Safety Study of Eltrombopag Combined With Azacitidine to Treat Myelodysplastic Syndrome (MDS) (NMDSG10A)
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|ClinicalTrials.gov Identifier: NCT01481220|
Recruitment Status : Completed
First Posted : November 29, 2011
Last Update Posted : May 3, 2013
Patients with Myelodysplastic Syndromes (MDS) often suffer from low platelet levels which may lead to bleeding complications. Treatment with cytotoxic agents can decrease the platelet levels further. Eltrombopag is a relatively new drug that increases the platelet level in the blood by working directly on the bone marrow. It is available for treatment of the disease Immunological Thrombocytopenic Purpura (ITP). In this study patients with MDS and low platelet levels that are treated with the cytotoxic agent Azacitidine will also receive Eltrombopag. The administration of Eltrombopag to MDS patients treated with Azacitidine may result in less dose reductions and less treatment delays for Azacitidine and may reduce the need for thrombocyte transfusions and lower the risk of bleeding complications.
This is a phase I study, meaning that our major goal is to investigate the safety and tolerability for Eltrombopag in this patient group. It will also generate a basis for a phase II-III-study.
|Condition or disease||Intervention/treatment||Phase|
|Myelodysplastic Syndrome Thrombocytopenia||Drug: Eltrombopag||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||12 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Pilot Phase I Dose Finding Safety Study of a Thrombopoietin-receptor Agonist, Eltrombopag, in Patients With Myelodysplastic Syndrome Treated With Azacitidine|
|Study Start Date :||October 2011|
|Actual Primary Completion Date :||May 2013|
|Actual Study Completion Date :||May 2013|
|Experimental: Azacitidine + Eltrombopag||
In this study 4 different doses of oral eltrombopag (50mg, 100mg, 200mg and 300mg) will be tested. A modified 3+3 patient cohorts design will be used so no new patients are accepted to start on a higher dose without prior tolerance at the previous dose. Patients will be given eltrombopag once daily starting one week before the start of azacitidine treatment and then continue throughout the study, which duration will be approximately 3 months (three Azacytidine cycles). Patients will be evaluated continuously by clinical and laboratory assessments as well as bone marrow examinations during the treatment period until 4 weeks after discontinuation of Eltrombopag. Response, AEs/SAEs and DLTs will be monitored throughout the study.
- Safety and tolerability parameters [ Time Frame: week 26 ]
- Non-hematological clinical, laboratory Grade 3/Grade 4 toxicities
- Change in bone marrow or peripheral blood blast counts from baseline
- Adverse events and interactions at increasing doses of eltrombopag
- Azacitidine treatment delays and dose reductions [ Time Frame: week 26 ]
- Need for thrombocyte transfusions [ Time Frame: week 26 ]
- Bleeding complications [ Time Frame: week 26 ]
- Possible signs of antineoplastic effects (blood values and bone marrow picture) [ Time Frame: week 26 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01481220
|Uppsala, Stockholm, Göteborg, Umeå, Sweden|
|Study Director:||Tobias Svensson, M.D.||Nordic MDS Group|