Venetoclax + Azacitidine in Patients With Acute Myeloid Leukemia

NCT ID: NCT06668558

Last Updated: 2025-09-19

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

29 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-12-16

Study Completion Date

2028-07-31

Brief Summary

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The VERDI study is an investigator-initiated, multicenter, multicohort, phase II trial with combination of venetoclax + azacitidine for patients treated for AML under according to an intensive chemotherapy protocol (CETLAM-20) failing to achieve or maintain MRD negativity at pre-established time-points: at chemotherapy completion for ELN favorable subtypes, and prior to alloHCT for non-favorable European LeukemiaNet (ELN) AML patients.

The primary objective is to determine Ven/Aza treatment activity in MRD clearance in patients diagnosed with AML with persistent MRD or MRD reappearance after frontline chemotherapy, or prior to alloHCT.

Detailed Description

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The trial will enroll competitively between 25 and 29 patients.

Patients will be recruited in two independent cohorts depending on the pre-established time point for the intervention and ELN risk subtype:

Cohort 1: Patients diagnosed with a favorable ELN subtype AML, not intended for alloHCT in first complete remission (CR1), but who present an MRD failure by after frontline intensive chemotherapy, as defined in the 2021 update on MRD guidelines elaborated by the European LeukemiaNet MRD Working Party (Heuser et al. 2023):

In patients with persistent low-level MRD (\<2%) after consolidation chemotherapy, an increase of MRD ≥1log10 between 2 positive samples Confirmed MRD conversion of MRD negativity to MRD positivity AML during subsequent follow-up (up to 3 years after chemotherapy completion

Cohort 2: Patients diagnosed with a non-favorable ELN AML subtype, intended to undergo alloHCT, in first complete morphological remission but harboring detectable MRD at time of alloHCT (\>0.1%).

Conditions

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Acute Myeloid Leukemia

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

investigator-initiated, multicenter, multicohort, phase II trial
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1

Patients diagnosed with a favorable ELN subtype AML, not intended for alloHCT in CR1, but who present an MRD failure by after frontline intensive chemotherapy, as defined in the 2021 update on MRD guidelines elaborated by the European LeukemiaNet MRD Working Party (Heuser et al. 2021)

Group Type EXPERIMENTAL

Azacitidine (AZA)

Intervention Type DRUG

Dosing is 75 mg/m2 x 7 days (Q28days) After 2 courses, a first decision-making for allo-HCT based on bone marrow MRD assessment will be performed. After 4th course, a new MRD assessment will be performed.

For cohort 1 treatment may continue up to cycle 12 (prioritized over allo-HCT). For cohort 2 treatment may continue up to cycle 24 (prioritizing allo-HCT)

Venetoclax

Intervention Type DRUG

Dosing: 400 mg daily After 2 courses, a first decision-making for allo-HCT based on bone marrow MRD assessment will be performed. After 4th course, a new MRD assessment will be performed.

For cohort 1 treatment may continue up to cycle 12 (prioritized over allo-HCT). For cohort 2 treatment may continue up to cycle 24 (prioritizing allo-HCT)

Cohort 2

Patients diagnosed with a non-favorable ELN AML subtype, intended to undergo alloHCT, in first complete morphological remission but harboring detectable MRD at time of alloHCT (\>0.1%)

Group Type EXPERIMENTAL

Azacitidine (AZA)

Intervention Type DRUG

Dosing is 75 mg/m2 x 7 days (Q28days) After 2 courses, a first decision-making for allo-HCT based on bone marrow MRD assessment will be performed. After 4th course, a new MRD assessment will be performed.

For cohort 1 treatment may continue up to cycle 12 (prioritized over allo-HCT). For cohort 2 treatment may continue up to cycle 24 (prioritizing allo-HCT)

Venetoclax

Intervention Type DRUG

Dosing: 400 mg daily After 2 courses, a first decision-making for allo-HCT based on bone marrow MRD assessment will be performed. After 4th course, a new MRD assessment will be performed.

For cohort 1 treatment may continue up to cycle 12 (prioritized over allo-HCT). For cohort 2 treatment may continue up to cycle 24 (prioritizing allo-HCT)

Interventions

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Azacitidine (AZA)

Dosing is 75 mg/m2 x 7 days (Q28days) After 2 courses, a first decision-making for allo-HCT based on bone marrow MRD assessment will be performed. After 4th course, a new MRD assessment will be performed.

For cohort 1 treatment may continue up to cycle 12 (prioritized over allo-HCT). For cohort 2 treatment may continue up to cycle 24 (prioritizing allo-HCT)

Intervention Type DRUG

Venetoclax

Dosing: 400 mg daily After 2 courses, a first decision-making for allo-HCT based on bone marrow MRD assessment will be performed. After 4th course, a new MRD assessment will be performed.

For cohort 1 treatment may continue up to cycle 12 (prioritized over allo-HCT). For cohort 2 treatment may continue up to cycle 24 (prioritizing allo-HCT)

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Patients must have confirmation of with acute myeloid leukemia (AML) with persistent measurable residual disease (MRD) or MRD reappearance after frontline intensive chemotherapy (including at least one cycle of cytarabine and anthracycline), and prior to allogeneic hematopoietic cell transplantation (allo-HCT).

1. In patients with NPM1 mutation, qRT-PCR of NPM1 will be the method used to establish a molecular failure, defined as failure to achieve molecular response after consolidation therapy (NPM1mut/ABL1·100 \> 0.1) or MRD reappearance after molecular response. All cases of molecular failure must be confirmed with a second MRD assessment in 2 to 4 weeks.
2. In patients with core-binding factor AML, qRT-BCR of RUNX1-RUNX1T1 and CBFb-MYH11 transcripts will be used. Patients failing to achieve a major MRD reduction after consolidation therapy (i.e., RUNX1-RUNX1T1/ABL1·100\>0.1 or CBFb-MYH11/ABL1·100\>0.1), a log increase in MRD between two positive samples or confirmed MRD reappearance after molecular response will be considered as molecular failures and could be included in the trial.
3. In the remaining cases, an appropriate leukemia-associated immunophenotype (LAIP) measured by multiparameter flow cytometry will be used for MRD surveillance. A cutoff of 0.1% will be used to define MRD positivity.
2. Age ≥18 years.
3. Without clinical signs of active central nervous system disease.
4. Patients must have an Eastern Cooperative Oncology Group (ECOG) Performance status of ≤2 or Karnofsky performance status (KPS) equivalent.
5. Patients must have adequate renal function as demonstrated by a calculated creatinine clearance ≥ 30 mL/min; determined via urine collection for 24-hour creatinine clearance or by the Cockcroft Gault formula.
6. Patients must have adequate liver function as demonstrated by:

1. aspartate aminotransferase (AST) ≤ 3.0 × upper limit normal (ULN)
2. alanine aminotransferase (ALT) ≤ 3.0 × ULN
3. bilirubin ≤ 1.5 × ULN, unless due to Gilbert\'s syndrome
7. Non-sterile male patients must use contraceptive methods with partner(s) prior to beginning study drug administration and continuing up to 3 months after the last dose of study drug. Male patients must agree to refrain from sperm donation from initial study drug administration until 3 months after the last dose of study drug.
8. WOCBP must agree to use two reliable forms of contraception simultaneously or to practice complete abstinence from heterosexual intercourse during the following time periods related to this study: 1) for at least 28 days before starting therapy; 2) throughout the entire duration of treatment; 3) during dose interruptions; and 4) for at least 6 months after discontinuation of therapy (last dose of study drug).
9. Patients must voluntarily sign and date an informed consent, approved by an Institutional Review Board (IRB), prior to the initiation of any research directed screening procedures.

Exclusion Criteria

1. Patient has received other prior rescue treatment for MRD.
2. Patient is known to be positive for Human immunodeficiency virus (HIV) infection with the exception of those with an undetectable viral load under correct virological control throughout the study.

Note: HIV testing is not required.
3. Patient is known to be positive for hepatitis B (HBV) or C (HCV) infection with the exception of those with an undetectable viral load.

Note: Hepatitis B or C testing is not required and patients with serologic evidence of prior vaccination to HBV (i.e., HBsAg-, anti-HBs+ and anti-HBc-) may participate.
4. Patient has known active central nervous system (CNS) involvement from AML.
5. Patient has received within 7 days prior to the first dose of study drug: steroid therapy ≥ 20 mg/day (prednisone or equivalent) for antineoplastic intent; strong and moderate CYP3A inhibitors; strong and moderate CYP3A inducers.
6. Patient has consumed grapefruit, grapefruit products, Seville oranges (including marmalade containing Seville oranges) or Star fruit within 3 days prior to the initiation of study treatment.
7. Patient has any history of clinically significant condition(s) that in the opinion of the investigator would adversely affect his/her participating in this study including, but not limited to:

1. New York Heart Association heart failure \> class 2.
2. Renal, neurologic, psychiatric, endocrinologic, metabolic, immunologic, hepatic, cardiovascular disease, or bleeding disorder independent of leukemia.
8. Patient has a malabsorption syndrome or other condition that precludes the enteral route of administration.
9. Patient exhibits evidence of uncontrolled systemic infection requiring therapy (viral, bacterial or fungal).
10. Patient has a history of other malignancies within the prior year to study entry, except for:

1. Adequately treated in situ carcinoma of the breast or cervix uteri.
2. Basal cell carcinoma of the skin or localized squamous cell carcinoma of the skin.
3. Prostate cancer with no plans for therapy of any kind.
4. Previous malignancy confined and surgically resected (or treated with other modalities) with curative intent.
11. Pregnant and breastfeeding females.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Grupo Cooperativo de Estudio y Tratamiento de las Leucemias Agudas y Mielodisplasias

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Jordi Esteve, M.D.; Ph.D.

Role: STUDY_CHAIR

Hematology department Institute Clínic of Hematological and Oncological diseases (ICMHO) Hospital Clínic of Barcelona

Locations

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University Hospital Son Espases

Palma de Mallorca, Balearic Islands, Spain

Site Status RECRUITING

Hospital Son Llatzer

Palma de Mallorca, Balearic Islands, Spain

Site Status RECRUITING

Institut Catala D oncologia Badalona

Badalona, Catalonia, Spain

Site Status RECRUITING

Hospital Del Mar

Barcelona, Catalonia, Spain

Site Status RECRUITING

Hospital De La Santa Creu I Sant Pau

Barcelona, Catalonia, Spain

Site Status RECRUITING

Hospital Universitari Vall D Hebron

Barcelona, Catalonia, Spain

Site Status RECRUITING

Hospital Clinic De Barcelona

Barcelona, Catalonia, Spain

Site Status RECRUITING

Institut Catala D oncologia Girona

Girona, Catalonia, Spain

Site Status RECRUITING

Institut Catala D oncologia Hospitalet

L'Hospitalet de Llobregat, Catalonia, Spain

Site Status RECRUITING

Hospital Universitari Arnau De Vilanova De La Gerencia Territorial De Lleida

Lleida, Catalonia, Spain

Site Status RECRUITING

Hospital Universitari Joan XXIII De Tarragona

Tarragona, Catalonia, Spain

Site Status RECRUITING

Fundacio Assistencial De Mutua De Terrassa

Terrassa, Catalonia, Spain

Site Status RECRUITING

Hospital General Universitario Gregorio Maranon

Madrid, Madrid, Spain

Site Status RECRUITING

Hospital Clinico Universitario De Valencia

Valencia, Valencia, Spain

Site Status RECRUITING

Countries

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Spain

Central Contacts

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A responsible person Designated by the sponsor

Role: CONTACT

0034934344412

Facility Contacts

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A responsible person Designated by the Sponsor

Role: primary

0034934344412

A responsible person Designated by the Sponsor

Role: primary

0034934344412

A responsible person Designated by the Sponsor

Role: primary

0034934344412

A responsible person Designated by the Sponsor

Role: primary

0034934344412

A responsible person Designated by the Sponsor

Role: primary

0034934344412

A responsible person Designated by the Sponsor

Role: primary

0034934344412

A responsible person Designated by the Sponsor

Role: primary

0034934344412

A responsible person Designated by the Sponsor

Role: primary

0034934344412

A responsible person Designated by the Sponsor

Role: primary

0034934344412

A responsible person Designated by the Sponsor

Role: primary

0034934344412

A responsible person Designated by the Sponsor

Role: primary

0034934344412

A responsible person Designated by the Sponsor

Role: primary

0034934344412

A responsible person Designated by the Sponsor

Role: primary

0034934344412

A responsible person Designated by the Sponsor

Role: primary

0034934344412

Other Identifiers

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2024-510648-29-00

Identifier Type: CTIS

Identifier Source: secondary_id

CETLAM-LMA-AC-2024-01

Identifier Type: -

Identifier Source: org_study_id

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