Trial of Venetoclax (ABT-199) and Dexamethasone for Relapsed or Refractory Systemic AL Amyloidosis
|Study Design:||Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
|Official Title:||A Phase I Trial of Venetoclax (ABT-199) and Dexamethasone for Relapsed or Refractory Systemic AL Amyloidosis|
- Participants with treatment related adverse events using NCI CTCAE version 4.03. [ Time Frame: Up to 8 months after beginning study drug ]
- 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 ]
|Study Start Date:||January 2017|
|Estimated Study Completion Date:||January 2021|
|Estimated Primary Completion Date:||January 2019 (Final data collection date for primary outcome measure)|
Experimental: Venetoclax and Dexamethasone
Venetoclax will be given at one of four escalating doses (100 mg/day, 200 mg/day, 400 mg/day, or 800 mg/day) by mouth on each day of the cycle. Dexamethasone will be given at 20mg by mouth on days 1, 8, 15, and 22 of each cycle.
Venetoclax at one of four escalating doses
Other Name: ABT-199Drug: Dexamethasone
Dexamethasone 20mg by mouth on days 1, 8, 15, and 22 of each cycle.
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT03000660
|Contact: George Mensingfirstname.lastname@example.org|
|Contact: Elizabeth Kendricken, RN, BSNemail@example.com|
|United States, Massachusetts|
|Tufts Medical Center||Recruiting|
|Boston, Massachusetts, United States, 02111|
|Contact: George Mensing 617-636-2683 firstname.lastname@example.org|
|Contact: Elizabeth Kendricken, RN, BSN 617-636-5558 email@example.com|
|Principal Investigator: Raymond Comenzo, MD|
|Sub-Investigator: Cindy Varga, MD|
|Sub-Investigator: Kellie Sprague, MD|
|Principal Investigator:||Raymond Comenzo, MD||Tufts Medical Center|