Know Cancer

forgot password

A Single Arm Pilot Study of Azacitidine in Myelodysplastic Syndrome / Acute Myeloid Leukaemia, With Eltrombopag Support for Thrombocytopenia.

Phase 2
18 Years
Open (Enrolling)
Myelodysplastic Syndromes (MDS), Acute Myeloid Leukaemia (AML)

Thank you

Trial Information

A Single Arm Pilot Study of Azacitidine in Myelodysplastic Syndrome / Acute Myeloid Leukaemia, With Eltrombopag Support for Thrombocytopenia.

Inclusion Criteria:

- 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

Exclusion Criteria:

- Subjects with the diagnosis acute promyelocytic leukaemia

- Prior treatment with azacitidine or any other methyl-transferase inhibitor (e.g.

- 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

- 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.

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Number of events of non-haematological toxicities related to the combination of eltrombopag and azacitidine

Outcome Description:

The number of events of Grade III/IV non-haematological toxicities will be measured based on the possibly, probably or definitely relatedness to the combination of eltrombopag and azacitidine

Outcome Time Frame:

At 6 cycles of therapy (approx 6 months)

Safety Issue:


Principal Investigator

Michael Dickinson, Dr

Investigator Role:

Principal Investigator

Investigator Affiliation:

Peter MacCallum Cancer Centre, Australia


Australia: Department of Health and Ageing Therapeutic Goods Administration

Study ID:




Start Date:

December 2010

Completion Date:

May 2014

Related Keywords:

  • Myelodysplastic Syndromes (MDS)
  • Acute Myeloid Leukaemia (AML)
  • Leukemia
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid
  • Myelodysplastic Syndromes
  • Preleukemia
  • Thrombocytopenia