Clinical Trial: Efficacy and Safety of Oral Rigosertib in Transfusion-dependent, Low or Int-1 or Trisomy 8 Int-2 Myelodysplastic Syndrome

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: 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

Brief Summary: The primary objectives of this study are to determine if rigosertib sodium, given orally in the form of soft gel capsules, is safe and is associated with a reduction in the number of blood transfusion units that are needed in patients with myelodysplastic syndrome (MDS) classified as Low or Intermediate-1 (Int-1) (any cytogenetics) or trisomy 8 Intermediate 2 (Int-2) in the International Prognostic Scoring System (IPSS) who are transfusion-dependent. Rigosertib will be taken on days 1 to 21 of a 21-day cycle.

Detailed Summary:

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 rigosertib BID for 21 consecutive days of a 21-day cycle.

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. Rigosertib dosing adjustment policies are described in Protocol.


Sponsor: Onconova Therapeutics, Inc.

Current Primary Outcome: Number of units of red blood cell transfusions [ Time Frame: 8 weeks ]

Number of units of red blood cell transfusions will be compared with the pretreatment transfusion number in the previous 8 weeks.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Number of Adverse Events (AEs) [ Time Frame: From date of randomization until 30 days after last dose of study drug ]
    AEs reported by the patient or observed by the Investigator or study site personnel Safety assessments will be counted and documented on Case Report Forms and source documents. All AEs from signature of the ICF through 30 days after a patient discontinues from the study will be included.
  • Bone marrow blasts [ Time Frame: 4 weeks ]
    Change in number of bone marrow blasts will be compared to pretreatment.
  • Complete blood count [ Time Frame: 4 weeks ]
    Complete blood count with differential.


Original Secondary Outcome: Same as current

Information By: Onconova Therapeutics, Inc.

Dates:
Date Received: April 23, 2012
Date Started: May 2012
Date Completion:
Last Updated: August 1, 2016
Last Verified: August 2016