Myelodysplastic Syndromes
Conditions
Keywords
Myelodysplastic Syndromes
Brief summary
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.
Interventions
DRUGOmacetaxine
Comparison of different doses of omacetaxine in combination with azacitidine and G-CSF
DRUGAzacitidine
Comparison of different doses of omacetaxine in combination with azacitidine and G-CSF
DRUGG-CSF
Comparison of different doses of omacetaxine in combination with azacitidine and G-CSF
Sponsors
Teva Pharmaceutical Industries, Ltd.
University of Florida
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The maximum tolerated dose (MTD) of omacetaxine in combination with azacitidine and G-CSF in patients with relapsed and/or refractory low- and intermediate-risk MDS. | 28 days |
Secondary
| Measure | Time frame |
|---|---|
| Number of participants with Hematologic Improvement (HI) as measured by hemoglobin, platelet count and neutrophil count. | 12 months |
| Number of participants with disease response as defined by International Working Group (IWG) 2006 criteria. | 12 months |
| Length of time of survival for participants | 24 months |
| Incidences of Grade 3/4 adverse events directly related to the drug combination | 24 months |
| Number of participants who achieve complete remission and how long the response lasts | 24 months |
Other
| Measure | Time frame |
|---|---|
| Number of participants who demonstrate changes in chromosome karyotype and genetic mutations | 12 months |
Countries
United States
Outcome results
None listed