NCT02210858

Tipifarnib in Treating Patients With Chronic Myeloid Leukemia, Chronic Myelomonocytic Leukemia, or Undifferentiated Myeloproliferative Disorders

Phase I/II Study of Tipifarnib [Zarnestra, Farnesyltransferase Inhibitor R115777 (NSC 702818)] in Patients With Myeloproliferative Disorders

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 31 (actual)
• Sponsor: National Cancer Institute (NCI) · NIH
• Sex: All
• Age: 21 Years
• Healthy volunteers: Accepted

Summary

This phase 1-2 trial studies the side effects and how well tipifarnib works in treating patients with chronic myeloid leukemia, chronic myelomonocytic leukemia, or undifferentiated myeloproliferative disorders. Tipifarnib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

Detailed description

PRIMARY OBJECTIVES: * To describe the toxicities of R115777 (tipifarnib) in adult patients with myeloproliferative disorders. * To assess hematologic responses, including changes in white blood cell count and erythroid responses. SECONDARY OBJECTIVES: * To assess bone marrow cytogenetic responses to R115777. * To analyze for the presence of neuroblastoma (N)/Kirsten rat sarcoma viral oncogene homolog (K-Ras) mutations in patient bone marrow samples. * To analyze the effect of R115777 on Ras /DnaJ (Hsp40) homolog, subfamily A, member 1(HDJ-2) farnesylation in patient bone marrow/peripheral blood mononuclear cells. * To analyze the effect of R115777 on mitogen-activated protein (MAP) kinase activation in patient bone marrow mononuclear cells. * To perform colony forming unit granulocyte-macrophage (CFU-GM) cytotoxicity assays using patients' hematopoietic cells with R115777. OUTLINE: Patients receive tipifarnib orally (PO) twice daily (BID) on days 1-21. Treatment repeats every 28 days for up to 4 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a good hematologic response may continue treatment at the discretion of the treating physician.

Conditions

• Accelerated Phase of Disease
• Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative
• Chronic Myelogenous Leukemia, BCR-ABL1 Positive
• Chronic Myelomonocytic Leukemia
• Chronic Phase of Disease
• Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable
• Recurrent Disease

Interventions

Type	Name	Description
OTHER	Laboratory Biomarker Analysis	Correlative studies
DRUG	Tipifarnib	Given PO

Timeline

• Start date: 2000-05-01
• Primary completion: 2004-11-12
• Completion: 2017-03-01
• First posted: 2014-08-07
• Last updated: 2018-06-04

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study