Study Description
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). 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.
Interventions
Eltrombopag
Placebo
Eligibility Criteria
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) 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.
Study Location
Novartis Investigative Site
Recruiting
Kurume-city,Fukuoka,830-8543,Japan
Novartis Investigative Site
Recruiting
Fukushima city,Fukushima,960 1295,Japan
Novartis Investigative Site
Recruiting
Osaka Sayama,Osaka,589 8511,Japan
Novartis Investigative Site
Recruiting
Matsumoto-city,Nagano,399-8701,Japan
Novartis Investigative Site
Recruiting
Gifu shi,Gifu,500 8513,Japan
Novartis Investigative Site
Recruiting
Osaka,534-0021,Japan
Novartis Investigative Site
Recruiting
Mito,Ibaraki,310-0015,Japan
Novartis Investigative Site
Recruiting
Isehara,Kanagawa,259-1193,Japan
Novartis Investigative Site
Recruiting
Sendai city,Miyagi,980 8574,Japan
Novartis Investigative Site
Recruiting
Nagasaki-city,Nagasaki,852-8501,Japan
Novartis Investigative Site
Recruiting
Itabashi ku,Tokyo,173 8606,Japan
Novartis Investigative Site
Recruiting
Shimonoseki,Yamaguchi,750-0061,Japan
Novartis Investigative Site
Recruiting
Aomori,030 8553,Japan
Novartis Investigative Site
Recruiting
Narita,Chiba,286-8523,Japan
Novartis Investigative Site
Recruiting
Kanazawa,Ishikawa,920-0853,Japan
Novartis Investigative Site
Recruiting
Yamagata,990 9585,Japan
Novartis Investigative Site
Recruiting
Bunkyo-ku,Tokyo,113-8603,Japan
Novartis Investigative Site
Recruiting
Nishinomiya,Hyogo,663 8501,Japan
Novartis Investigative Site
Recruiting
Kumamoto-city,Kumamoto,860-0008,Japan
Novartis Investigative Site
Recruiting
Chiba,260-0852,Japan
Novartis Investigative Site
Recruiting
Ohtake,Hiroshima,739-0696,Japan
Worldwide Contacts
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