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

Phase 1
Withdrawn
Conditions
Myelodysplastic Syndromes
Interventions
Registration Number
NCT02835794
Lead Sponsor
University of Florida
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.

Detailed Description

Not available

Recruitment & Eligibility

Status
WITHDRAWN
Sex
All
Target Recruitment
Not specified
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.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Arm 1OmacetaxineOmacetaxine - escalating doses subcutaneous twice daily on Days 1-5 and 8-12 Azacitidine 50 mg/m2 subcutaneous/intravenous daily on Days 8-12 G-CSF 5mcg/kg subcutaneous daily on Days 15-19 and 22-26
Arm 1G-CSFOmacetaxine - escalating doses subcutaneous twice daily on Days 1-5 and 8-12 Azacitidine 50 mg/m2 subcutaneous/intravenous daily on Days 8-12 G-CSF 5mcg/kg subcutaneous daily on Days 15-19 and 22-26
Arm 1AzacitidineOmacetaxine - escalating doses subcutaneous twice daily on Days 1-5 and 8-12 Azacitidine 50 mg/m2 subcutaneous/intravenous daily on Days 8-12 G-CSF 5mcg/kg subcutaneous daily on Days 15-19 and 22-26
Primary Outcome Measures
NameTimeMethod
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 Outcome Measures
NameTimeMethod
Number of participants with disease response as defined by International Working Group (IWG) 2006 criteria.12 months
Number of participants who achieve complete remission and how long the response lasts24 months
Number of participants with Hematologic Improvement (HI) as measured by hemoglobin, platelet count and neutrophil count.12 months
Length of time of survival for participants24 months
Incidences of Grade 3/4 adverse events directly related to the drug combination24 months

Trial Locations

Locations (1)

University of Florida Health Shands Cancer Hospital

🇺🇸

Gainesville, Florida, United States

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