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A randomized, double-blind, placebo-controlled, Japan local phase II clinical study comparing the eltrombopag monotherapy versus placebo in adult lower risk myelodysplastic syndromes (MDS) patients with platelet transfusion dependence

A randomized, double-blind, placebo-controlled, Japan local phase II clinical study comparing the eltrombopag monotherapy versus placebo in adult lower risk myelodysplastic syndromes (MDS) patients with platelet transfusion dependence

Yamauchi Kyosuke

Novartis Pharma. K.K.

Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo 105-6333, Japan

rinshoshiken.toroku2@novartis.com

Yamauchi Kyosuke

Novartis Pharma. K.K.

Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo 105-6333, Japan

+81-120-003-293

rinshoshiken.toroku2@novartis.com

Not Recruiting

May. 20, 2021

Interventional

randomized controlled trial

double blind

placebo control

parallel assignment

treatment purpose

- Japanese adult patients aged 20 years or older
- Patients diagnosed with MDS according to the WHO classification revised 4th edition (International Agency for Research on Cancer 2017) 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 (definition: receiving platelet transfusion regularly with a frequency of 2 or more transfusions within 4 weeks prior to randomization. Platelet transfusion should be performed for a patient with platelet counts < 20 x 109/L or in the presence of hemorrhagic symptoms and platelet counts < 30 x 109/L). Meeting the criteria with transient platelet transfusions for reasons not directly due to MDS (e.g. bleeding, surgical procedure, hemolysis, infections) will not be regarded as platelet transfusion dependence.
Refractory, intolerant to, or ineligible for MDS treatments (except supportive treatment e.g. ESAs, G-CSF) per local standard medical practice.

- Patients with a history of prior administration of eltrombopag, romiplostim, or other TPO-RA
- Therapy-related MDS per WHO classification revised 4th edition (International Agency for Research on Cancer 2017)
- MDS/myeloproliferative neoplasms including chronic myelomonocytic leukaemia per the WHO classification revised 4th edition (International Agency for Research on Cancer 2017)
- MDS with excess blasts (EB) per WHO classification revised 4th edition (International Agency for Research on Cancer 2017)
- 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)

Both

lower-risk myelodysplastic syndromes

Eltrombopag or placebo will be started with a dose of 25 mg/day. The dose can be increased if response on platelet counts is insufficient.

platelet transfusion independence, platelet response and clinically significant bleeding events

+81-23-633-1122

April. 06, 2021

Yes

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

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