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Efficacy and Safety of ON 01910.Na in Myelodysplastic Syndrome (MDS) Patients With Trisomy 8 or Classified as Intermediate-1, -2 or High Risk
Trial ID: NCT01326377
This study will explore the efficacy and safety of a regimen of ON 01910.Na as a 48-hour continuous intravenous infusion once a week for 3 weeks of a 4-week cycle in MDS patients with Trisomy 8 or classified as Intermediate-1, -2 or High Risk who are not responding to current therapeutic options. The rationale for this trial is based upon data from laboratory studies with ON 01910.Na and upon activity that has been observed in other clinical trials with ON 01910.Na in patients with MDS.
A Phase 2, Single-Arm Study To Assess The Efficacy and Safety Of 72-Hour Continuous Intravenous Dosing Of ON 01910.Na Administered Every Other Week in Myelodysplastic Syndrome Patients With Trisomy 8 or Classified as Intermediate-1, 2 or High Risk
- Diagnosis of MDS confirmed within 2 weeks prior to study entry according to the World
Health Organization (WHO) Criteria or the French-American-British (FAB)
- Trisomy 8 cytogenetics (simple or combined to other karyotypes) or patient classified
as Intermediate-1 with bone marrow blasts equal to or greater than 5%, Intermediate-2
or High Risk MDS according to the IPSS score, or Patients with peripheral blood blasts
equal to or greater than 5%.
- At least one cytopenia (Absolute Neutrophil Count < 1800/µl or Platelet Count
<100,000/µl or Hemoglobin < 10 g/dL).
- Failure of, or insufficient response to Azacytidine or Decitabine administered for 4
to 6 cycles in patients classified as Intermediate-2 or High risk or to Erythrocyte
stimulating agents (failure or insufficient response defined as transfusion dependence
or Hemoglobin remaining below 10 g/dl) in Low or Intermediate-1 Risk Trisomy 8
- Failed to respond to, relapsed following, or opted not to participate in bone marrow
- Off all other treatments for MDS (including filgrastim (G-CSF) and erythropoietin) for
at least four weeks. As an exception, filgrastim (G-CSF) can be used before, during
and after the protocol treatment for patients with documented febrile neutropenia (<
- ECOG Performance Status 0, 1 or 2.
- Willing to adhere to the prohibitions and restrictions specified in this protocol.
- Patient (or his/her legally authorized representative) must have signed an informed
consent document indicating that he/she understands the purpose of and procedures
required for the study and is willing to participate in the study.
- Anemia due to factors other than MDS (including hemolysis or gastrointestinal
- 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.
- History of HIV-1 seropositivity.
- 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 > 1.5 mg/dL not related to hemolysis or Gilbert's disease, ALT or AST
> 2 X ULN.
- Serum creatinine > 2.0 mg/dL or calculated creatinine clearance < 60 ml/min/1.73 m^2.
- Ascites requiring active medical management including paracentesis, or hyponatremia
(defined as serum sodium value of <134 Meq/L).
- Women patients who are pregnant or lactating; Male patients with female sexual
partners who are unwilling to follow the strict contraception requirements described
in this protocol; Patients who do not agree to use adequate contraceptive [including
prescription oral contraceptives (birth control pills), contraceptive injections,
intrauterine device (IUD), double-barrier method (spermicidal jelly or foam with
condoms or diaphragm), contraceptive patch, or surgical sterilization] before entry
and throughout the study; Female patients with reproductive potential who do not have
a negative serum beta-HCG pregnancy test at screening.
- Major surgery without full recovery or major surgery within 3 weeks of ON 01910.Na
- Uncontrolled hypertension (defined as a systolic pressure equal to or greater than 160
mmHg and/or a diastolic pressure equal to or greater than 110 mmHg).
- New onset seizures (within 3 months prior to the first dose of ON 01910.Na) or poorly
- Any concurrent investigational agent or chemotherapy, radiotherapy or immunotherapy.
- Treatment with standard MDS therapies or investigational therapy within 4 weeks of
starting ON 01910.Na.
- Psychiatric illness/social situations that would limit the patient's ability to
tolerate and/or comply with study requirements.
drug: ON 01910.Na
drug: ON 01910.Na
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