Amyloidosis is a disorder in which abnormal proteins build up in tissues and organs. Clumps of the abnormal proteins are called amyloid deposits. Amyloidosis may be treated with chemotherapy, but the disease often returns.
The purpose of this study is to see if treatment with the investigational drug MLN9708 is more effective than standard therapies in patients with systemic light chain amyloidosis that persists or has returned despite prior therapy. Patients will be randomly assigned to receive MLN9708 or a standard oral therapy chosen by the study investigators. All patients will also receive dexamethasone (a steroid commonly used as part of cancer therapy).
MLN9708 is a “proteasome inhibitor” similar to another drug called bortezomib. These drugs inhibit many proteins (known as proteasomes) that cancer cells need to survive and multiply. Unlike bortezomib, which is given intravenously (by vein), MLN9708 is taken orally (by mouth).