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Undersøgelse af effektivitet og sikkerhed af Eltrombopag hos MDS-patienter med lavere risiko med blodpladetransfusionsafhængighed

16. oktober 2026 opdateret af: Novartis Pharmaceuticals

Et randomiseret, dobbeltblindt, placebokontrolleret, Japansk lokalt fase II klinisk studie, der sammenligner Eltrombopag monoterapi versus placebo i voksne med lavere risiko for myelodysplastiske syndromer (MDS) patienter med blodpladetransfusionsafhængighed

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).

Studieoversigt

Status

Aktiv, ikke rekrutterende

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

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.

Undersøgelsestype

Interventionel

Tilmelding (Faktiske)

36

Fase

  • Fase 2

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiesteder

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

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

20 år til 100 år (Voksen, Ældre voksen)

Tager imod sunde frivillige

Ingen

Køn, der er berettiget til at studere

Alle

Beskrivelse

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.

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: Randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: Eltrombopag Arm

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

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

Andre navne:
  • ETB115
Placebo komparator: Placebo Arm

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

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

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Percentage of participants who achieve platelet transfusion independence at Week 24
Tidsramme: 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

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Time to platelet transfusion independence
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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))
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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.

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Novartis Pharmaceuticals

Efterforskere

  • Studieleder: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

25. maj 2021

Primær færdiggørelse (Faktiske)

26. april 2025

Studieafslutning (Anslået)

2. december 2026

Datoer for studieregistrering

Først indsendt

4. marts 2021

Først indsendt, der opfyldte QC-kriterier

11. marts 2021

Først opslået (Faktiske)

15. marts 2021

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

20. oktober 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

16. oktober 2026

Sidst verificeret

1. oktober 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • CETB115L11201

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

JA

IPD-planbeskrivelse

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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