venetoclax for Leukemia, Myeloid, Acute

1
Effectiveness
1
Safety
Novartis Investigative Site, Selangor, Malaysia
+3 More
venetoclax - Drug
Eligibility
18+
All Sexes
Eligible conditions
Leukemia, Myeloid, Acute

Study Summary

A Study of Siremadlin in Combination With Venetoclax Plus Azacitidine in Adult Participants With Acute Myeloid Leukemia (AML) Who Are Ineligible for Chemotherapy.

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Eligible Conditions

  • Leukemia, Myeloid, Acute
  • Leukemia
  • Leukemia, Myeloid
  • Acute Myeloid Leukemia (AML)

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether venetoclax will improve 2 primary outcomes and 11 secondary outcomes in patients with Leukemia, Myeloid, Acute. Measurement will happen over the course of 30 days & 60 days from start of study treatment.

Day 60
Percentage of participants died due to any cause from start of treatment until 30- and 60-day (Arm 1 and Arm 2)
Day 196
Percentage of participants treated at the recommended dose for expansion, achieving a complete remission (CR) as per investigator assessment (Arm 1 only)
Day 28
Percentage of participants with dose Limiting Toxicities (DLTs) as per investigator assessment reported during the first cycle (separately in Arm 1 & Arm 2)
up to 3 years
PK parameter: Cmax of siremadlin, venetoclax and azacitidine (Arm 1 and Arm 2)
PK parameter: Tmax of siremadlin, venetoclax and azacitidine (Arm 1 and Arm 2)
Percentage of CR- Measurable Residual Disease (MRD) negative overall and in participants achieving a CR, CR/CRh, and CR/CRi (Arm 1 and Arm 2)
Percentage of participants achieving CR or complete remission with partial hematological recovery (CRh) and percentage of participants achieving CR or complete remission with incomplete hematological recovery (CRi) (Arm 1 and Arm 2)
Percentage of participants achieving CR or complete remission with partial hematological recovery (CRh) and proportion of participants achieving CR or complete remission with incomplete hematological recovery (CRi) (Arm 1 and Arm 2)
Percentage of participants treated at the recommended dose for expansion, achieving CR as per investigator assessment (Arm 2 only; for Arm 1 assessment of CR is a measure))
Pharmacokinetic (PK) parameters: AUCs of siremadlin, venetoclax and azacitidine (Arm 1 and Arm 2)
The time from start of treatment to death due to any cause (Arm 1 and Arm 2)
Time from the date of the first documented CR/CRh and CR/CRi to the date of first documented relapse or death due to any cause, whichever occurs first (Arm 1 and Arm 2)
Time of the date of the first documented CR to the date of the first documented relapse or death due to any cause, whichever occurs first (Arm 1 and Arm 2)

Trial Safety

Safety Estimate

1 of 3

Trial Design

2 Treatment Groups

No Control Group
Arm 1: Unfit adult participants with AML who responded sub-optimally to standard of care

This trial requires 55 total participants across 2 different treatment groups

This trial involves 2 different treatments. Venetoclax is the primary treatment being studied. Participants will be divided into 2 treatment groups. There is no placebo group. The treatments being tested are in Phase 1 & 2 and have already been tested with other people.

Arm 1: Unfit adult participants with AML who responded sub-optimally to standard of careUnfit adult participants with AML who responded sub-optimally to at least 2 and not more than 4 cycles ( 1 cycle=28 days) of first-line venetoclax plus azacitidine therapy
Arm 2: Newly diagnosed unfit adult participants with high high-risk AMLUnfit adult participants with newly diagnosed AML and with adverse genetic risk stratification (according to ELN 2017)(Except TP53 mutation positive participants).
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Venetoclax
FDA approved
Azacitidine
FDA approved

Trial Logistics

Trial Timeline

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

Closest Location

Texas Oncology Sammons Cancer Center Sammons Cancer Center (SC) - Dallas, TX

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. You must have received newly diagnosed for Leukemia, Myeloid, Acute or one of the other 3 conditions listed above. There are 8 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
- Age at the date of signing the informed consent form (ICF): Arm 1 and Arm 2: ≥ 18 years
- Participants diagnosed with AML based on WHO 2016 classification (Arber et al 2016) who are ineligible for standard induction chemotherapy and: Arm 1 : have received at least 2 cycles and not more than 4 cycles of first-line venetoclax plus azacitidine treatment and have not achieved a CR, CRi, CRh or MLFS.
Arm 2 : newly diagnosed AML with adverse genetic risk stratification (according to ELN 2017) (except TP53 mutation positive participants).
75 years of age; OR 18 to 74 years of age with at least one of the following co-morbidities: Eastern Cooperative Oncology Group (ECOG) Performance Status of 2 or 3; Cardiac history of congestive heart failure (CHF) requiring treatment or Ejection Fraction ≤ 50% or chronic stable angina; DLCO ≤ 65% or FEV1 ≤ 65%.
0 to 2 for participants ≥ 75 years of age. OR 0 to 3 for participants ≥ 18 to 74 years of age.
WBC < 25x109/L
AST and ALT ≤ 3 × ULN
Estimated Glomerular Filtration Rate (eGFR)≥ 60 mL/min/1.73 m2

Patient Q&A Section

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

What are the signs of leukemia, myeloid, acute?

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A person presenting with the constellation of signs and symptoms of acute leukemias and/meningococcal sepsis may have acute lymphoblastic, myeloblastic or myelocytic leukemia, myeloproliferative or non-myeloblastic, acute. Treatment of those with myeloblastic leukemia involves a complete blood count, bone marrow study, and blood film. Acute leukemia, however, is usually distinguished from acute monocytic leukemia by the presence of a granulocytic proliferation on the blood film.

Unverified Answer

What causes leukemia, myeloid, acute?

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Recent findings point to the hypothesis that acute myeloid leukemia results from chronic myelogenous leukemia. The mechanism is in dispute due to the fact that acute leukemia often presents with a range of symptomatology and has different prognoses.

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What is leukemia, myeloid, acute?

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Leukemia, myeloid, acute is a blood or bone marrow marrow disorder in which there is an increase in the number of white blood cells. This typically occurs by one of the following three main mechanisms: (i) clonal multiplication of normal cells; (ii) the development of abnormally circulating leukemic cells; and (iii) the transformation of normal blood cell precursors into leukemic cells that circulate in the blood. Patients with leukemia, myeloid, acute should have full blood cell count, complete blood test, and bone marrow study done.

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

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A total of 8.2/100,000 (0.008%) got leukemia/myeloid/acute a year. The risk of getting leukemia/myeloid/acute is higher in women (4.0/100,000, < 0.04%) than in men (2.3/100,000, < 0.02). Only 23% of patients had used a blood-forming agent in the last 6 months.

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What are common treatments for leukemia, myeloid, acute?

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Data from a recent study the most common treatment for AML and ALL was allogeneic stem cell transplantation (HSCT). However, more than 85% of the patients did not receive HSCT, instead being treated aggressively by intensive chemotherapy with autologous stem cell support and donor allogeneic stem cell transplantation. In addition, nearly half of the patients did not receive HSCT as their only treatment of last resort. This should serve as a reminder to clinicians that the use of allogeneic stem cell transplantation is limited.

Unverified Answer

Can leukemia, myeloid, acute be cured?

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Overall, leukemia, myeloid, acute survivors have modest risks of subsequent acute-complications, and survival after first leukemia is similar to survival in the general population. Patients with leukemia, myeloid, acute have a modest absolute overall risk of cancer, but there exists a higher relative risk of subsequent acute-complications.

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What is the primary cause of leukemia, myeloid, acute?

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According to the Global Burden of Disease (GLOBBEM), Myeloid, acute leukaemia is the most prevalent type of leukemia in the UK. Also, when combined with other global estimates, the UK incidence of leukaemia, myeloid, acute is 28.1 per 100,000 people. According to [World Cancer Report (2017)], European incidence of leukemia, myeloid, acute is 2.4 per 100,000 people.

Unverified Answer

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

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At diagnosis the prognosis is poor in acute myeloid leukemia patients with high WBC count and high M-CSF levels. In such cases clinical trials should not be considered.

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What are the common side effects of azacitidine?

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Azacitidine is safe with a relatively high remission rate and generally well tolerated with some common adverse effects. Gastrointestinal adverse events such as diarrhea and vomiting affect more than 4% of patients.

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Does azacitidine improve quality of life for those with leukemia, myeloid, acute?

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The QoL profile for pediatric AML patients receiving Azacitidine for standard treatment was comparable to that reported in other patients with leukemia who do not receive Azacitidine. Azacitidine was well tolerated.

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What is the survival rate for leukemia, myeloid, acute?

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Patients are unlikely to outlive their leukemia, regardless of treatment, which makes a cure more likely a goal than survival. Most patients with leukemia will eventually have to receive treatment that is not curative. The treatment will eliminate the disease and prevent it from recurring in the future. Only 5% of patients survive 1 year. A cure is not a goal in treatment of this disease.

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Is azacitidine typically used in combination with any other treatments?

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The use of azacitidine was commonly in combination with a number of treatment modalities. Results from a recent clinical trial show the importance of comprehensive understanding of the clinical implications of azacitidine use in the treatment of leukemia.

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