Clinical trial NCT02835794: A Clinical Trial of Omacetaxine, Azacitidine, and Growth-Colony Stimulating Factor (G-CSF) for Myelodysplastic Syndromes (MDS)
Official title: A Phase I/II Clinical Trial of Omacetaxine, Azacitidine, and G-CSF for Relapsed and/or Refractory Myelodysplastic Syndromes
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE1
PHASE2
Conditions: Myelodysplastic Syndromes
Keywords: Myelodysplastic Syndromes
Lead sponsor: University of Florida
Sponsor class: OTHER
Collaborators: name: Teva Pharmaceutical Industries, Ltd.
First posted: 2016-07-18
Enrollment: 0
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: 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.
timeFrame: 28 days
Secondary outcomes:
measure: Number of participants with Hematologic Improvement (HI) as measured by hemoglobin, platelet count and neutrophil count.
timeFrame: 12 months
measure: Number of participants with disease response as defined by International Working Group (IWG) 2006 criteria.
timeFrame: 12 months
measure: Number of participants who achieve complete remission and how long the response lasts
timeFrame: 24 months
measure: Length of time of survival for participants
timeFrame: 24 months
measure: Incidences of Grade 3/4 adverse events directly related to the drug combination
timeFrame: 24 months
Other outcomes:
measure: Number of participants who demonstrate changes in chromosome karyotype and genetic mutations
timeFrame: 12 months
Locations:
city: Gainesville
country: United States
facility: University of Florida Health Shands Cancer Hospital
state: Florida
Study officials:
affiliation: University of Florida
name: Maxim N. Norkin, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: