Tipifarnib in Treating Patients With Myeloproliferative Disorders

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00005846
Recruitment Status : Unknown
Verified July 2005 by National Cancer Institute (NCI).
Recruitment status was:  Active, not recruiting
First Posted : January 27, 2003
Last Update Posted : June 7, 2011
National Cancer Institute (NCI)
Information provided by:
National Cancer Institute (NCI)

Brief Summary:

RATIONALE: Tipifarnib may stop the growth of cancer cells by blocking the enzymes necessary for cancer cell growth.

PURPOSE: This phase I/II trial is studying the side effects of tipifarnib and to see how well it works in treating patents with myeloproliferative disorders.

Condition or disease Intervention/treatment Phase
Leukemia Myelodysplastic/Myeloproliferative Neoplasms Drug: tipifarnib Phase 1 Phase 2

Detailed Description:


  • Determine the toxic effects of tipifarnib in adult patients with myeloproliferative disorders.
  • Determine hematological responses, including changes in WBC count and erythroid responses, in this patient population treated with this drug.
  • Determine the cytogenetic response in bone marrow of patients treated with this drug.

OUTLINE: This is a multicenter study. Patients are stratified according to prior substantive treatment (yes vs no).

Patients receive oral tipifarnib twice daily on days 1-21. Treatment repeats every 4 weeks for a maximum of 4 courses in the absence of unacceptable toxicity or disease progression. Patients with continued hematologic response after completion of the fourth course may receive additional courses at the discretion of the investigator.

PROJECTED ACCRUAL: A total of 25 patients (12-13 per stratum) will be accrued for this study within 25 months.

Study Type : Interventional  (Clinical Trial)
Primary Purpose: Treatment
Official Title: Phase I/II Study of the Farnesyltransferase Inhibitor R115777 (NSC 702818) in Patients With Myeloproliferative Disorders
Study Start Date : June 2000

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Ages Eligible for Study:   21 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Diagnosis of chronic myelogenous leukemia (CML)

    • Philadelphia chromosome (Ph) positive OR
    • BCR-ABL positive by polymerase chain reaction
    • Must meet 1 of the following 2 conditions:

      • Chronic phase

        • Persistent or progressive disease on maximum tolerated interferon or imatinib mesylate, as evidenced by increasing WBC count, peripheral blood myeloid immaturity, progressive anemia, and/or persistence or relapse of abnormal cytogenetics and/or molecular findings
        • Interferon or imatinib mesylate intolerant
      • Accelerated phase

        • Persistent or progressive disease on imatinib mesylate
      • Patients who have not received interferon or imatinib mesylate due to allergy or refusal are eligible OR
  • Diagnosis of chronic myelomonocytic leukemia

    • Proliferative type (WBC at least 12,000/mm3)
    • Less than 5% blasts in peripheral blood and no more than 20% blasts in bone marrow OR
  • Diagnosis of undifferentiated myeloproliferative disorder OR
  • Diagnosis of atypical CML (Ph negative)
  • No blast crisis phase of CML, atypical CML, or undifferentiated myeloproliferative disorders

    • No more than 20% blasts in peripheral blood or bone marrow
  • Diagnosed more than 3 months before study entry



  • 21 and over

Performance status:

  • ECOG 0-2

Life expectancy:

  • More than 4 months


  • See Disease Characteristics
  • No requirement for platelet transfusion
  • No thrombocytopenia-related bleeding


  • Bilirubin no greater than 1.5 times upper limit of normal (ULN)
  • AST/ALT no greater than 2 times ULN


  • Creatinine no greater than 2.0 mg/dL


  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • Capable of swallowing capsules
  • No other concurrent severe disease that would preclude study compliance
  • No septicemia or other severe infection
  • No iron deficiency

    • If marrow aspirate not available, transferrin saturation at least 20% and ferritin greater than 50 ng/mL
  • No other contributing causes of anemia (e.g., autoimmune or hereditary hemolytic disorders, gastrointestinal blood loss, B12 or folate deficiency, or hypothyroidism)


Biologic therapy:

  • See Disease Characteristics
  • At least 4 weeks since prior interferon
  • At least 4 weeks since prior hematopoietic growth factors
  • No prior allogeneic bone marrow transplantation


  • At least 4 weeks since prior cytotoxic chemotherapy (6 weeks for mitomycin or nitrosoureas) except for hydroxyurea which may be used to manage elevated cell counts through the beginning of the second course of study therapy

Endocrine therapy:

  • No concurrent androgens
  • No concurrent corticosteroids (e.g., greater than 10 mg/day prednisone or equivalent steroid dosage) except as premedication for transfusions


  • Not specified


  • Not specified


  • No other concurrent standard or investigational cytotoxic agents

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00005846

United States, California
Veterans Affairs Medical Center - Palo Alto
Palo Alto, California, United States, 94304
Stanford Cancer Center at Stanford University Medical Center
Stanford, California, United States, 94305
United States, New York
James P. Wilmot Cancer Center at University of Rochester Medical Center
Rochester, New York, United States, 14642
Sponsors and Collaborators
Stanford University
National Cancer Institute (NCI)
Study Chair: Peter L. Greenberg, MD Stanford University

Publications of Results:
Gotlib J, Loh M, Vattikuti S, et al.: Phase I/II study of tipifarnib (ZARNESTRA, farnesyltransferase inhibitor [FTI] R115777) in patients with myeloproliferative disorders(MPDs): preliminary results. [Abstract] Blood 100 (Suppl 1): 798a, 2002. Identifier: NCT00005846     History of Changes
Other Study ID Numbers: CDR0000067864
First Posted: January 27, 2003    Key Record Dates
Last Update Posted: June 7, 2011
Last Verified: July 2005

Keywords provided by National Cancer Institute (NCI):
relapsing chronic myelogenous leukemia
chronic phase chronic myelogenous leukemia
accelerated phase chronic myelogenous leukemia
chronic myelogenous leukemia, BCR-ABL1 positive
Philadelphia chromosome negative chronic myelogenous leukemia
chronic myelomonocytic leukemia
atypical chronic myeloid leukemia, BCR-ABL1 negative
myelodysplastic/myeloproliferative neoplasm, unclassifiable

Additional relevant MeSH terms:
Myeloproliferative Disorders
Myelodysplastic-Myeloproliferative Diseases
Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Antineoplastic Agents