Status:
COMPLETED
A Single Arm Pilot Study of Azacitidine in Myelodysplastic Syndromes (MDS) / Acute Myeloid Leukaemia (AML), With Eltrombopag Support for Thrombocytopenia
Lead Sponsor:
Peter MacCallum Cancer Centre, Australia
Collaborating Sponsors:
GlaxoSmithKline
Celgene Corporation
Conditions:
Myelodysplastic Syndromes (MDS)
Acute Myeloid Leukaemia (AML)
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief Summary
Myelodysplastic Syndrome (MDS) is a disease of the bone marrow characterized by anemia,neutropenia, and thrombocytopenia (low red blood cell, white blood cell, and platelet counts). MDS patients with ...
Eligibility Criteria
Inclusion
- Patients with low platelet count (\<=150 x109/L)and in addition disease diagnosis of either MDS, or nonproliferative CMML or low marrow blast count AML not suitable for induction chemotherapy
- Age \>18 years
- ECOG score 0-2 at screening
- Life expectancy ≥12 weeks
- Ability to comply with the adequate contraception in patients of childbearing potential.
Exclusion
- Subjects with the diagnosis acute promyelocytic leukaemia
- Prior treatment with azacitidine or any other methyl-transferase inhibitor (e.g. decitabine)
- Prior treatment with eltrombopag, romiplostim, or other TPO-receptor agonist
- AML or MDS requiring cytoreductive therapy (eg hydroxyurea, ara-c, thioguanine etc) in the month prior to study entry
- Known uncontrolled medical conditions which may compromise participation in this study including but not limited to:
- Poorly controlled congestive heart failure: ejection fraction \<30% measured in past 6 months) or NYHA class IV
- Arrhythmia known to increase the risk of thromboembolic events.
- Unstable angina or an ischaemic cardiac event requiring hospital admission in the previous 12 months.
- Unresolved GI disease that may significantly alter the absorption of eltrombopag
- Known pro-thrombotic condition as defined by a history ≥1 unprovoked deep venous thrombosis or pulmonary embolism, or any DVT/PE with a procoagulant condition screen suggesting the presence of a procoagulant condition (prothrombin gene mutation homozygosity, factor V leiden homozygosity, antithrombin deficiency, lupus anticoagulant syndrome).
- History of Ischaemic neurological event (TIA or stroke) within the preceding 2 years.
- Inadequate renal function (eGFR \<30 ml/min by Cockcroft-Gault (C-G) formula, or as measured by 24 hour urinary creatinine clearance)
- Inadequate hepatic function:
- bilirubin ≥1.5xULN - ≤80µmol/L acceptable if attributed to haemolysis, ineffective erythropoiesis or iron overload). This applies also for patients with Gilbert's Syndrome.
- AST or ALT ≥2xULN (≤3xULN acceptable if attributed to transfusion-associated iron overload)
- Patients with known liver cirrhosis.
- Other concurrent severe and/or uncontrolled medical conditions including a history of malignancy other than MDS/AML in the preceding 2 years requiring chemotherapy and/or radiotherapy. Non melanotic skin cancers requiring low dose local radiotherapy or topical agents are allowed on study if considered clinically stable or healed.
- Women who are pregnant or breast-feeding.
- Treatment with growth factors such as erythropoietin, GCSF or stem cell factor in the 21 days prior to commencement of study therapy.
- Active or uncontrolled infections.
- Subjects with known HIV infection.
- Has any other clinically important abnormalities as determined by the investigator that may interfere with his or her participation in or compliance with the study
- Bone marrow fibrosis that leads to an inability to aspirate marrow for quality cytological assessment, termed a "dry tap".
- Presence of any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule. This condition must be discussed with the patient prior to signing consent and registration in the trial.
- Splenomegaly \>14cm on the screening ultrasound examination.
Key Trial Info
Start Date :
December 1 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 1 2015
Estimated Enrollment :
25 Patients enrolled
Trial Details
Trial ID
NCT01488565
Start Date
December 1 2010
End Date
May 1 2015
Last Update
February 15 2016
Active Locations (2)
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1
Peter MacCallum Cancer Centre
East Melbourne, Victoria, Australia, 3002
2
Cabrini Hospital
Malvern, Victoria, Australia, 3144