Clinical Trial: Trial of Venetoclax (ABT-199) and Dexamethasone for Relapsed or Refractory Systemic AL Amyloidosis

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Phase I Trial of Venetoclax (ABT-199) and Dexamethasone for Relapsed or Refractory Systemic AL Amyloidosis

Brief Summary: This is a study to determine the safety, tolerability and maximum tolerated dose of Venetoclax (ABT-199) and dexamethasone in relapsed or refractory amyloid light chain (AL) amyloidosis patients.

Detailed Summary:

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. In this study, varying doses of Venetoclax will be given to determine the maximum tolerated and safe dose for further study. The researchers may also gain a better understanding of whether Venetoclax and dexamethasone can counter the plasma cell disease that causes AL amyloidosis.


Sponsor: Tufts Medical Center

Current Primary Outcome: Participants with treatment related adverse events using NCI CTCAE version 4.03. [ Time Frame: Up to 8 months after beginning study drug ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Hematologic response based on serum free light chain (FLC) response criteria. [ Time Frame: Up to 8 months after beginning study drug ]
    Complete response is normalization of FLC levels and ratio with negative immunofixation studies of serum and urine. Very good partial response is reduction of the difference between the involved and uninvolved FLC to less than 40mg/L. Partial response is reduction of the difference between involved and uninvolved FLC of greater than 50% baseline. No response or stable disease is none of the above.
  • Proportion of subjects with progression-free survival [ Time Frame: Up to 8 months after beginning study drug ]
  • Overall survival of subjects [ Time Frame: Up to 8 months after beginning study drug ]


Original Secondary Outcome: Same as current

Information By: Tufts Medical Center

Dates:
Date Received: December 12, 2016
Date Started: January 2017
Date Completion: January 2021
Last Updated: January 10, 2017
Last Verified: January 2017