Status:
ACTIVE_NOT_RECRUITING
Study of Efficacy and Safety of Eltrombopag in Lower-risk MDS Patients With Platelet Transfusion Dependence
Lead Sponsor:
Novartis Pharmaceuticals
Conditions:
Myelodysplastic Syndromes
Eligibility:
All Genders
20-99 years
Phase:
PHASE2
Brief 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).
Detailed Description
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 transfusio...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
May 25 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 9 2026
Estimated Enrollment :
36 Patients enrolled
Trial Details
Trial ID
NCT04797000
Start Date
May 25 2021
End Date
December 9 2026
Last Update
October 21 2025
Active Locations (20)
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1
Novartis Investigative Site
Narita, Chiba, Japan, 286-8523
2
Novartis Investigative Site
Kurume, Fukuoka, Japan, 830-8543
3
Novartis Investigative Site
Fukushima, Fukushima, Japan, 960 1295
4
Novartis Investigative Site
Ohtake, Hiroshima, Japan, 739-0696