Myelodysplastic Syndrome MedDRA version: 14.1 Level: PT Classification code 10028533 Term: Myelodysplastic syndrome System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Conditions
Interventions
Product Name: rigosertib
Product Code: ON 01910.Na
Pharmaceutical Form: Capsule, soft
INN or Proposed INN: rigosertib
CAS Number: 592542-60-4
Current Sponsor code: ON 01910.NA
Concentration unit: mg m
Sponsors
Onconova Therapeutics, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 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. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 20 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 20
Exclusion criteria
Exclusion criteria: 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 2 weeks) of corticosteroids (> 10 mg/24 hr equivalent prednisone) within 4 weeks of Screening; t. Investigational therapy within 4 weeks of Screening; u. Psychiatric illness or social situation that would limit the patient’s ability to tolerate and/or comply with study requirements.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary efficacy objective is to determine the onset of hematological improvement according to the 2006 International Working Group (IWG) criteria.;Secondary Objective: The secondary efficacy objectives are to evaluate rigosertib with respect to: Efficacy: - Overall response (complete and partial responses) according to 2006 IWG criteria including the onset and duration of response; - Bone marrow response according to 2006 IWG criteria. Safety will also be evaluated.;Primary end point(s): Haematological improvement will be assessed according to the 2006 IWG criteria to include: 1. RBC transfusion independence, ie no RBC transfusions administered over a period of 8 consecutive weeks. 2. erythroid response, ie transfusion reduction of at least 4 RBC units over 8 weeks after Baseline/First Dose compared to the number of transfusions in the 8 weeks before treatment 3. neutrophil or platelet response ;Timepoint(s) of evaluation of this end point: 1. RBC transfusion independence and 2. erythroid response: transfusion history to be recorded at screening, baseline (Day 1) and weekly throughout study for a maximum period of 48 weeks. 3. neutrophil or platelet response: complete blood count will be measured at screening, baseline (Day 1) and every week throughout study. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Efficacy response will be assessed according to the following outcomes: 1. Number and dates of transfusions of red blood cell (RBC) units 2. Hgb values before each RBC transfusion 3. Number and dates of transfusions of platelet (PLT) units 4. Platelet counts before each PLT transfusion 5. Overall survival 6. Blastic response in bone marrow (BM) 7. Change in BM cytogenetics 8. Changes in Hgb and in RBC transfusion requirements 9. Changes in absolute neutrophil count (ANC) 10. Changes in PLT count and PLT transfusion requirements Safety will be assessed as treatment emergent adverse events;Timepoint(s) of evaluation of this end point: 1, 3: Transfusion history to be recorded at screening, baseline (Day 1) and weekly throughout study; 2, 4, 8, 9, 10: Complete blood count will be measured at screening, baseline (Day 1) and every week throughout study; 5: throughout study 6: Bone marrow aspirate and/or biopsy will be performed at screening and for patients with more than 5% pre-treatment BMBL every 15 weeks throughout study; 7: Bone marrow cytogenetics will be performed at screening and if abnormal will repeated every 15 weeks throughout study Safety will be monitored throughout study | — |
Countries
France, Germany, United States
Contacts
Public ContactProject Manager 09-07
Ockham Oncology
Outcome results
None listed