A Phase I Feasibility Study Assessing the Role of Maintenance Oral Clofarabine in Older Adults With Acute Myeloid Leukemia
Standard induction chemotherapy (chemotherapy given with the intent of inducing a
remission/disappearance of the cancer) can lead to a complete remission (CR) in a large
percentage (60% to 80%) of younger patients with newly diagnosed AML. However, the majority
of patients relapse (their disease returns) despite intensive consolidation chemotherapy. In
most chemotherapy studies, only a small percentage (30% to 40%) of patients who achieve a CR
still are disease-free 5 years later. The outcome for older adults is even less favorable.
Approximately 75% of elderly patients who achieve CR will relapse after 2 years or less and
the toxicities seen with treatment are significant. A number of studies have shown that
chemotherapy given to older adults after a remission has not improved this outcome;
therefore new treatments need to be investigated. Clofarabine is a drug that has been
studied in older adults who have achieved a CR. The treatment was found to have fewer side
effects then other consolidation therapies. This study will give participants the drug for a
longer period of time to see if doing this will extend the cancer remission. Clofarabine has
been approved by the FDA for the treatment of acute lymphoblastic leukemia (ALL), another
form of acute leukemia. The study drug is considered experimental for AML because it has not
been approved by the Food and Drug Administration (FDA) for the treatment of AML.
Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Determine the maximum tolerated dose of oral clofarabine.
at study completion (estimated at 2 years)
Jessica Altman, MD
United States: Food and Drug Administration