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Phase II Trial of Sorafenib in Patients With Myelodysplastic Syndrome

Phase 2
18 Years
Open (Enrolling)
Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic

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Trial Information

Phase II Trial of Sorafenib in Patients With Myelodysplastic Syndrome

Inclusion Criteria:

- Patients must have a diagnosis of primary or therapy-related myelodysplastic syndrome
or myelodysplastic/ myeloproliferative disorders as defined by the WHO

- Refractory anemia with excess blasts - 1 or 2

- Chronic myelomonocytic leukemia type 2

- Refractory anemia, refractory anemia with ringed sideroblasts, refractory
cytopenia with multilineage dysplasia, refractory cytopenia with multilineage
dysplasia with ringed sideroblasts, 5q- syndrome, myelodysplastic syndrome
unclassified or chronic myelomonocytic leukemia type 1 if at least one of the
following criteria is met: HgB < 10 g/dl, Platelets < 50,000/ul,ANC < 1,000 ul,
Transfusion dependent defined as 2 transfusions within an 8 week period.

- Patients may have low, intermediate-1, intermediate-2 or high risk MDS or CMML.

- Patients are eligible without regard to prior treatment status except for allogenic
bone marrow transplant.

- Patients must be 18 years of age or older.

- Patient has an estimated or measured creatinine clearance ≥30 ml/min at study

- AST, ALT, total bilirubin ≤ than 2.5 times the upper limit of normal.

- ECOG performance status of 0-2.

- Voluntary written informed consent before performance of any study-related procedure
not part of normal medical care.

- Female subject is either post-menopausal or surgically sterilized or willing to use
an acceptable method of birth control.

- Male subject agrees to use an acceptable method for contraception for the duration of
the study therapy and for 2 weeks after study completion.

Exclusion Criteria:

- Female subject is pregnant or lactating. Confirmation that the subject is not
pregnant must be established by a negative serum B-human chorionic gonadotropin
(B-hCG) pregnancy test result within 2 weeks of enrollment. Pregnancy testing is not
required for post-menopausal or surgically sterilized women.

- Patient has received other investigational drugs for this disease within 14 days of

- No growth factor support with erythropoietin, GCSF, or GMCSF within 28 days of
enrolling in the study.

- Serious medical or psychiatric illness likely to interfere with participation in this
clinical study.

- Patients with another malignancy within the last one year (from documentation of
remission) other than basal or squamous cell skin cancer or CIS of the cervix.

- Patients who underwent allogeneic stem cell transplant will be excluded.

- History of leukemia (having more than 20% blasts in blood or marrow)

- Current treatment with coumadin, heparin and its derivatives.

- Major surgery (including needle biopsy of visceral organs) for 1-month prior to study
and fully recovered. In addition, no placement of a subcutaneous or tunneled venous
access device for 3 days prior to study and adequately healed.

- Significant cardiac or vascular events within 6 months: acute MI, unstable angina,
severe peripheral vascular disease (ischemic pain at rest class 3 or worse,
non-healing ulcers/wounds, congestive heart failure (NHYA class ≥ 2), uncontrolled
cardiac arrhythmias, and disseminated intravascular coagulation.

- No use of hematopoetic growth factors within 4 weeks of starting sorafenib.

- Known severe hypersensitivity to Sorafenib or any component of the formulation.

- Caution should be exercised with the concomitant use of other CYP3A4 inducers, such
as rifampin, St. John's Wort, phenytoin, phenobarbital and dexamethasone.

- Uncontrolled hypertension with a systolic blood pressure greater than 160 or a
diastolic blood pressure greater than 100 despite treatment.

Type of Study:


Study Design:

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Hematological response

Outcome Time Frame:

5 years

Safety Issue:


Principal Investigator

David A Rizzieri, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Duke University


United States: Institutional Review Board

Study ID:




Start Date:

July 2006

Completion Date:

December 2012

Related Keywords:

  • Myelodysplastic Syndromes
  • Leukemia, Myelomonocytic, Chronic
  • Sorafenib
  • Myelodysplastic Syndromes
  • MDS
  • Leukemia
  • Leukemia, Myelomonocytic, Chronic
  • Myelodysplastic Syndromes
  • Preleukemia



Duke University Medical Center Durham, North Carolina  27710