Oral Rigosertib in Low Risk MDS Patients Refractory to ESAs
Study type: Interventional What is this?
Describes the nature of a clinical study. Types include:
- Observational study — observes people and measures outcomes without affecting results.
- Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
- Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
Study phase: II What is this?
During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.
- Rochester, Minnesota: 13-009041
NCT ID: NCT01904682
Sponsor Protocol Number: 09-07
About this study
The study will enroll low risk MDS patients who need red blood cell transfusions and who are refractory to or are not using erythropoiesis-stimulating agents. The purpose of the study is to determine whether oral rigosertib treatment results in hematological improvements according to the 2006 International Working Group criteria in these patients. The study will also record any side effects that may occur during the study.
Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.
See eligibility criteria
- Diagnosis of MDS according to World Health Organization (WHO) criteria (Appendix 2) or French-American-British (FAB) classification that must be confirmed by bone marrow (BM) aspirate and/or biopsy within 6 weeks prior to Screening.
- Myelodysplastic syndrome (MDS) classified as Low risk or Int-1 risk, according to International Prognostic Scoring System (IPSS) classification; in addition, patients should never have been classified as Int-2 or High-risk since their MDS was diagnosed;
- Transfusion dependency defined by transfusion of at least 4 units of Red blood cells (RBC) within 56 days before Screening (pre-transfusion Hgb values values must be ≤ 9 g/dL to be taken into account).
- Refractory to 8- to 12-week course of Erythropoiesis-stimulating agent (ESA) administered within the past 2 years before enrollment, or erythropoietin (EPO) level ˃ 500 mU/mL and off ESA for at least 8 weeks before Screening.
- Off all other treatments for MDS (azacitidine, decitabine, lenalidomide, ESA, chemotherapy, immunotherapy) for at least 2 weeks prior to Screening.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2.
- Willing to adhere to the prohibitions and restrictions specified in this protocol.
- The patient must signed an informed consent form (ICF) indicating that s/he understands the purpose of, and procedures required for, the study and is willing to participate.
- Ongoing clinically significant anemia due to factors such as iron, vitamin B12, or folate deficiencies, auto-immune or hereditary hemolysis, or gastrointestinal (GI) bleeding.
- Serum ferritin < 50 ng/mL.
- Hypoplastic MDS (cellularity <10%)
- Any active malignancy within the past year, except basal cell or squamous cell skin cancer or carcinoma in situ of the cervix or breast.
- Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia.
- Active infection not adequately responding to appropriate therapy.
- Total bilirubin ≥ 2.0 mg/dL not related to hemolysis or Gilbert's disease.
- Alanine transaminase (ALT) or aspartate transaminase (AST) ≥ 2.5 x the upper limit of normal (ULN).
- Serum creatinine ≥ 2.0 mg/dL.
- Ascites requiring active medical management including paracentesis.
- Hyponatremia (defined as serum sodium value of < 130 mEq/L).
- Female patients who are pregnant or lactating.
- Patients who are unwilling to follow strict contraception requirements.
- Female patients with reproductive potential who do not have a negative urine pregnancy test at Screening.
- Major surgery without full recovery or major surgery within 3 weeks of Screening.
- Uncontrolled hypertension (defined as a systolic pressure ≥ 160 mmHg and/or a diastolic pressure ≥ 110 mmHg).
- New onset seizures (within 3 months prior to the first dose of rigosertib) or poorly controlled seizures.
- Any other concurrent investigational agent or chemotherapy, radiotherapy, or immunotherapy.
- Chronic use (˃ 2 weeks) of corticosteroids (˃ 10 mg/24 hr equivalent prednisone) within 4 weeks of Screening.
- Investigational therapy within 4 weeks of Screening.
- Psychiatric illness or social situation that would limit the patient's ability to tolerate and/or comply with study requirements.
Participating Mayo Clinic locations
Study statuses change often. Please contact us for help.
|Mayo Clinic Location
Mayo Clinic principal investigator
Aref Al-Kali, M.D.
Closed for enrollment
Cancer Center Clinical Trials Referral Office