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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

36 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-05-25

Study Completion Date

2026-12-09

Brief Summary

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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).

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Detailed Description

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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.

Conditions

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Myelodysplastic Syndromes

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Participants, investigators, site staffs and site monitors will remain blinded to the identity of the treatment from the time of randomization until database lock for final analysis, and the clinical study team members and anyone involved in the Japan registration activities will be blinded until database lock for the primary analysis. Randomization data are kept strictly confidential until the time of unblinding and will not be accessible by anyone else involved in the study with the following exceptions: DMC members and who will perform data analysis for DMC.

Study Groups

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Eltrombopag Arm

Participants randomized to a 1: 1 ratio will take eltrombopag.

Group Type EXPERIMENTAL

Eltrombopag

Intervention Type DRUG

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

Placebo Arm

Participants randomized to a 1: 1 ratio will take Placebo.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

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

Interventions

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Eltrombopag

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

Intervention Type DRUG

Placebo

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

Intervention Type DRUG

Other Intervention Names

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ETB115

Eligibility Criteria

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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)

Minimum Eligible Age

20 Years

Maximum Eligible Age

99 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No