Efficacy and Safety of Oral Rigosertib in Transfusion-dependent, Low or Int-1 or Trisomy 8 Int-2 Myelodysplastic Syndrome (ONTARGET)
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|ClinicalTrials.gov Identifier: NCT01584531|
Recruitment Status : Completed
First Posted : April 25, 2012
Last Update Posted : June 26, 2017
|Condition or disease||Intervention/treatment||Phase|
|Myelodysplastic Syndrome MDS Trisomy 8||Drug: rigosertib||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||82 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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 8 Intermediate-2 Myelodysplastic Syndrome Patients Based on IPSS Classification|
|Study Start Date :||May 2012|
|Actual Primary Completion Date :||May 2015|
|Actual Study Completion Date :||November 2015|
Experimental: 21-Day Regimen
560 mg oral rigosertib in the morning and 280 mg rigosertib in the afternoon on days 1 to 21 of 21-day cycle
Rigosertib sodium will be available as soft gel capsules in strengths of 280 mg and 70 mg. Rigosertib will be administered on an outpatient basis.
Patients will take a 560 mg dose (e.g., 2 x 280 mg capsules) of oral rigosertib in the morning and 280 mg dose (e.g., 1 x 280 mg capsules) of oral rigosertib every day of 21-day cycles. Rigosertib should be taken in a fasting state (defined by at least 30 minutes before next meal) BID at 12 hr intervals (with a window of 2 hr). Any vomited dose will be reported as a missed dose.
The patient will fill a diary indicating the day and time of drug intake.
- 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.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01584531
|United States, Arizona|
|Scottsdale, Arizona, United States, 85259|
|United States, Georgia|
|Winship Cancer Institute, Emory University|
|Atlanta, Georgia, United States, 30322|
|United States, Minnesota|
|Rochester, Minnesota, United States, 55905|
|United States, New York|
|Columbia University Medical Center|
|New York, New York, United States, 10032|
|United States, South Carolina|
|Bon Secours St. Francis Hospital|
|Greenville, South Carolina, United States, 29601|
|Study Director:||Steven M. Fruchtman, MD||Onconova Therapeutics, Inc.|