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Tipifarnib in Treating Patients With Acute Myeloid Leukemia in Remission

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ClinicalTrials.gov Identifier: NCT00093470
Recruitment Status : Active, not recruiting
First Posted : October 8, 2004
Results First Posted : November 10, 2015
Last Update Posted : November 16, 2016
Information provided by (Responsible Party):
National Cancer Institute (NCI)

Brief Summary:
This randomized phase III trial studies tipifarnib in treating patients with acute myeloid leukemia (AML) in remission. Tipifarnib may stop the growth of cancer cells by blocking the enzymes necessary for their growth. It is not yet known whether tipifarnib is more effective than observation alone in preventing the recurrence of AML.

Condition or disease Intervention/treatment Phase
Adult Acute Myeloid Leukemia Adult Acute Myeloid Leukemia in Remission Procedure: Clinical Observation Drug: Tipifarnib Phase 3

Detailed Description:


I. To compare R115777 (tipifarnib) maintenance therapy to observation only with respect to disease-free survival (DFS) in patients with acute myeloid leukemia (AML) in second or subsequent complete remission or in complete response (CR) following primary induction failure.


I. To compare overall survival of patients in both arms. II. To evaluate the long-term safety and toxicity of extended administration of R115777 in AML patients in remission.

OUTLINE: Patients are randomized to 1 of 2 treatment arms.

ARM A: Patients receive tipifarnib orally (PO) twice daily (BID) on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.

ARM B: Patients undergo observation only.

After completion of study treatment, patients are followed up for 5 years.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 144 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase III Randomized Study of Farnesyl Transferase Inhibitor R115777 in Acute Myeloid Leukemia (AML) Patients in Second or Subsequent Remission or in Remission After Primary Induction Failure or Patients Over Age 60 in First Remission
Study Start Date : August 2004
Primary Completion Date : February 2015

Arm Intervention/treatment
Experimental: Arm A (tipifarnib)
Patients receive tipifarnib PO BID on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Drug: Tipifarnib
Given PO
Other Names:
  • R115777
  • Zarnestra
Arm B (clinical observation)
Patients undergo observation only.
Procedure: Clinical Observation
Undergo observation

Primary Outcome Measures :
  1. Disease-free Survival [ Time Frame: Assessed monthly for the first 6 months then every 3 months or as clinically indicated, up to 5 years. ]
    Disease-free survival (DFS) is defined as the time from randomization to relapse or death without relapse.

Secondary Outcome Measures :
  1. Overall Survival [ Time Frame: Assessed monthly for the first 6 months then every 3 months or as clinically indicated, up to 5 years. ]
    Overall survival (OS) is defined as the time from randomization to death from any cause.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Patients eligible to enter this study must fall into one of these categories:

    • Patients in first remission following primary induction failure and have received at least two chemotherapy induction regimens
    • Patients in second or subsequent remission
    • Patients > 60 years old in first remission
  • Patients must be in complete remission (CR) or morphologic remission (MR) by blood counts and bone marrow studies to enter the study

    • Confirmatory bone marrow must be performed =< 2 weeks prior to randomization
  • Patients must have morphologic proof (from bone marrow aspirate, smears or touch preps of marrow biopsy) that they had AML of one of the following types prior to achievement of CR/MR

    • Acute myeloblastic leukemia, minimal differentiation (French-American-British [FAB] M0)
    • Acute myeloblastic leukemia without differentiation (FAB M1)
    • Acute myeloblastic leukemia with maturation (FAB M2)
    • Acute myelomonocytic leukemia (FAB M4)
    • Acute monocytic leukemia (FAB M5)
    • Acute erythroleukemia (FAB M6)
    • Acute megakaryocytic leukemia (FAB M7)
    • Refractory anemia with excess blasts in transformation (RAEB-T)
    • AML by World Health Organization (WHO) criteria
    • Acute myeloid leukemia with multilineage dysplasia
  • Patients must be registered within 60 days of completion of therapy for the current remission
  • All of the patients below are eligible for study entry:

    • Patients who have received consolidation therapy
    • Patients who have not received any consolidation or post remission therapy
    • Patients who have had an autologous stem cell transplant
  • Patients with a history of extramedullary disease are eligible if they are in complete remission at the time of study entry and no longer requiring therapy for their extramedullary disease
  • Women of childbearing potential and sexually active males are strongly advised to use an accepted and effective method of contraception
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0-2
  • Absolute neutrophil count (ANC) >= 1000/mm^3
  • Platelet count >= 50,000/mm^3

Exclusion Criteria:

  • Patients with acute promyelocytic leukemia (FAB M3)
  • Patients who have received an allogeneic transplant (bone marrow transplant [BMT] or peripheral stem cell transplant [PSCT]) in their current remission are ineligible; patients who have had an allogeneic transplant in a previous remission and are currently in remission after subsequent relapse are eligible
  • Pregnant or breast-feeding
  • Allergy to imidazole drugs, such as clotrimazole ketoconazole, miconazole, econazole, or terconazole; this does not include fluconazole, voriconazole, or itraconazole
  • Active cardiac or pulmonary disease; but patient will be eligible if disease is medically controlled
  • Active renal disease
  • Active hepatic disease
  • Patients who are taking a hepatic enzyme-inducing anti-convulsant

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 ClinicalTrials.gov identifier (NCT number): NCT00093470

  Show 221 Study Locations
Sponsors and Collaborators
National Cancer Institute (NCI)
Study Chair: Selina Luger, M.D. University of Pennsylvania

Responsible Party: National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT00093470     History of Changes
Other Study ID Numbers: NCI-2009-00535
NCI-2009-00535 ( Other Identifier: CTRP (Clinical Trial Reporting Program) )
E2902 ( Other Identifier: ECOG-ACRIN Cancer Research Group )
U10CA180820 ( U.S. NIH Grant/Contract )
U10CA021115 ( U.S. NIH Grant/Contract )
First Posted: October 8, 2004    Key Record Dates
Results First Posted: November 10, 2015
Last Update Posted: November 16, 2016
Last Verified: November 2016

Keywords provided by National Cancer Institute (NCI):
Acute Myeloid Leukemia
Farnesyl Transferase Inhibitor

Additional relevant MeSH terms:
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Neoplasms by Histologic Type
Antineoplastic Agents