De-Escalation Study Evaluating Venetoclax and Azacitidine Discontinuation in AML Responding Patients (STOP VEN)

Institut Paoli-Calmettes

Status and phase: Enrolling; Phase 2

Conditions: Acute Myeloid Leukemia

Treatments: Drug: Azacitidine; Drug: Venetoclax

Study type: Interventional

Funder types: Other

Identifiers

NCT06557421

STOP VEN-IPC 2024-001

Details and patient eligibility

About

The goal of this clinical trial is to test efficacy and safety of a VENETOCLAX-AZACITIDINE (VEN-AZA) de-escalation strategy in Acute Myeloid Leukemia responding patients. The main objectives of the study are:

• Evaluation of the efficacy of VEN-AZA de-escalation strategy by measuring the effect of VEN-AZA discontinuation in term of Disease-Free Survival.
• Evaluation of the other efficacy parameters and safety of VEN-AZA de-escalation strategy.

Patients from the prospective study will be compared to a retrospective cohort of patients who will be selected on the basis of identical eligibility criteria.

Participants will:

• Stop VEN-DASA treatment
• Be closely monitored by regular evaluation of the disease

Enrollment: 50 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria:

1. Female/Male ≥ 18 years of age;
2. Diagnosis of previously untreated AML according to the 2022 International Consensus Classification of Myeloid Neoplasms and Acute Leukemias;
3. VEN-AZA given as first-line treatment;
4. Duration of VEN-AZA therapy of 12 months (+/- 28 days), regardless of duration of VEN-AZA cycles and the doses;
5. Patients in first composite complete remission (CRc) defined as complete remission (CR) or CR with incomplete hematologic recovery (CRi) or CR with partial hematologic recovery (CRh);
6. Absence of detectable minimal residual disease (MRD) performed locally (i.e. MRDneg defined as MCF MRD <0.1% of CD45 expressing cells with the target immunophenotype in bone marrow, or NPM1 or RUNX1-RUNX1T1 or CBFB-MYH11 MRD copy numbers <0.1% in the blood);
7. ECOG <3;
8. Absence of any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule;
9. Affiliated to the French Social Security or beneficiary of such a health Insurance;
10. Signed informed consent.

Non inclusion Criteria:

1. VEN-AZA given as salvage therapy;
2. Prior allogeneic stem cell transplant;
3. Discontinuation of treatment because of absence or loss of response;
4. Patient in emergency situation or unable to give consent;
5. Severe medical or mental condition precluding the follow up procedures after treatment discontinuation.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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50 participants in 1 patient group

VEN-AZA de-escalation

Experimental group

Description:

VEN-AZA de-escalation

Treatment:

Drug: Venetoclax

Drug: Azacitidine