Azacitidine for Leukemia, Myeloid, Acute

1
Effectiveness
2
Safety
City of Hope, Duarte, CA
Leukemia, Myeloid, Acute+3 More
Azacitidine - Drug
Eligibility
18+
All Sexes
Eligible conditions
Leukemia, Myeloid, Acute

Study Summary

Venetoclax + Azacitidine vs. Induction Chemotherapy in AML

See full description

Eligible Conditions

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

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether Azacitidine will improve 1 primary outcome and 19 secondary outcomes in patients with Leukemia, Myeloid, Acute. Measurement will happen over the course of Up to 8 weeks.

Month 12
Treatment-related toxicity
Day 100
100-Day post-transplant mortality
Year 3
Event free survival
Month 6
Rate of response
Day 30
30-day mortality
Day 60
60-day mortality
Year 3
Rate of stem cell transplantation (SCT) following induction
Month 6
Rate of Minimal Residual Disease (MRD) negativity
Year 3
Overall survival (OS)
Day 100
Incidence of grade 3 or greater acute graft versus host disease (GVHD)
Up to 8 weeks
Incidence of neutropenic infections
up to 1 year
Cost of care
Health care utilization - Intensive care unit admissions
Health care utilization - days alive and out of the hospital
Health care utilization - hospitalizations
up to one year
Patient reported quality of life (QOL)
Patient-reported anxiety symptoms
Patient-reported depression symptoms
Patient-reported post-traumatic stress symptoms
Patient-reported symptom burden

Trial Safety

Safety Estimate

2 of 3
This is better than 68% of similar trials

Trial Design

2 Treatment Groups

No Control Group
Investigational (Venetoclax and Azacitidine)

This trial requires 172 total participants across 2 different treatment groups

This trial involves 2 different treatments. Azacitidine 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 2 and have already been tested with other people.

Investigational (Venetoclax and Azacitidine)Participants will receive azacitidine on days 1-7 and venetoclax daily for up to (3) three 28-day study cycles and evaluated for response or benefit. If benefit/response is achieved, azacitidine on days 1-7 and venetoclax on days 1-28 (or less if deemed necessary per protocol) will be given in repeating 28-day cycles until benefit/response is no longer achieved or until patient proceeds to transplantation.
Standard of Care (Conventional Induction)Randomized participants will receive cytarabine and idarubicin [or daunorubicin) per standard of care as follows: Induction: cytarabine on days 1-7 and idarubicin (or daunorubicin) on days 1-3 of induction. Second Induction (if needed): Cytarabine on days 1-5 and idarubicin (or daunorubicin) on days 1-2 of re-induction. Consolidation (if needed): If < 60 years, cytarabine days 1,3,5 of consolidation cycles, and if ≥60 years, cytarabine days 1-5 of consolidation cycles Those with secondary or therapy-related AML can receive liposomal daunorubicin and cytarabine (Vyxeos) per standard of care as follows: Induction: Liposomal daunorubicin and cytarabine (Vyxeos) on Days 1,3, 5 of induction. Second Induction (if needed): Liposomal daunorubicin and cytarabine (Vyxeos) on days 1,3 of re-induction Consolidation (if needed): liposomal daunorubicin and cytarabine (Vyxeos) on days 1,3 of consolidation cycles
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Daunorubicin
FDA approved
Cytarabine
FDA approved
Idarubicin
FDA approved
Azacitidine
FDA approved
Venetoclax
FDA approved

Trial Logistics

Trial Timeline

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

Who is running the study

Principal Investigator
A. F.
Amir Fathi, Principal Investigator
Massachusetts General Hospital

Closest Location

City of Hope - Duarte, CA

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 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
De Novo: AML in patients with no clinical history of prior myelodysplastic syndrome (MDS), myeloproliferative disorder, or exposure to potentially leukemogenic therapies or agents
ECOG performance status ≤2 (Karnofsky ≥60%, see Appendix A)
Left ventricular ejection fraction > 50% as measured by echocardiogram or MUGA scan
Total bilirubin ≤ 2.0 x ULN (unless bilirubin rise is known to be due to Gilbert's syndrome or of non-hepatic origin)
Age ≥ 18 years
Secondary AML (sAML): refers to an acute leukemic process (1) evolving from known prior myelodysplasia, myeloproliferative disorder, or aplastic anemia with or without treatment or; (2) as a product of previous exposure to a proven leukemogenic chemotherapeutic agent
Eligible for intensive induction chemotherapy, according to their treating physician
Must not have received systemic prior antineoplastic therapy for treatment for the newly diagnosed AML, including radiation therapy, except hydroxyurea for the purposes of cytoreduction. Patients may also have received all-trans retinoic acid (ATRA) if there is an early suspicion of acute promyelocytic leukemia (APL, M3-AML), although if confirmed to have APL these patients will be excluded from the study.
Aspartate transaminase (AST) and alanine transaminase (ALT) ≤ 3.0X ULN
The effects of venetoclax on the developing human fetus are unknown. For this reason and because other chemotherapeutic agents are known to be teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Women should use contraceptives for at least 30 days following the last dose of venetoclax. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after completion of therapy.

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?

Add answer

Symptoms vary between people with leukemia and those with myeloid acute leukemia. For people with acute leukemia the most common symptoms are fever, chills, headache, and malaise. Fatigue, easy bruising or bleeding, feeling nauseated or tired and shortness of breath are also common.\n

Unverified Answer

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

Add answer

About 3500 people die from myeloid leukemia in the US each year. About 20% of myeloid leukemia deaths occur in children under 15, 20-30% of all leukemia deaths occur in people 35-44 years of age, and approximately 70% of all leukemia deaths occur in men. About 5000 people die from lymphoid leukemia every year. About 20% of lymphoid leukemia deaths occur in children under 15, 20-30% of lymphoid leukemia deaths occur in people 35-44 years of age, and approximately 80% of lymphoid leukemia deaths occur in men. About 2000 people die from acute leukemia in the US each year. Approximately 75% of acute leukemia deaths involve acute promyelocytic leukemia.

Unverified Answer

What is leukemia, myeloid, acute?

Add answer

Leukemia, myeloid, acute is the second most frequent cause of cancer-related deaths in the United States, following lung cancer. The annual incidence of leukemia-myeloid-acute is about 40 new cases per 100,000 people per year. About 40% of leukemia-myeloid-acute people also have myelodysplastic syndrome (MDS) at diagnosis, so the incidence of leukemia-myeloid-acute will increase due to the increased incidence of MDS. About 80% of people with leukemia-myeloid-acute are in good clinical grade at presentation, so the treatment of leukemia-myeloid-acute could be improved.

Unverified Answer

What are common treatments for leukemia, myeloid, acute?

Add answer

The common treatments for leukemia, myeloid, acute are diverse and include chemotherapy, allerbutics, biomedicines, antimetabolites, and leukotriene modifiers. Leukotriene modifiers appear to represent a valid treatment option for patients with acute myeloid leukemias or myelodysplastic syndromes who cannot tolerate standard chemotherapies.

Unverified Answer

Can leukemia, myeloid, acute be cured?

Add answer

Patients with AML-M are rare and have dismal prognoses. AML-M is currently not curable for most patients. Patients with AML-M without an M1-M3 or a bulky M3 have good to excellent survival, while patients with M1 or bulky M3 have fatal disease. AML-M can be treated and patients can benefit from treatment. For the M1-M3 patients, more effective treatments are warranted. Patients with bulky M3 disease may benefit from newer agent- combinations.

Unverified Answer

What causes leukemia, myeloid, acute?

Add answer

Multiple myeloma can be caused by a genetic predisposition as well as exposure to environmental agents. Many types of leukemias originate in the bone marrow. The exact cause is unknown. The exact cause of acute myelogenous leukemia is unknown, but it is known to be associated with viruses and certain environmental agents.

Unverified Answer

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

Add answer

Azacitidine improves QoL in patients with AML and myeloid, acute leukemias. The improvements in QoL are not only related to cytoreduction and marrow aplasia reduction but also to symptomatic relief, fatigue/mood enhancement (at 12 weeks), and better treatment tolerance (tuberous sclerosis complex, or TSC). Azacitidine also seems to have a modest role in treatment-resistant myeloid, acute leukemias.

Unverified Answer

What are the common side effects of azacitidine?

Add answer

Azacitidine was well tolerated. Commonly reported general tolerability adverse effects were nausea (24%; 5/17 patients), fatigue (17%; 4/17 patients), and loss of appetite (17%; 4/17 patients).

Unverified Answer

Is azacitidine typically used in combination with any other treatments?

Add answer

Patients receiving azacitidine in combination with other treatments generally experience more disease progression at time of report than those receiving azacitidine alone. Because many patients experience a delay in referral by their oncologists after initiating treatment, the inclusion of azacitidine in the treatment of pediatric patients undergoing induction therapy with chemotherapy has a number of important implications for clinical trials of pediatric hematologic malignant neoplasms, especially those involving chronic myeloid leukemia.

Unverified Answer

Does leukemia, myeloid, acute run in families?

Add answer

This is the largest family study of acute lymphocytic leukemia susceptibility among Caucasians to date. Findings from a recent study show that this disorder is likely to be due to multiple genes and environmental exposures in an additive manner.

Unverified Answer

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

Add answer

Results from a recent clinical trial suggest that survival varies for leukemia, myeloid, acute survivors. The survival rate increases consistently with age at diagnosis for leukemia and myeloid, acute. In leukemia patients, survivors seem to have different survival rates depending on the time of diagnosis. Patients who were diagnosed>5 years ago had higher survival rates than survivors who were diagnosed<5 years ago. We do not know if this was a real effect of earlier treatment or treatment-related variables.

Unverified Answer

How does azacitidine work?

Add answer

Although clinical trials suggest that the response to azacitidine in MDS is less than 10% in patients with CMML, our results indicate that azacitidine may be an important medication in CMML treatment. More clinical trials are warranted.

Unverified Answer
See if you qualify for this trial
Get access to this novel treatment for Leukemia, Myeloid, Acute by sharing your contact details with the study coordinator.