NIPH Clinical Trials Search

JAPANESE
国立保健医療科学院
JRCT ID: jRCT2021210004

Registered date:26/04/2021

A randomized, double-blind, placebo-controlled, Japan local phase II clinical study comparing the eltrombopag monotherapy versus placebo in adult lower risk myelodysplastic syndromes (MDS) patients with platelet transfusion dependence

Basic Information

Recruitment status Recruiting
Health condition(s) or Problem(s) studiedlower-risk myelodysplastic syndromes
Date of first enrollment25/05/2021
Target sample size44
Countries of recruitment
Study typeInterventional
Intervention(s)Eltrombopag or placebo will be started with a dose of 25 mg/day. The dose can be increased if response on platelet counts is insufficient.

Outcome(s)

Primary Outcomeplatelet transfusion independence, platelet response and clinically significant bleeding events
Secondary Outcome

Key inclusion & exclusion criteria

Age minimum>= 20age old
Age maximumNot applicable
GenderBoth
Include criteria- Japanese adult patients aged 20 years or older - Patients diagnosed with MDS according to the WHO classification revised 4th edition (International Agency for Research on Cancer 2017) 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 (definition: receiving platelet transfusion regularly with a frequency of 2 or more transfusions within 4 weeks prior to randomization. Platelet transfusion should be performed for a patient with platelet counts < 20 x 109/L or in the presence of hemorrhagic symptoms and platelet counts < 30 x 109/L). Meeting the criteria with transient platelet transfusions for reasons not directly due to MDS (e.g. bleeding, surgical procedure, hemolysis, infections) will not be regarded as platelet transfusion dependence. Refractory, intolerant to, or ineligible for MDS treatments (except supportive treatment e.g. ESAs, G-CSF) per local standard medical practice.
Exclude criteria- Patients with a history of prior administration of eltrombopag, romiplostim, or other TPO-RA - Therapy-related MDS per WHO classification revised 4th edition (International Agency for Research on Cancer 2017) - MDS/myeloproliferative neoplasms including chronic myelomonocytic leukaemia per the WHO classification revised 4th edition (International Agency for Research on Cancer 2017) - MDS with excess blasts (EB) per WHO classification revised 4th edition (International Agency for Research on Cancer 2017) - 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)

Related Information

Contact

Public contact
Name Kazuyuki Suzuki
Address Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo 105-6333, Japan Tokyo Japan 105-6333
Telephone +81-120-003-293
E-mail rinshoshiken.toroku2@novartis.com
Affiliation Novartis Pharma. K.K.
Scientific contact
Name Kazuyuki Suzuki
Address Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo 105-6333, Japan Tokyo Japan 105-6333
Telephone +81-120-003-293
E-mail rinshoshiken.toroku2@novartis.com
Affiliation Novartis Pharma. K.K.