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Protocol No. UW16113

Principal Investigator Mattison, Ryan

Phase III

Age Group Adult

Scope National

Sponsor Type Industry

Title A Phase III, International, Randomized, Controlled Study of Rigosertib versus Physician's Choice of Treatment in Patients with Myelodysplastic Syndrome after Failure of a Hypomethylating Agent

Objective We want to find out if a drug called Rigosertib is a better treatment for Myelodyplastic syndrome (MDS) than treatment with Azacitidine and Decitabine. At this time Rigosertib has not been approved by the US Food and Drug Administration (FDA) for the treatment of MDS and is therefore considered investigational for this research study. Both Azacitidine and Decitabine have been approved by the FDA in treating MDS.

Treatment Rigosertib is an investigational drug. This means that rigosertib is not approved by the U.S. Food and Drug Administration (FDA) or any other regulatory authority, and it is not commercially available. It is currently being used for research purposes only. Rigosertib is referred to as "study drug" in this consent form. Rigosertib is being studied as a new potential anticancer drug and as potential treatment for myelodysplasia. Although the mechanism of action of rigosertib is not fully known, it is thought to kill cancer cells by two different actions: it stops the tumor cells from dividing and growing, and it blocks the proteins involved in cell division, causing cells to die.

Description You will be randomly assigned (as in the flip of a coin) to 1 of 2 groups, one group will receive rigosertib. The other group will receive Physician's Choice,which means your health care provider will choose the therapy that is deemed most appropriate for you after you have failed a hypomethylating agent. If you are in Group 1, you will receive rigosertib as a continuous 72-hour intravenous infusion every other week for 16 weeks, and every 4 weeks thereafter. If you are in Group 2, you will receive Physician's Choice, and you will not receive rigosertib. You will have a higher chance of receiving rigosertib than the Physician's Choice. Two (2) out of every 3 participants will be placed in Group 1 rigosertib. Neither you nor the study doctor will be able to pick which study group you are in. This will be randomly assigned.

Key Eligibility
  • MDS classification and cytogenetics
  • At least one cytopenia (ANC < 1800/μL or platelet count < 100,000/μL or hemoglobin [Hgb] <10 g/dL)
  • Progression at any time after initiation of AZA or DAC treatment or failure to achieve complete or partial response or HI after at least six 4-week cycles of AZA or either four 4-week or four 6-week cycles of DAC or relapse after initial complete or partial response or HI or intolerance to AZA or DAC
  • Total duration of prior HMA therapy ≤ 9 months and/or total ≤ 9 cycles of prior HMA therapy in ≤ 12 months
  • Has failed to respond to, relapsed following, not eligible for, or opted not to participate in allogenic stem cell transplantation
  • Off all other treatments for MDS (including AZA and DAC) for ≥ 4 weeks before randomization
  • Patients with 5q- syndrome should have failed to respond to or progressed on treatment with lenalidomide, where available and indicated
  • Patients are ineligible who have previously participated in a clinical study of IV or oral rigosertib; however, patients who failed screening for other rigosertib studies may be screened for participation in this study
  • Patients are excluded if they are eligible to receive induction chemotherapy, such as 7-10 days of cytosine arabinoside and 2-3 days of an anthracycline, or high-dose cytarabine (HDAC)
  • Patients are ineligible if previously diagnosed with AML
  • Patient is ineligible if they are a suitable candidate to receive allogeneic stem cell transplantation; patient is eligible for study if a suitable candidate refuses to undergo an allogeneic stem cell transplant or a suitable donor cannot be found
  • Patient is ineligible if they have known active HIV, hepatitis B or hepatitis C
  • Female patients of child-bearing potential (pre-menopausal and not surgically sterilized) who are breast-feeding or have a positive pregnancy test at Screening are ineligible
  • Patients with controlled hypertension are ineligible
  • Patients using any other concurrent investigational agent or chemotherapy, radiotherapy, or immunotherapy, or corticosteroids are ineligible
  • Patients treated with cytarabine at any dose, lenalidomide, or any other therapy targeted for the treatment of MDS are ineligible

Applicable Disease Sites Hematologic cancers, other

Therapies Involved Cytotoxic Chemotherapy

Drugs Involved 5-AC (azacitidine); 5-AZA (azacitidine); 5-azacitidine (azacitidine); Dacogen (decitabine); ON 01910.Na Concentrate (Rigosertib); Rigosertib; azacitidine; decitabine

Status Open

Participating Institutions UW Hospital and Clinics