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: Sep 06, 2022

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).

Condition

Myelodysplastic Syndromes

Phase

Phase 2

Overall status

Recruiting

Start date

May 25, 2021

Completion date

Jan 31, 2025

Gender

All

Age(s)

20 Years and older (Adult, Older Adult)

Interventions

Drug

Eltrombopag

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

Drug

Placebo

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

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.