Efficacy and Safety of IV Rigosertib in MDS Patients With Excess Blasts Progressing After Azacitidine or Decitabine
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: III 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: 13-003625
NCT ID: NCT01928537
Sponsor Protocol Number: 04-24
About this study
This study will examine the effect intravenously administered rigosertib has on the relationship between bone marrow blasts response and overall survival in myelodysplastic syndromes (MDS) patients who have 5-30% bone marrow blasts and who progressed on or after treatment with azacitidine or decitabine.
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 confirmed within 6 weeks prior to Screening according to WHO criteria or French-American-British (FAB) classification.
- MDS classified as follows, according to WHO criteria and FAB classification:
- RAEB-1 (5% to 9% BM blasts)
- RAEB-2 (10% to 19% BM blasts)
- CMML (10% to 20% BM blasts) and white blood cells (WBC) < 13,000/μL
- RAEB-t (20% to 30% BM blasts), meeting the following criteria: WBC < 25,000/μL at study entry; or, Stable White Blood Cell (WBC) at least 4 weeks prior to Screening and not requiring intervention for WBC control with hydroxyurea, chemotherapy, or leukopheresis.
- At least one cytopenia (Absolute Neutrophil Count (ANC) < 1800/μL or Platelet (PLT) count < 100,000/μL or hemoglobin (Hgb) < 10 g/dL).
- Progression (according to 2006 IWG criteria) at any time after initiation of subcutaneous or intravenous azacitidine or decitabine treatment per labeling during the past 2 years, defined as follows:
- For patients with ˂ 5% BMBL, ≥ 50% increase in BMBL to ˃ 5% BMBL
- For patients with 5-10% BMBL, ≥ 50% increase in BMBL to ˃ 10% BMBL
- For patients with 10-20% BMBL, ≥ 50% increase in BMBL to ˃ 20% BMBL
- For patients with 20-30% BMBL, ≥ 50% increase in BMBL to ˃ 30% BMBL
- Any of the following: ≥ 50% decrease from maximum remission/response levels in granulocytes or PLT; Decrease in Hgb concentration by ≥ 2 g/dL; or, Transfusion dependence, defined as administration of at least 4 RBC units in the past 8 weeks before Screening (patients must have Hgb values ˂ 9 g/dL prior to transfusion to be considered), in the absence of another explanation.
- Has failed to respond to, relapsed following, not eligible, or opted not to participate in bone marrow transplantation.
- Off all other treatments for MDS for at least 4 weeks, except for azacitidine or decitabine. Filgrastim (G-CSF) and erythropoietin are allowed before and during the study as clinically indicated.
- No medical need for induction chemotherapy.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2.
- Willing to adhere to the prohibitions and restrictions specified in this protocol.
- Patient must signed an informed consent form.
- Previous participation in a clinical study of IV or oral rigosertib.
- Anemia due to factors other than MDS (including hemolysis or gastrointestinal [GI] bleeding) unless stabilized for 1 week after RBC transfusion.
- 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.
- Active infection not adequately responding to appropriate therapy.
- Total bilirubin ≥ 1.5 mg/dL not related to hemolysis or Gilbert's disease.
- ALT/AST ≥ 2.5 x upper limit of normal (ULN).
- Serum creatinine ≥ 2.0 mg/dL.
- Ascites requiring active medical management including paracentesis, or 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 beta-human chorionic gonadotropin (βHCG) pregnancy test at Screening.
- Major surgery without full recovery or major surgery within 3 weeks of Baseline/Cycle 1 Day 1 visit.
- Uncontrolled hypertension (defined as a systolic pressure ≥160 mmHg and/or a diastolic pressure ≥ 110 mmHg).
- New onset seizures (within 3 months prior to Baseline) or poorly controlled seizures.
- Any other concurrent investigational agent or chemotherapy, radiotherapy, or immunotherapy. Prior treatment with low-dose cytarabine during the past 2 years.
- Investigational therapy within 4 weeks of Baseline/Day 1 visit.
- 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