A Pilot Phase I Dose Finding Safety Study of a Thrombopoietin-receptor Agonist, Eltrombopag, in Patients With Myelodysplastic Syndrome Treated With Azacitidine (NMDSG10A)

NCT01481220 Phase 1 COMPLETED

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.

Details

Lead sponsor	Nordic MDS Group
Phase	Phase 1
Status	COMPLETED
Enrolment	12
Start date	2011-10
Completion	2013-05

Conditions

• Myelodysplastic Syndrome
• Thrombocytopenia

Interventions

• Eltrombopag

Primary outcomes

• Safety and tolerability parameters — week 26
  Including: * 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

Countries

Sweden