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Tipifarnib in Treating Older Patients With Previously Untreated Acute Myeloid Leukemia

Phase 2
Completed
Conditions
Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome
Adult Acute Basophilic Leukemia
Adult Acute Eosinophilic Leukemia
Adult Acute Erythroid Leukemia (M6)
Adult Acute Megakaryoblastic Leukemia (M7)
Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
Adult Acute Monoblastic Leukemia (M5a)
Adult Acute Monoblastic Leukemia and Acute Monocytic Leukemia (M5)
Adult Acute Monocytic Leukemia (M5b)
Adult Acute Myeloblastic Leukemia With Maturation (M2)
Interventions
Other: laboratory biomarker analysis
Registration Number
NCT00027872
Lead Sponsor
National Cancer Institute (NCI)
Brief Summary

Tipifarnib may stop the growth of cancer cells by blocking the enzymes necessary for their growth. Phase II trial to study the effectiveness of tipifarnib in treating older patients who have previously untreated acute myeloid leukemia

Detailed Description

PRIMARY OBJECTIVES:

I. To determine the complete response rate of R115777 (tipifarnib) in previously untreated acute myeloid leukemia (AML) in (a) elderly patients (age \>= 75) and (b) patients (age \>= 65) with AML preceded by myelodysplastic syndrome (MDS), using a chronic dosing schedule.

SECONDARY OBJECTIVES:

I. To determine progression-free and overall survival in patients with previously untreated AML treated with R115777, using a chronic dosing schedule.

II. To determine the duration of response in patients with previously untreated AML treated with R115777, using a chronic dosing schedule.

III. To determine the effect of R115777 on the phosphorylation of mitogen-activated protein kinase (MAPK) and phosphatidylinositol-4,5-bisphosphate 3-kinase, catalytic subunit alpha (PI3K) in leukemic cells.

IV. To determine the effect of R115777 on processing of the farnesylated protein HDJ-2.

V. To determine the toxicities of R115777 when given in a chronic dosing schedule.

OUTLINE: This is a multicenter study.

Patients receive oral tipifarnib twice daily on days 1-21. Patients with a complete or partial response, hematologic improvement, or stable disease continue treatment every 29-63 days in the absence of disease progression or unacceptable toxicity. Patients with a complete response after the second course of therapy receive 2 additional courses of therapy.

Patients are followed for survival.

PROJECTED ACCRUAL: A total of 125 patients will be accrued for this study within 11-17 months.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
125
Inclusion Criteria
  • Pathologic confirmation of the diagnosis of AML (>= 20% marrow blasts)

  • ECOG performance status 0 or 1

  • Patients must be able to give informed consent

  • SGOT and SGPT =< 2.5 x normal limits (grade 1)

  • Serum creatinine =< 1.5 x normal limits (grade 1)

  • AML (any of the following):

    • Newly diagnosed AML in adults >= 75 years
    • Newly diagnosed AML arising from MDS in adults >= 65 years
  • Hyperleukocytosis with >= 30,000 leukemic blasts/uL

Exclusion Criteria
  • Acute promyelocytic (FAB M3) subtype
  • Previously treated with chemotherapy for leukemia (except for hydroxyurea)
  • Disseminated intravascular coagulation (laboratory or clinical)
  • Active central nervous system leukemia
  • Concomitant radiation therapy, chemotherapy, or immunotherapy; previous therapy for another malignancy is permitted, provided that at least 1 month has occurred since patient received any of these treatments
  • Intrinsic impaired organ function (as stated above)
  • Symptomatic neuropathy (grade 2 or worse)
  • Known allergy to imidazole drugs, such as ketoconazole, miconazole, econazole, teconazole, clotrimazole, fenticonazole, isoconazole, sulconazole, or ticonazole
  • Physical or psychiatric conditions that in the estimation of the principal investigator (PI) or designee place the patient at high risk of toxicity or non-compliance, e.g. severe congestive heart failure (CHF), unstable angina, or poorly controlled psychosis

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Treatment (tipifarnib)laboratory biomarker analysisPatients receive oral tipifarnib twice daily on days 1-21. Patients with a complete or partial response, hematologic improvement, or stable disease continue treatment every 29-63 days in the absence of disease progression or unacceptable toxicity. Patients with a complete response after the second course of therapy receive 2 additional courses of therapy.
Treatment (tipifarnib)tipifarnibPatients receive oral tipifarnib twice daily on days 1-21. Patients with a complete or partial response, hematologic improvement, or stable disease continue treatment every 29-63 days in the absence of disease progression or unacceptable toxicity. Patients with a complete response after the second course of therapy receive 2 additional courses of therapy.
Primary Outcome Measures
NameTimeMethod
Complete remission (CR) rateUp to 8 years

CR rates will be calculated with 95% confidence intervals for each age group separately.

Secondary Outcome Measures
NameTimeMethod
Partial remission (PR) rateUp to 8 years

Will be estimated by observed proportions and 95% confidence intervals.

Toxicity rates assessed using NCI CTCAE version 3.0Up to 8 years

Will be estimated by observed proportions and 95% confidence intervals.

Duration of responseFrom the time measurement criteria are met for CR or PR (whichever is first recorded) until the first date that recurrent or progressive disease is objectively documented, assessed up to 8 years

Duration of response and survival will be summarized by the Kaplan-Meier estimate of the survival distribution.

Duration of survivalFrom time of enrollment onto this study to the time of death, assessed up to 8 years

Duration of response and survival will be summarized by the Kaplan-Meier estimate of the survival distribution.

Trial Locations

Locations (1)

Johns Hopkins University

🇺🇸

Baltimore, Maryland, United States

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