study for patients with relapsed or refractory acute myeloid leukemia (aml)

Conditions: Leukemia, Myeloid, Acute

Summary: this is a phase 2, single-arm, open-label, multi-center study to establish the safety and efficacy of troxatyl™ (troxacitabine) administered as a continuous infusion for 5 days to subjects with aml.

this is a phase 2, single-arm, open-label, multi-center study to establish the safety and efficacy of troxatyl™ (troxacitabine) administered as a continuous infusion for 5 days to subjects with aml. the study will primarily assess the complete response (cr) rate of a 5-day continuous infusion of troxacitabine at 12 mg/m2/day given as second salvage therapy in adult patients with aml, with secondary objectives to determine the overall, relapse-free and event-free survival and remission duration; to determine the duration of response; to determine the complete response with incomplete platelet recovery (crp) rate; to evaluate the tolerability and safety of 5-day continuous intravenous (iv) infusion of troxacitabine; and to determine the relationship between troxacitabine plasma concentrations, anti-leukemic activity and adverse events. additional cycles of treatment may be given at the investigator's discretion, provided that the subject does not have progressive disease or experience a dose limiting toxicity. bone marrow transplantation in responding subjects will be allowed.

Eligibility criteria:
inclusion criteria: * confirmed diagnosis of acute myeloid leukemia (aml) in the second salvage setting: refractory to two prior courses of therapy or primary refractory without response to two previous courses of leukemia therapy. * patients must have received at least two previous courses of induction chemotherapy to be considered in the second salvage setting. * patients who are in second relapse, must have had a duration of their second cr or crp of less than six months. * subjects must have adequate organ and immune function as indicated by the following laboratory values: * creatinine clearance ≥ 45 ml/min and ≤ 125 ml/min; * total bilirubin ≤ 2.0 mg/dl (≤ 34.2 µmol/l); * ast (sgot) and alt (sgpt) ≤ 3 x upper limit of normal (uln). * subjects must have an eastern cooperative oncology group (ecog) performance status of \< 2, and an estimated life expectancy of at least eight weeks. exclusion criteria: * clinical evidence of active central nervous system (cns) leukemic involvement * active and uncontrolled infection * uncontrolled medical problems unrelated to the malignancy that impair patient ability to give informed consent or unacceptably reduce the safety of the proposed treatment * neurologic or psychiatric disorders that would interfere with informed consent or study follow-up * known or suspected intolerance or hypersensitivity to troxatyl® or closely related compounds such as lamivudine or any of the stated ingredients * a recent history of alcohol or other substance abuse * subjects who have used another investigational agent or participated in a clinical trial within the last 14 days prior to enrolment * females with a positive pregnancy test at screening * subjects who have previously been enrolled into this study and subsequently withdrew

Keywords: acute myeloid leukemia aml refractory relapsed