This trial is evaluating whether Venetoclax will improve 3 primary outcomes, 1 secondary outcome, and 5 other outcomes in patients with Amyloidosis. Measurement will happen over the course of Up to 30 days.
This trial requires 6 total participants across 1 different treatment group
This trial involves a single treatment. Venetoclax is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 1 and are in the first stage of evaluation with people.
"Most patients received treatment for at least some period of time and most patients experienced success with treatment. Although there is relatively little research on what therapy to use in amyloidosis there is a common tendency to use the same agents that are used for AL." - Anonymous Online Contributor
"We believe that in about 35% of cases, genetical factors are involved in determining amyloidosis. These factors must be related to the production of the immunoglobulin, but some other factor is involved in the process of the deposition of the protein. These factors could be autoimmune disease or chronic inflammation of any tissue." - Anonymous Online Contributor
"Amyloidosis is a collection of pathological changes to tissues which are composed of abnormal proteins and carbohydrates. The predominant protein is amyloid protein. Amyloidosis is caused by multiple causes, but the primary cause is genetic abnormalities, especially hereditary disorders. The diagnosis is made based on a combination of medical history, physical examination and laboratory tests. The mainstay of treatment is alleviation of symptoms and control of the disease." - Anonymous Online Contributor
"It is difficult to say whether or not curing amyloidosis is possible. In our analysis, treatment failed to show any significant improvement on patient health status at 1-, 2-, and 5-years, despite improvement in skin biopsy-based measurements. More intensive therapy is required to positively affect the patients' quality of life and survival, and patients with high pretreatment organ failure need to be considered. Amyloidosis cannot be cured at this time with any therapy available." - Anonymous Online Contributor
"Approximately 13,400 patients die of one of the two main types of amyloidosis (or AL amyloidosis) a year in the U. S. This number (and proportion of these) is increasing. While the majority of patients who die of AA amyloidosis are younger men without signs and symptoms of organ failure, a substantial minority are older men, women, and patients with other forms of amyloidoses associated with organ failure." - Anonymous Online Contributor
"Patients presenting with amyloidosis should have laboratory tests for monoclonal proteins, kidney function and urinalysis. Patients presenting with an acute deterioration in kidney function should have an urgent CT or MRI of the adrenal glands and liver. Patients presenting with unexplained elevations of gamma globulin should be referred for biopsy." - Anonymous Online Contributor
"Amyloidosis is a chronic process that is probably multifactorial and is probably due to dysregulation of protein homeostasis. Its development is probably related to a common defect in the proteins involved in the degradation of amyloid precursor proteins. Abnormal aggregation of misfolded proteins may be the primary cause of amyloidosis." - Anonymous Online Contributor
"In patients with AL, sVXd3 showed improved quality of life compared with sVXg. Recent findings highlight the importance of early identification of patients' disease evolution, in particular that of patients with symptomatic advanced AL." - Anonymous Online Contributor
"Survival is a dynamic measure. Survival has increased over the past 10 years but survival has not increased to an equal degree for people, who have survived with chronic disease over a longer period of time. These data suggest, that the outcome of amyloidosis is no poorer than that for chronic disease." - Anonymous Online Contributor
"Venetoclax is a highly effective treatment to achieve responses in the treatment of most patients with AL amyloidosis. Combination of venetoclax with a bortezomib regimen is a promising approach, which may be associated with a better PFS in the treatment of patients with SAA amyloidosis." - Anonymous Online Contributor
"Venetoclax can be used as a steroid sparing agent in the treatment of advanced or relapsed chronic lymphocytic leukaemia in patients with relapsed or refractory disease, with or without CLL associated cytokine secretion failure, including patients with a Philadelphia chromosome-negative molecular subtype." - Anonymous Online Contributor
"Although one in four patients presenting with AL amyloidosis is eligible for some type of biological therapy—even though clinical trials with these agents have shown high failure rates. We suggest that patients for clinical trials would benefit from being identified in the early stages of the disease." - Anonymous Online Contributor