Venetoclax for Amyloidosis

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
City of Hope Comprehensive Cancer Center, Duarte, CA
Amyloidosis+3 More
Venetoclax - Drug
Eligibility
18+
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether a combination of drugs can treat light chain amyloidosis.

See full description

Eligible Conditions

  • Amyloidosis
  • Recurrent AL Amyloidosis
  • Refractory AL Amyloidosis

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Amyloidosis

Study Objectives

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.

Year 5
Overall response rate (complete hematologic response)
Baseline
Expression of BCL-2, BCL-XL, BAX, BAK, BIM, NOXA, and MCL-1
Before and during treatment
Immune profile in the peripheral blood
Up to 2.5 years
Characterization of CD138+ plasma cell with t(11;14)
Hematologic response rates
Presence of minimal residual disease
Up to 30 days
Incidence of adverse events
Up to the end of cycle 1
Maximum tolerated dose
Recommended phase 2 dose (RP2D)

Trial Safety

Safety Progress

1 of 3

Other trials for Amyloidosis

Trial Design

1 Treatment Group

Treatment (venetoclax, ixazomib citrate, dexamethasone)
1 of 1
Experimental Treatment

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.

Treatment (venetoclax, ixazomib citrate, dexamethasone)Patients receive venetoclax PO QD on days 1-28, ixazomib citrate PO on days 1, 8 and 15, and dexamethasone PO on days 1, 8, 15 and 22. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ixazomib
FDA approved
Dexamethasone
FDA approved
Venetoclax
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 2.5 years
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly up to 2.5 years for reporting.

Closest Location

City of Hope Comprehensive Cancer Center - Duarte, CA

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. You must have received 1 prior treatment for Amyloidosis or one of the other 3 conditions listed above. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
You have t(11;14) by FISH on bone marrow biopsy, to be confirmed at screening. show original
Histologically-proven systemic anti-light chain amyloidosis (AL) confirmed by positive Congo red staining with green birefringence on polarized light microscopy and evidence of a measurable clonal disease that requires active treatment. Patients must have measurable disease with dFLC >= 4 mg/dL (40mg/L). For patients who are African-American, mass spectrometry must be performed to confirm subtyping
Evidence of relapsed, refractory, or progressive disease following at least one line of treatment as defined by National Comprehensive Cancer Network (NCCN) guidelines (no limit on the number of prior treatments). Hematologic relapse/progression is defined by the reappearance of a detectable monoclonal protein or abnormal serum free light-chain ratio after having achieved a hematologic complete response or a 50% increase in serum M protein or urine M protein to > 0.5 g/dL or > 200 mg/day, respectively, or a free light-chain increase of 50% to >100 mg/L in those with stable disease or partial response. Refractory disease is progression on a previous line of therapy without response
Children under 18 years of age are excluded from this study. show original
Eastern Cooperative Oncology Group (ECOG) performance status =< 2 (Karnofsky >= 60%)
Leukocytes must be at least 3,000/mcL. show original
Absolute neutrophil count >= 1,000/mcL. Screening absolute neutrophil count (ANC) should be independent of granulocyte- and granulocyte/macrophage colony stimulating factor (G-CSF and GM-CSF) support for at least 1 week and of pegylated G-CSF for at least 2 weeks
Platelets must be at least 75,000/mcL before study enrollment. show original
Total bilirubin =< 1.5 x institutional upper limit of normal (ULN)
Aspartate aminotransferase (AST)(serum glutamic-oxaloacetic transaminase [SGOT])/alanine aminotransferase (ALT)(serum glutamate pyruvate transaminase [SGPT]) =< 3 x institutional ULN

Patient Q&A Section

What are common treatments for amyloidosis?

"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

Unverified Answer

What causes amyloidosis?

"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

Unverified Answer

What is amyloidosis?

"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

Unverified Answer

Can amyloidosis be cured?

"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

Unverified Answer

How many people get amyloidosis a year in the United States?

"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

Unverified Answer

What are the signs of amyloidosis?

"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

Unverified Answer

What is the primary cause of amyloidosis?

"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

Unverified Answer

Does venetoclax improve quality of life for those with amyloidosis?

"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

Unverified Answer

What is the survival rate for amyloidosis?

"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

Unverified Answer

Is venetoclax typically used in combination with any other treatments?

"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

Unverified Answer

What is venetoclax?

"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

Unverified Answer

Who should consider clinical trials for amyloidosis?

"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

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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