A Clinical Trial of Omacetaxine, Azacitidine, and Growth-Colony Stimulating Factor (G-CSF) for Myelodysplastic Syndromes (MDS)

A Phase I/II Clinical Trial of Omacetaxine, Azacitidine, and G-CSF for Relapsed and/or Refractory Myelodysplastic Syndromes

The purpose of this study is to determine the safety and establish the maximum tolerated dose (MTD) of omacetaxine in combination with azacitidine and G-CSF in patients with relapsed and/or refractory MDS.

Inclusion Criteria:

• Age >= 18 years;

• Informed consent;

• Low- and intermediate-risk MDS that has failed to achieve any hematologic improvement after at least 4 cycles of induction therapy or has relapsed after any duration of any hematologic response. Prior therapy with azanucleosides (i.e., azacitidine, decitabine), biologic therapies (i.e., lenalidomide, rigosertib) and hematopoietic cell transplant are permissible;

• Performance status must be Eastern Cooperative Oncology Group (ECOG) 0, 1, or 2;

• Women of child-bearing potential (i.e., women who are pre-menopausal or not surgically sterile) may participate, provided they meet the following conditions:

  • Must agree to use physician-approved contraceptive methods throughout the study and for three months following the last dose of omacetaxine and
  • Must have a negative serum or urine pregnancy test within 7 days prior to beginning treatment on this trial;

• Males with female partners of child-bearing potential must agree to use physician-approved contraceptive methods throughout the study and should avoid conceiving a child for 6 months following the last dose of omacetaxine.

Exclusion Criteria:

• Subjects who are eligible for hematopoietic stem cell transplant;
• History of atrial fibrillation related to azanucleoside therapy in the past;
• Active, uncontrolled, clinically significant infection;
• Pregnant and nursing patients are excluded because the effects of omacetaxine on a fetus or nursing child are unknown;
• Treatment with any anticancer therapy (standard or investigational) within the previous 14 days prior to the first dose of study drug or less than full recovery from the clinically significant toxic effects of that treatment.