bleeding scale.
Week 24, Year 1, Year 2
Overall survival (OS)
時間枠：Week 24, Year 1, Year 2

OS is defined as time from randomization to death due to any cause.
Week 24, Year 1, Year 2
Quality of Life measured using QLQ-C30
時間枠：Baseline, every 4 weeks until week 52, every 8 weeks after week 52 up to end of treatment or end of post-treatment follow-up, approximately 3.5 years.

The changes from baseline to each visit where measured using QLQ-C30. EORTC-QLQ-C30 is a 30-item questionnaire developed to assess the quality of life of cancer patients. Subject's responses to 28 questions about their physical functioning, disease symptoms, global health status and utilities are scored on a 4-point scale (1=Not at all to 4=Very much), a low score indicates a high / healthy level of functioning. And the responses to 2 questions about health-related QoL are scored on a 7-point scale (1=Very poor to 7=Excellent), a high score indicates a high / healthy level of functioning.

Using linear transformation, raw scores are standardized, so that scores range from 0 to 100.
Baseline, every 4 weeks until week 52, every 8 weeks after week 52 up to end of treatment or end of post-treatment follow-up, approximately 3.5 years.
Pharmacokinetics (PK): Cmax
時間枠：Intensive: pre-dose, 1, 2, 4, 6, 8, and 24 hours post-dose. Trough: 15th days after starting the initial dose or each dose modification until reaching the maximum dose.

Full PK profile of eltrombopag over 24 hours at steady state at the initial dose, and at 125 mg and 150 mg when applicable, in subset of participants.
Intensive: pre-dose, 1, 2, 4, 6, 8, and 24 hours post-dose. Trough: 15th days after starting the initial dose or each dose modification until reaching the maximum dose.
PK: Tmax
時間枠：Intensive: pre-dose, 1, 2, 4, 6, 8, and 24 hours post-dose. Trough: 15th days after starting the initial dose or each dose modification until reaching the maximum dose.

Full PK profile of eltrombopag over 24 hours at steady state at the initial dose, and at 125 mg and 150 mg when applicable, in subset of participants.
Intensive: pre-dose, 1, 2, 4, 6, 8, and 24 hours post-dose. Trough: 15th days after starting the initial dose or each dose modification until reaching the maximum dose.
PK: AUC
時間枠：Intensive: pre-dose, 1, 2, 4, 6, 8, and 24 hours post-dose. Trough: 15th days after starting the initial dose or each dose modification until reaching the maximum dose.

Full PK profile of eltrombopag over 24 hours at steady state at the initial dose, and at 125 mg and 150 mg when applicable, in subset of participants.
Intensive: pre-dose, 1, 2, 4, 6, 8, and 24 hours post-dose. Trough: 15th days after starting the initial dose or each dose modification until reaching the maximum dose.
Trough concentrations of eltrombopag at steady state
時間枠：Intensive: pre-dose, 1, 2, 4, 6, 8, and 24 hours post-dose. Trough: 15th days after starting the initial dose or each dose modification until reaching the maximum dose.

at each dose level in all the participants
Intensive: pre-dose, 1, 2, 4, 6, 8, and 24 hours post-dose. Trough: 15th days after starting the initial dose or each dose modification until reaching the maximum dose.

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。

スポンサー

Novartis Pharmaceuticals

捜査官

  • スタディディレクター：Novartis Pharmaceuticals、Novartis Pharmaceuticals

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 (実際)

2021年5月25日

一次修了 (実際)

2025年4月26日

研究の完了 (推定)

2026年12月2日

試験登録日

最初に提出

2021年3月4日

QC基準を満たした最初の提出物

2021年3月11日

最初の投稿 (実際)

2021年3月15日

学習記録の更新

投稿された最後の更新 (実際)

2026年10月20日

QC基準を満たした最後の更新が送信されました

2026年10月16日

最終確認日

2026年10月1日

詳しくは

本研究に関する用語

キーワード

追加の関連 MeSH 用語

その他の研究ID番号

  • CETB115L11201

個々の参加者データ (IPD) の計画

個々の参加者データ (IPD) を共有する予定はありますか?

はい

IPD プランの説明

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

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いいえ

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いいえ

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