Vanderbilt-Ingram Cancer Center

This is a clinical research study. This trial is sponsored by the pharmaceutical company named Novartis. The study drug is called panobinostat (or LBH589) and is manufactured and developed by Novartis. The purpose of this study is to find out the effect that this drug may have on the patient's disease, both good and bad. Patients are being asked to take part in this study because they have confirmed AML with cell changes, are older than 18 years old but younger than 65 years old, are not pregnant, and do not have a weak heart function. Panobinostat is a drug which has not been approved by the Federal Drug Administration (FDA) or any Health Authority for the treatment of people with the patient's medical condition. The medicine being tested in this study is currently not -on the market- (available to buy) in any country. Panobinostat is a drug that may slow down the spread of the patient's disease by blocking certain substances needed by the cancer cells (proteins produced by cells). Panobinostat has shown effects against diseases such as the patient's in lab studies, and in studies in humans. However, it is not known if this drug will show the same activity or cause improvement in the disease when tested in larger studies in humans and this is the reason for this study to happen. Patients asked to take part in this study may or may not have had several treatments in the past. While other treatments may be useful, your doctor has judged that panobinostat could help slow your disease. As of June 2009, about 800 patients have already received treatment with either an intravenous (IV - given through a vein) or oral form (capsule) of panobinostat. Only the capsule form of panobinostat will be used in this study. About 40 patients will join in this study at about 7 centers potentially in North America, Europe, and Australia. About 2 patients will take part in this study at Vanderbilt.

Criteria
 Inclusion Criteria:

 • Newly diagnosed adult patients = 65 years old with a cytopathologically confirmed diagnosis of high-risk AML

 • = 20% bone marrow blasts via bone marrow aspiration or biopsy

 • The patient has not yet been treated for AML

 • 1º or 2º AML patients with high-risk category features

 • ECOG PS = 2

 • Renal function and liver function limits.

 Exclusion Criteria:

 • Patient with a 'favorable' or 'better-risk' cytogenetic profile = t(15;17); t(8;21); or inv(16) or t(16;16)

 • Patient has clinical symptoms suggestive of CNS leukemia and/or CSF findings for CNS leukemia

 • Prior treatment with deacetylase inhibitors (DACi) including, panobinostat

 • Impaired cardiac function

 • Female patient who is pregnant or breast feeding

 • Male patient who is not willing to use a barrier method of contraception

 Other protocol-defined inclusion/exclusion criteria may apply