A Phase I, Open-label, Multi-center Study of Clofarabine (JC0707) in Japanese Patients With Acute Myeloid Leukemia (AML)
This is a Phase I, open-label, multi-center study of Clofarabine administered to Japanese
patients with Acute Myeloid Leukemia (AML) who are relapsed/refractory or elderly untreated
AML for whom standard induction chemotherapy is unlikely to be of benefit.
Cohort 1 will receive 20 mg/m2/day of Clofarabine once daily for five consecutive days,
Cohort 2 will receive 30 mg/m2/day, and Cohort 3 will receive 40 mg/m2/day. Patients will
receive one cycle as a rule. However, if there is evidence of some hematologic response
after one cycle of treatment with Clofarabine, patients may receive up to a maximum of three
cycles. If patients fail to achieve CR or CRp after two cycles of treatment with
Clofarabine, further dosing for such patients should be stopped.
Three patients constituting a cohort will receive Clofarabine and will then be assessed for
dose limiting toxicities (DLT) at Cycle 1. If none of these three patients develops DLT, the
next cohort will be introduced. If one of them develops DLT, three new patients will be
added to the cohort, so that six patients in total are included in the tolerability
assessment. In this case, treatment of the next cohort is allowed only in the case the
number of patients who develop DLT is still one in this six-patient cohort. If two of the
six patients develop DLT, however, the tolerability is ruled out. However, if two DLTs are
observed at the 20mg/m2/day dose cohort, new patients will be enrolled at cohort -1
15mg/m2/day; and if no more than one of six patients in the cohort develop DLT, it will be
considered as the last cohort for this study.
Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Maximum Tolerated Dose (MTD) as determined by Dose Limiting Toxicities (DLTs)
28 days (1st cycle)
Japan: Pharmaceuticals and Medical Devices Agency