A Single-arm Study to Assess the Efficacy and Safety of Oral Rigosertib in Transfusion-dependent, Low or Intermediate-1, Myelodysplastic Syndrome Patients Based on the International Prognostic Scoring System

Onconova Therapeutics, Inc.0 sites40 target enrollmentApril 22, 2013

Overview

No summary available.

Registry

who.int

Start Date

April 22, 2013

End Date

TBD

Last Updated

10 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Sponsor

•Male and female patients who meet all of the following criteria are eligible for enrollment in the trial:
•a. \=18 years of age;
•b. Diagnosis of MDS according to World Health Organization (WHO) criteria (Appendix 2\) or French\-American\-British (FAB) classification, that must be confirmed by BM aspirate and/or biopsy within 6 weeks prior to Screening;
•c. MDS classified as Low risk or Int\-1 risk, according to IPSS classification; in addition, patients should never have been classified as Int\-2 or High\-risk since their MDS was diagnosed;
•d. Transfusion dependency defined by transfusion of at least 4 units of RBC within 56 days (ie, 8 weeks) before Screening (pre\-transfusion Hgb values values must be \= 9 g/dL to be taken into account);
•e. Refractory to 8\- to 12\-week course of ESA administered within the past 2 years before enrollment, or EPO level \> 500 mU/mL and off ESA for at least 8 weeks before Screening;
•f. Off all other treatments for MDS (AZA, decitabine, lenalinomide, ESA, chemotherapy, immunotherapy) for at least 2 weeks prior to Screening;
•g. ECOG performance status of 0, 1 or 2;
•h. Willing to adhere to the prohibitions and restrictions specified in this protocol;
•i. The patient must sign an informed consent form (ICF) indicating that s/he understands the purpose of, and procedures required for, the study and is willing to participate.

•Patients with any of the following will not be enrolled in the study:
•a. Ongoing clinically significant anemia due to factors such as iron, vitamin B12, or folate deficiencies, auto\-immune or hereditary hemolysis, or gastrointestinal (GI) bleeding;
•b. Serum ferritin \< 50 ng/mL;
•c. Hypoplastic MDS (cellularity \<10%);
•d. Any active malignancy within the past year, except basal cell or squamous cell skin cancer or carcinoma in situ of the cervix or breast;
•e. Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia;
•f. Active infection not adequately responding to appropriate therapy;
•g. Total bilirubin \= 2\.0 mg/dL not related to hemolysis or Gilbert’s disease;
•h. Alanine transaminase (ALT) or aspartate transaminase (AST) \= 2\.5 x the upper limit of normal (ULN);
•i. Serum creatinine \= 2\.0 mg/dL;