Study of Efficacy and Safety of Eltrombopag in Lower-risk MDS Patients With Platelet Transfusion … (NCT04797000) | Clinical Trial Compass
Active — Not RecruitingPhase 2
Study of Efficacy and Safety of Eltrombopag in Lower-risk MDS Patients With Platelet Transfusion Dependence
Japan36 participantsStarted 2021-05-25
Plain-language summary
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).
Who can participate
Age range
20 Years – 100 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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) or erythroid mutation agent in anemic patients or granulocyte-colony stimulating factor (G-CSF) in patients with severe neutropenia and recurrent infections is allowed if at stable dos…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Percentage of participants who achieve platelet transfusion independence at Week 24