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Active, not recruiting Phase 2

Study of Efficacy and Safety of Eltrombopag in Lower-risk MDS Patients With Platelet Transfusion Dependence

NCT04797000 · tracked via the Priya Life Science Japan tracker
Phase
Phase 2
Started
2021-05-25
Last updated
2026-09-22

Condition(s) studied

Myelodysplastic Syndromes

Investigational drug(s) / intervention(s)

Eltrombopag →Placebo

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

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

Study summary

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).

Eligibility

Sex
ALL
Min age
20 Years
Max age
100 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

  • Percentage of participants who achieve platelet transfusion independence at Week 24 — 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.

Trial sites (20)

FacilityCityRegionStatus
Novartis Investigative Site Narita Chiba
Novartis Investigative Site Kurume Fukuoka
Novartis Investigative Site Ohtake Hiroshima
Novartis Investigative Site Nishinomiya Hyōgo
Novartis Investigative Site Kanazawa Ishikawa-ken
Novartis Investigative Site Isehara Kanagawa
Novartis Investigative Site Yokohama Kanagawa
Novartis Investigative Site Sendai Miyagi
Novartis Investigative Site Nagasaki Nagasaki
Novartis Investigative Site Sakai Osaka
Novartis Investigative Site Bunkyo-ku Tokyo
Novartis Investigative Site Itabashi Ku Tokyo
Novartis Investigative Site Shimonoseki Yamaguchi
Novartis Investigative Site Aomori Japan
Novartis Investigative Site Chiba Japan
Novartis Investigative Site Fukushima Japan
Novartis Investigative Site Gifu Japan
Novartis Investigative Site Kumamoto Japan
Novartis Investigative Site Osaka Japan
Novartis Investigative Site Yamagata Japan
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04797000 on ClinicalTrials.gov ↗ ← All trials in Japan