Study of Efficacy and Safety of Eltrombopag in Lower-risk MDS Patients With Platelet Transfusion Dependence
A Randomized, Double-blind, Placebo-controlled, Japan Local Phase II Clinical Study Comparing Eltrombopag Monotherapy Versus Placebo in Adult Lower-risk Myelodysplastic Syndromes (MDS) Patients With Platelet Transfusion Dependence
ClinicalTrials.gov Identifier: NCT04797000
Novartis Reference Number: CETB115L11201
Last Update: Jan 20, 2023
All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.
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).
Interventions
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 Locations
Contacts
Have a question?
Call 1-888-669-6682 or email [email protected]