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Acute Myeloid Leukemia in RemissionPhase 2NCT04062266

AZA + Venetoclax as Maintenance Therapy in Patients With AML in Remission

A Phase 2 study testing Azacitidine and Venetoclax in people with Acute Myeloid Leukemia in Remission and FLT3 Gene Mutation, run by M.D. Anderson Cancer Center at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

This phase II trial studies how well azacitidine and venetoclax work in treating patients with acute myeloid leukemia that is in remission. Drugs used in chemotherapy, such as azacitidine and venetoclax, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from… Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Azacitidine and Venetoclax
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
36 participants
Where
1 siteUnited States
Study dates
13 Sep 2019to 12 Oct 2026
Phase
Phase 2

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Patients aged >/= 18 years AML who have achieved their FIRST CR or CRi and are not immediately candidates for allogeneic stem cell transplant.
  • Patients who have received intensive therapy (defined as receiving standard or higher dose cytarabine-based therapy) to achieve remission (CR/CRi) should have received remission induction therapy and at least 1 consolidation cycle. These patients are eligible as long as they are not greater than 2 months from their last consolidation therapy and will be enrolled in COHORT 1.
  • Patients who have received lower intensity therapy (defined as receiving low-dose cytarabine (LDAC) or hypomethylating agent (HMA)-based therapy) to achieve remission should have received at least 2 cycles of lower intensity therapy between the time they have achieved CR/CRi and enrollment on this protocol. They will be treated on COHORT 2.
  • For either subgroup (lower or higher intensity), patients who have measurable residual disease may be enrolled on their respective cohort at any time without maximum 'time from consolidation' requirement.
  • ECOG performance status of < or = 3
  • Adequate organ function as follows:
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  • Serum total bilirubin < or = to 1.5 X the Upper Limit of Normal (ULN)
  • Serum creatinine < or = to 2.5 x ULN
  • Adequate BM reserve:
  • Absolute neutrophil count (ANC) > 0.5 x k/uL
  • Platelet count > or = 30 x k/uL
  • For females of childbearing age, they may participate if they:
  • Have a negative serum or urine pregnancy test within 10 to 14 days of enrolling
  • Agree to either abstinence or 2 effective contraceptive methods (such as barrier methods or hormonal contraception) throughout the treatment period and up to 30 days after discontinuing treatment.
  • For male patients with a female partner of childbearing age, they may participate if they agree to either abstinence or 2 effective contraceptive methods throughout the treatment period and up to 30 days after discontinuing treatment.
  • Ability to understand and sign informed consent.

Who cannot join

  • Diagnosis of acute promyelocytic leukemia (APL), AML - M3 by FAB classification based on morphology, immunophenotype, molecular, or cytogenetic s studies.
  • Diagnosis of AML associated t(15;17) or APL variant. Patients with t(9;22) are also ineligible unless they are unable or unwilling to receive therapy with a tyrosine kinase inhibitor.
  • Uncontrolled intercurrent illness including, but not limited to active uncontrolled infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
  • Patients with active CNS (central nervous system) disease.
  • Patients with documented hypersensitivity to any components of the study program.
  • Females who are pregnant or lactating or intending to become pregnant during the study.
  • Patients with history of extramedullary AML, except for CNS involvement that is currently controlled, will not be eligible for enrollment.
  • Patient should be removed from current trial if they wish to participate and get treatment on another trial.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • M D Anderson Cancer Center

    Houston, Texas, 77030

Other trials in United States

About this study

Official title: Phase II Study of 5-Azacytidine (AZA) + Venetoclax as Maintenance Therapy in Patients With AML in Remission

This phase II trial studies how well azacitidine and venetoclax work in treating patients with acute myeloid leukemia that is in remission. Drugs used in chemotherapy, such as azacitidine and venetoclax, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.

Read the detailed description

PRIMARY OBJECTIVES:

  1. To assess relapse-free survival (RFS) of patients (pts) with acute myeloid leukemia (AML) treated with 5-azacytidine (azacitidine; AZA) combined with venetoclax (VEN) as maintenance therapy after achieving remission.

SECONDARY OBJECTIVES:

  1. To assess modified RFS of pts with AML treated with 5-azacytidine combined with venetoclax as maintenance therapy.

II. To assess overall survival (OS) of pts with AML treated with 5-azacytidine combined with venetoclax as maintenance therapy.

III. To assess event-free survival (EFS) of pts with AML treated with 5-azacytidine combined with venetoclax as maintenance therapy.

IV. To assess the duration of remission (CRd) of pts with AML treated 5-azacytidine combined with venetoclax as maintenance therapy.

  1. To assess toxicity and safety of 5-azacytidine combined with venetoclax as maintenance therapy in pts with AML.

VI. To assess the effects of 5-azacytidine combined with venetoclax on dynamics of minimal residual disease (MRD) and their relationship to outcomes.

OUTLINE:

Patients receive azacitidine subcutaneously (SC) or intravenously (IV) over 1 hour daily on days 1-5, and venetoclax orally (PO) daily on days 1-14. Treatment repeats every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up at 30 days and then every 6-12 months thereafter.

Being tested
Azacitidine, Venetoclax
Medical subject headings
Hematologic Diseases, Leukemia, Myeloid, Acute

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • AzacitidineDrug

    Also called 5 AZC, 5-AC, 5-Azacytidine, 5-AZC

    Given SC or IV

  • VenetoclaxDrug

    Also called ABT-0199, ABT-199, ABT199, GDC-0199

    Given PO

Study groups in NCT04062266
Study groupRoleWhat they receive
Treatment (azacytidine, venetoclax)ExperimentalPatients receive azacitidine SC or IV over 1 hour daily on days 1-5, and venetoclax PO daily on days 1-14. Treatment repeats every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity.
Receives: Drug: Azacitidine; Drug: Venetoclax

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Relapse-free survival (RFS)

    The study will be continuously monitored for the primary endpoint, RFS, using the method of Thall, Wooten, and Tannir (2005). Will also summarize the posterior distribution of lambda-E (i.e., median RFS) assuming posterior mean and 95% credible interval.

    Measured: From date of complete remission (CR) or complete remission with incomplete count recovery (CRi), assessed for up to 10 years

Secondary outcomes (5)

  1. Incidence of toxicity

    Toxicities are defined as any treatment -related clinically significant grade 3 or 4 non-hematologic adverse events occurred during cycles 1-2 of the trial. Will continuously monitor treatment-related toxicities using the Bayesian approach of Thall, Simon, Estey (1995).

    Measured: Up to 10 years

  2. Modified RFS

    Distribution assessed using Kaplan-Meier method.

    Measured: Time from enrollment on study until date of first objective documentation of relapse or death, assessed for up to 10 years

  3. Overall survival (OS)

    Distribution assessed using Kaplan-Meier method.

    Measured: From the start of study treatment until date of death due to any cause, assessed for up to 10 years

  4. Event free survival (EFS)

    Distribution assessed using Kaplan-Meier method.

    Measured: From the start of study treatment until date of first confirmed relapse, withdrawal from study due to adverse event, or death due to any cause, assessed for up to 10 years

  5. Complete remission duration (CRd)

    Distribution assessed using Kaplan-Meier method.

    Measured: Time from CR or CRi until date of first objective documentation of confirmed relapse, assessed for up to 10 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 Aug 2019
  2. Study start13 Sep 2019
  3. Primary completion12 Oct 2026
  4. Study completion12 Oct 2026

Why it stopped: \<75% participation

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
36 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04062266
Sponsor's protocol ID
2019-0226
Other IDs
NCI-2019-04987, 2019-0226
Status last verified
Oct 2026
First submitted
16 Aug 2019
Last updated
16 Oct 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Bazinet A, Kantarjian H, Bataller A, Pemmaraju N, Borthakur G, Chien K, Alvarado Y, Bose P, Jabbour E, Yilmaz M, DiNardo C, Issa G, Montalban-Bravo G, Short N, Sasaki K, Bull-Linderman D, Daver N, Garcia-Manero G, Ravandi F, Kadia T. Reduced dose azacitidine plus venetoclax as maintenance therapy in acute myeloid leukaemia following intensive or low-intensity induction: a single-centre, single-arm, phase 2 trial. Lancet Haematol. 2024 Apr;11(4):e287-e298. doi: 10.1016/S2352-3026(24)00034-6.

    PubMed 38548404

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