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A Phase 2, Single-Arm Study To Assess The Efficacy and Safety Of 72-Hour Continuous Intravenous Dosing Of ON 01910.Na Administered Once Every 2 Weeks in Myelodysplastic Syndrome Patients With Trisomy 8 or Classified as Intermediate-1, 2 or High Risk

Phase 2
18 Years
Not Enrolling
Myelodysplastic Syndromes, Myelodysplastic Syndromes (MDS), Leukemia, Acute Myeloid Leukemia (AML)

Thank you

Trial Information

A Phase 2, Single-Arm Study To Assess The Efficacy and Safety Of 72-Hour Continuous Intravenous Dosing Of ON 01910.Na Administered Once Every 2 Weeks in Myelodysplastic Syndrome Patients With Trisomy 8 or Classified as Intermediate-1, 2 or High Risk

Primary Objective The primary objective of this study is to evaluate the efficacy and safety
of ON 01910.Na CIV 1800 mg/24h for 72-hour infusion administered every other week for 8
weeks and every 4 weeks afterwards in achieving by week 29 a complete or partial response or
hematological improvement as defined per the 2006 International Working Group (IWG) Criteria
in Myelodysplastic Syndrome (MDS) patients with Trisomy 8 or classified as Intermediate-1,
2, or High risk.

Secondary Objectives

The secondary objectives are to assess:

- Time and duration of bone marrow response

- Complete or partial response according to International Working Group (IWG) 2006

- Improvement of dyspoiesis as evaluated by the change in aneuploidy in bone marrow

- Change in International Prognostic Scoring System (IPSS)

- Responses in absolute neutrophil count, according to IWG 2006 criteria

- Responses in platelet count, according to IWG 2006 criteria

- Erythroid responses, according to IWG 2006 criteria

- Time to progression

- Overall survival at 29 and 53 weeks

- Proportion of patients transitioning to acute myeloid leukemia (AML) at 29 and 53 weeks

- Exploratory biomarkers on bone marrow aspirates (described in a separate companion

Inclusion Criteria:

- > 18 years

- Diagnosis of MDS via bone marrow aspirate and biopsy according to WHO Criteria and
FAB Classification

- Anemia requiring transfusion support with at least one unit of packed red blood cells
per month for greater than or equal to 2 months or Hemoglobin < 10 g/dL OR
Thrombocytopenia (platelet count < 100,000/ul) OR Neutropenia (absolute neutrophil
count < 1,500/ul)

- 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 (<500/ul)

- ECOG Performance Status 0, 1 or 2

- 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 for female patients of
reproductive potential

- Female patient with reproductive potential must have a negative serum beta-HCG
pregnancy test at screening

- 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

Exclusion Criteria:- Anemia due to factors other than MDS (including hemolysis or
gastrointestinal bleeding)

- Proliferative (WBC >= 12,000/mm^3) chronic myelomonocytic leukemia

- 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 > 1.5 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

- Major surgery without full recovery or major surgery within 3 weeks of ON 01910.Na
treatment start.

- Uncontrolled hypertension (defined as a systolic pressure >= 160 and/or a diastolic
pressure >= 110)

- New onset seizures (within 3 months prior to the first dose of ON 01910.Na) or poorly
controlled seizures

- Any concurrent investigational agent or chemotherapy, radiotherapy or immunotherapy

- Psychiatric illness/social situations that would limit the patient's ability to
tolerate and/or comply with study requirements

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Response as defined per the 2000 International Working Group (IWG) Criteria in patients with MDS.

Outcome Time Frame:

6-12 months

Safety Issue:


Principal Investigator

Peter L Greenberg

Investigator Role:

Principal Investigator

Investigator Affiliation:

Stanford University


United States: Food and Drug Administration

Study ID:




Start Date:

May 2009

Completion Date:

February 2011

Related Keywords:

  • Myelodysplastic Syndromes
  • Myelodysplastic Syndromes (MDS)
  • Leukemia
  • Acute Myeloid Leukemia (AML)
  • MDS
  • AML
  • Refractory Cytopenia
  • Refractory Anemia Dysplasia
  • Leukemia
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid
  • Myelodysplastic Syndromes
  • Preleukemia
  • Trisomy



Stanford University School of Medicine Stanford, California  94305-5317