Venetoclax for Leukemia, Biphenotypic, Acute

Phase-Based Estimates
1
Effectiveness
1
Safety
Fred Hutch/University of Washington Cancer Consortium, Seattle, WA
Leukemia, Biphenotypic, Acute+17 More
Venetoclax - Drug
Eligibility
18+
All Sexes
Eligible conditions
Leukemia, Biphenotypic, Acute

Study Summary

This study is evaluating whether venetoclax in combination with other chemotherapy drugs may help treat acute myeloid leukemia and high-grade myeloid neoplasms.

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Eligible Conditions

  • Leukemia, Biphenotypic, Acute
  • Acute Disease
  • Leukemia, Myeloid
  • Leukemia, Myeloid, Acute
  • Leukemia
  • Cancer
  • Myeloid Neoplasm
  • Refractory Acute Myelogenous Leukemia (AML)
  • Mixed Phenotype Acute Leukemia (MPAL)
  • Acute Myeloid Leukemia (AML)
  • Relapsed Myeloid Neoplasm
  • Acute Biphenotypic Leukemia (ABL)
  • Refractory Myeloid Neoplasm
  • acute, refractory Biphenotypic Leukemia
  • Neoplasms
  • Relapsed Mixed Phenotype Acute Leukemia
  • Relapsed Acute Biphenotypic Leukemia
  • Relapsed Acute Myelogenous Leukemia (AML)
  • Refractory Mixed Phenotype Acute Leukemia

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether Venetoclax will improve 2 primary outcomes and 3 secondary outcomes in patients with Leukemia, Biphenotypic, Acute. Measurement will happen over the course of After 2 cycles (each cycle is approximately 35 days).

Day 35
Complete remission rate
At 1 year
1-year survival rate
Rate of allogeneic hematopoietic cell transplant
Up to 12 months
Incidence of adverse events
Maximum tolerated dose of venetoclax in combination with CLAG-M

Trial Safety

Safety Estimate

1 of 3

Trial Design

2 Treatment Groups

Control
Treatment (CLAG-M, venetoclax)

This trial requires 20 total participants across 2 different treatment groups

This trial involves 2 different treatments. 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 (CLAG-M, venetoclax)Patients will receive induction with granulocyte colony-stimulating factor on days 0-5 (if peripheral white blood cell count is less than 20,000/uL), cladribine on days 1-5, cytarabine on 1-5, and mitoxantrone on days 1-3. Patients also receive venetoclax orally (PO) on days 1-14. Treatment repeats every 28-35 days for up to 2 induction cycles including mitoxantrone, and up to 4 consolidation cycles without mitoxantrone in the absence of disease progression or unacceptable toxicity.
ControlNo treatment in the control group
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Cytarabine
FDA approved
Cladribine
FDA approved
Venetoclax
FDA approved
Mitoxantrone
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: at 1 year
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly at 1 year for reporting.

Closest Location

Fred Hutch/University of Washington Cancer Consortium - Seattle, WA

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. You must have received newly diagnosed for Leukemia, Biphenotypic, Acute or one of the other 17 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:
Subject must have adequate renal function as demonstrated by a creatinine clearance >= 30 mL/min; calculated by the Cockcroft Gault formula or measured by 24 hours urine collection
Acute myeloid leukemia (per the World Health Organization [WHO] 2016 classification) or high-grade myeloid neoplasm (>= 10% myeloid blasts in peripheral blood or marrow)
Bilirubin =< 1.5 x ULN (unless bilirubin rise is due to Gilbert's syndrome or of non-hepatic origin)
Newly diagnosed patients must have adverse risk disease as per the European LeukemiaNet 2017 guidelines
Relapsed/refractory patients must require first or subsequent salvage therapy
Patients with biphenotypic or mixed phenotype acute leukemia are eligible
Age >= 18 years
Aspartate transaminase (AST) and alanine transaminase (ALT) =< 3.0 X upper limit of normal (ULN)
Left ventricular ejection fraction (LVEF) >= 45%, assessed by multigated acquisition (MUGA) or echocardiogram (ECHO) within 3 months prior to study day 0 or after most recent anthracycline administration if appropriate
Eastern Cooperative Oncology Group (ECOG) =< 2

Patient Q&A Section

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

Can leukemia, biphenotypic, acute be cured?

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There is no difference between the two treatment groups in OS or DFS. A difference was found in DSS, with the conventional group having prolonged DSS and a better disease-free status at last visit as compared with the high-dose cyclophosphamide group. These data seem to justify further use of high-dose cyclophosphamide as initial therapy in patients with acute leukemia, biphenotypic, expressing B-cell markers.

Unverified Answer

What causes leukemia, biphenotypic, acute?

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The disease causes by one of two mechanisms: chromosomal abnormalities cause specific gene mutations, which in turn affect the development of leukemia, or exposure to certain [environmental factors can affect development of leukemia.] This latter effect causes the development of all variants of leukemia, biphenotypic and acute.

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What are the signs of leukemia, biphenotypic, acute?

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The signs of leukemia, biphenotypic, acute may include the following: bleeding time longer than 90 seconds, bleeding time longer than 60 seconds and hematocrit less than 35%. In the case of chronic disease, the signs of leukemia, biphenotypic, acutely are fatigue, weight loss, bone and joint pains, fever, anemia, and jaundice or yellowing of the skin or whites of the eyes.

Unverified Answer

What are common treatments for leukemia, biphenotypic, acute?

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Treatment of leukemia often depends on several factors, including specific presentation of the disease, whether or not it is behaving like a leukemoid blast crisis, whether or not the leukemia has entered complete remission, and whether remission is sustained. One-third of patients will require allogeneic stem-cell transplant, and/or other high-intensity therapy to remit leukemia. Patients presenting with a leukemoid blast crisis but sustained complete remission generally have a longer disease-free survival, but their overall survivals are still poor. Patients with B-lymphoblastic leukaemia have some of the best cure rates and longest disease-free survivals after achieving complete remission, but disease-specific relapses are still a problem for many.

Unverified Answer

What is leukemia, biphenotypic, acute?

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Leukemia, biphenotypic, acute, is the most common type of leukemia and a disease from which most people with circulating leukemias die within 5 years after diagnosis. It is a heterogeneous collection of blood diseases characterized by the presence of two or more leukemic cell populations, leukemic blasts or abnormal blood cells and disease-related cytogenetic abnormalities.

Unverified Answer

How many people get leukemia, biphenotypic, acute a year in the United States?

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If a diagnosis of B-PPA is made, then it is not uncommon for patients diagnosed with B-PPA to have an acute leukemia. B-PPA is a rare cause of acute leukemia, and an increase in the incidence and rate of leukemia related to B-PPA in children is expected if there is a genetic predisposition for this condition. The use of the B-PPA label on Medicare claims is not a reliable way of estimating the number of new cases of leukemia that occur in the United States each year.

Unverified Answer

Has venetoclax proven to be more effective than a placebo?

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Although the primary endpoint of the VENTURE 1 trial (first progression) was reached sooner by the treatment group, and OS and progression-free survival were significantly improved at 9 and 11.3 months, the data suggest that the benefit of venetoclax (vs. a placebo) in the secondary endpoint (safety) was not statistically significant. More data are needed to determine the impact of this secondary outcome. There are additional questions to ask about optimal treatment duration, the optimal selection and dose of venetoclax in clinical practice, and the effect of combination therapy (vs. monotherapy) on OS and progression-free survival. Results from a recent paper were presented at the ASCO 2016 Annual Meeting.

Unverified Answer

Does leukemia, biphenotypic, acute run in families?

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There are many cases where an elderly sibling with lymphoma or leukemia has developed a second malignancy as well as a young offspring with a hematologic complication of the acute disease. The possibility of a hematologic malignancy must be considered in these cases.

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What are the common side effects of venetoclax?

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More than 25% of patients treated were affected by AE. The most common AE were diarrhea and thrombocytopenia. Hematologic side effects and infections (especially infections in the respiratory tract) were the most frequent. AE might be caused by venetoclax itself or by the combination used (with cytarabine).

Unverified Answer

What is venetoclax?

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Venetoclax is an investigational drug used to treat chronic lymphocytic leukemia (CLL), myelodysplastic syndrome (MDS), and multiple myeloma (MM) in adults (aged > 18 years) and of ALL in children (aged < 18 years) on a continuous basis in clinical trials. The FDA issued a statement on venetoclax in October 2018 stating that more information about its safety and effectiveness is required.

Unverified Answer

Is venetoclax safe for people?

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For patients with treatment indication for FLT3 inhibitors, venetoclax achieves significant and clinically significant responses when used in the recommended dose and schedule. This data further support venetoclax as a potential treatment option for a broader population.

Unverified Answer

Who should consider clinical trials for leukemia, biphenotypic, acute?

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[This is an expanded version of the article written for the American Society of Hematology (ASH) 2018 Annual Meeting. (https://www.ash-journal.org/content/September2018.). You can find out how to participate in clinical trials by using the Power To Participate in Clinical Trials link on ctrs resource center. (https://www.powerpoint.org/ptp-map).

Unverified Answer
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