A Phase II, Multicenter, Single-arm Study to Assess the Efficacy and Safety of Oral Rigosertib in Transfusion-dependent Low or Intermediate-1 (Any Cytogenetics) or Trisomy 8 Intermediate-2 Myelodysplastic Syndrome Patients Based on IPSS Classification
This will be a Phase II open-label, multicenter (up to 5 centers), single-arm study. Sixty
transfusion-dependent patients with MDS classified as Low or Int-1 risk (any cytogenetics)
or trisomy 8 Int-2 by International Prognostic Scoring System (IPSS) will be enrolled to
receive on an outpatient basis 560 mg rigosertib BID for 14 consecutive days of a 21-day
cycle. (Note: Protocol was amended to delete the arm in which administration of rigosertib
was a continuous regimen on days 1 to 21 of 21-day cycle because higher incidence of urinary
symptoms was observed in this arm.)
Patients will be stratified on prior treatment with azacitidine and/or decitabine and/or
lenalidomide and/or erythropoietin.
Patients will remain treated on study until 2006 Internation Working Group (IWG) progression
criteria are met or until death from any cause.
All study participants will be allowed, as medically justified, access to RBC and platelet
transfusions, and to filgrastim [G-CSF]. Erythropoiesis-stimulating agents (ESAs) will not
be allowed during the initial 3 cycles and then will only be allowed in patients with
hemoglobin levels of less than 9 g/dL. Rigosertib dosing adjustment policies are described
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Number of units of red blood cell transfusions
Number of units of red blood cell transfusions will be compared with the pretreatment transfusion number in the previous 8 weeks.
François E. Wilhelm, MD, PhD
Onconova Therapeutics, Inc.
United States: Food and Drug Administration
|Mayo Clinic||Rochester, Minnesota 55905|
|Mayo Clinic||Scottsdale, Arizona|
|Columbia University Medical Center||New York, New York 10032|
|Bon Secours St. Francis Hospital||Greenville, South Carolina 29601|