GlaxoSmithKline (GSK) has initiated a Phase III trial, SUPPORT (TRC112121), of eltrombopag in patients with myelodysplastic syndromes (MDS), a type of cancer.
In patients with MDS, bone marrow does not make enough healthy blood cells and there are abnormal (blast) cells in the blood and/or bone marrow.
The trial is designed to assess the platelet supportive care effects of eltrombopag (Promacta/Revolade) in combination with azacitidine (the current standard of care) versus placebo in combination with azacitidine in intermediate-1, intermediate-2 or high risk patients with MDS.
The company said that the global trial will evaluate the proportion of patients who are platelet transfusion free during the first four cycles of treatment.
MDS usually manifests itself with one or more cytopenias, or reductions in the number of blood cells, and patients typically present with complications related to anaemia (fatigue), neutropenia (infections), or thrombocytopenia (bleeding).
The disease may evolve into acute myeloid leukaemia (AML) in about 45% of patients.
GSK senior vice-president of oncology R&D Rafael Amado said: "Currently, there are no approved treatments for MDS that stimulate platelet production.
"The initiation of this study is an important step toward providing a potential treatment option that may support platelets and avoid transfusions in thromobocytopenic patients with MDS undergoing chemotherapy treatment."
SUPPORT (StUdy of eltromboPag in myelodysPlastic SyndrOmes Receiving azaciTidine) is a Phase III, randomised, double-blind, placebo-controlled, multi-centre trial of eltrombopag or placebo in combination with azacitidine in MDS patients.
A total of 350 patients across 30 countries and 156 study sites with a baseline platelet count of < 75Gi/L, and intermediate-1, intermediate-2 or high risk MDS will be enrolled in the Phase III trial.
In the trial, eligible patients will be given either eltrombopag (200mg once-daily [100mg for East Asians]) plus azacitidine (75mg/m2 subcutaneously once-daily for seven days) every 28 days, for at least six cycles, or placebo plus azacitidine.
The trial's primary efficacy endpoint will determine the platelet supportive care effects of eltrombopag in combination with azacitidine versus placebo in combination with azacitidine.
Secondary objectives of the trial will compare overall survival, disease response, haematologic improvement, platelet and red blood cell transfusions, adverse events, safety and tolerability, health related quality of life and medical resource utilization.
Image: Enlarged spleen due to myelodysplastic syndrome; CT scan coronal section. Spleen in red, left kidney in green. Photo: courtesy of Tdvorak.