Clinical trial · Interventional
Tipifarnib in Treating Young Patients With Refractory Leukemia
A Phase I Trial and Pharmacokinetic Study of R115777 in Pediatric Patients With Refractory Leukemia
NCT00022451CI-TRIAL-00006487completedPhase 1ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
RATIONALE: Tipifarnib may stop the growth of cancer cells by blocking the enzymes necessary for cancer cell growth. PURPOSE: Phase I trial to study the effectiveness of tipifarnib in treating young patients who have refractory leukemia.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Leukemia | Leukemia | ONTOLOGY_EXACT | 0.90 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| tipifarnib | Drug | Tipifarnib | ALIAS |
Eligibility
Eligibility (as posted)
- Sex
- All
- Maximum age
- 21 Years
Show eligibility criteria text
DISEASE CHARACTERISTICS: * Histologically confirmed acute lymphoblastic leukemia, acute nonlymphoblastic leukemia, juvenile myelomonocytic leukemia (JMML), or chronic myelogenous leukemia (CML) in blast crisis * Refractory to standard curative therapy * Acute promyelocytic leukemia refractory to tretinoin and arsenic trioxide * Philadelphia chromosome-positive CML refractory to imatinib mesylate * Greater than 25% blasts in bone marrow (M3 bone marrow) except for patients with JMML * Active extramedullary disease allowed * No active leptomeningeal leukemia PATIENT CHARACTERISTICS: Age: * 21 and under Performance status: * Karnofsky 50-100% (over 10 years of age) * Lansky 50-100% (10 years of age and under) Life expectancy: * Not specified Hematopoietic: * Not required to be normal Hepatic: * Bilirubin normal * SGPT and SGOT normal * No significant hepatic dysfunction * No grade 3 or 4 liver function test results within the past month Renal: * Creatinine normal OR * Creatinine clearance at least 60 mL/min * No significant renal dysfunction Cardiovascular: * No significant cardiac dysfunction Pulmonary: * No significant pulmonary dysfunction Neurologic: * No history of grand mal seizures grade 3 or greater except febrile seizures * No persistent sensory or motor neuropathy greater than grade 2 Other: * No clinically significant unrelated systemic illness * No serious infection * No organ dysfunction that would preclude study participation * No requirement for total parenteral nutrition * No known allergy to azoles (e.g., clotrimazole, fluconazole, ketoconazole, voriconazole) * Not pregnant or nursing * Negative pregnancy test * Fertile patients must use effective contraception PRIOR CONCURRENT THERAPY: Biologic therapy: * At least 1 week since prior colony-stimulating factor therapy (e.g., filgrastim \[G-CSF\] or sargramostim \[GM-CSF\]) except epoetin alfa * At least 3 months since prior myeloablative therapy followed by bone marrow or stem cell transplantation * No concurrent immunotherapy * No concurrent GM-CSF or interleukin-11 Chemotherapy: * At least 2 weeks since prior chemotherapy * No concurrent intrathecal chemotherapy * No other concurrent chemotherapy Endocrine therapy: * At least 1 week since prior corticosteroids * No concurrent corticosteroids (except for acute allergic reaction) Radiotherapy: * At least 4 weeks since prior radiotherapy * No concurrent radiotherapy Surgery: * Not specified Other: * Recovered from nonhematologic toxicity of all prior therapy * At least 1 week since prior retinoids * No antacids (magnesium- or aluminum-containing formulations) within 2 hours of study drug * No other concurrent investigational agents * No concurrent retinoids * No concurrent anticonvulsants
References
Publications (2)
- BACKGROUNDde Nigris F, Balestrieri ML, Napoli C. Targeting c-Myc, Ras and IGF cascade to treat cancer and vascular disorders. Cell Cycle. 2006 Aug;5(15):1621-8. doi: 10.4161/cc.5.15.3138. Epub 2006 Aug 1. PMID 16921263
- RESULTWidemann BC, Arceci RJ, Jayaprakash N, Fox E, Zannikos P, Goodspeed W, Goodwin A, Wright JJ, Blaney SM, Adamson PC, Balis FM. Phase 1 trial and pharmacokinetic study of the farnesyl transferase inhibitor tipifarnib in children and adolescents with refractory leukemias: a report from the Children's Oncology Group. Pediatr Blood Cancer. 2011 Feb;56(2):226-33. doi: 10.1002/pbc.22775. Epub 2010 Sep 21. PMID 20860038