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A Trial of Venetoclax and Dexamethasone for Systemic AL Amyloidosis
||A Phase I Trial of Venetoclax (ABT-199) and Dexamethasone for Relapsed or Refractory Systemic AL Amyloidosis
||Raymond Comenzo, MD
The study is being conducted to determine the safety, tolerability and maximum tolerated dose of Venetoclax and dexamethasone in relapsed or refractory amyloid light chain (AL) amyloidosis patients. AL amyloidosis is a disease involving cells called plasma cells that make antibodies as part of your immune system. These cells are not functioning the way they are supposed to and they start to produce abnormal fragments of antibodies that are toxic to your body and can form amyloid. The antibody fragments are called "light chains." They can cause damage to organs, especially the kidneys, heart, skin, liver, and lungs.
Researchers are looking for ways to stop the light chains from being formed to treat the disease. Under some circumstances, patients will receive chemotherapy drugs in order to manage the disease. However, researchers do not know what the best treatment is for relapsed AL amyloidosis, so the researchers are testing new drugs or new combinations of drugs to see what will work best with the least side effects.
The researchers want to find out if Venetoclax (ABT-199) in addition to dexamethasone will reduce or eliminate AL amyloidosis plasma cells.
- Histologic diagnosis of AL amyloidosis, confirmed by positive Congo red stained biopsy, with evidence of measurable clonal disease according that requires active treatment
- Relapsed or refractory after at least 1 prior therapy for AL amyloidosis and, in the investigator's opinion, require further treatment.
- Less than 30% plasma cells in the bone marrow biopsy and no bone lesions or hypercalcemia.
- Active fungal infection requiring continued therapy.
- Clinically overt multiple myeloma (bone marrow plasma cells > 30%) and bone lesions or hypercalcemia.
- Patients with non-AL amyloidosis.
In order to determine if the plasma cells appear to be killed by Venetoclax, a screening test on subject’s bone marrow plasma cells is required. In this study, varying doses of Venetoclax will be given to gain a better understanding of the dose of Venetoclax and dexamethasone that best counters AL amyloidosis with the least toxicity. Subjects will have a bone marrow aspirate in addition to the bone marrow biopsy. They will also have physical exams, ECG, ECHO, blood and urine collection as well as other tests and assessment per protocol.