Results From a French Temporary Utilization Authorization of First-line Acute Myeloid Leukemia (AML) Patients Ineligible for Intensive Chemotherapy (IC), Treated With Venetoclax Azacitidine

ConditionAML, Adult

Trial statusRecruiting

Trial phaseNot listed

Trial typeObservational

Biological sexAll

Age18+

SponsorFrench Innovative Leukemia Organisation

About this trial

Following the results of the phase 1b and the phase 3 studies, Venetoclax/Azacitidine (VEN/AZA) was available in France for newly diagnosed AML patients ineligible-IC patients through the early access program the so-called ATU program.

Venetoclax (VEN) has been available in France through the ATU since Feb 2021 and through the current post-ATU schema from the point of marketing authorization approval and up to the pending publication of reimbursement and price. Between February 15, 2021, and June 30, 2021, 285 requests for ATU were made to the pharmaceutical company (Abbvie) and led to the initiation of treatment of more than 230 patients. At the end of ATU period, all these 230 ATU patients continued to be treated by VEN/AZA as part of the current post-ATU period. Healthcare professionals and health care decision makers need real world data to better understand the benefit/risk profile of treatment. Early access to treatment in France is close to real-life setting condition.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Adult patients

treatment with VEN-AZA for newly diagnosed AML and ineligible to intensive chemotherapy

Treatment in the named-patients program (ATU)

Disqualifiers

Treatment with VEN-AZA for previously treated AML

Prior treatment for preexisting hematological malignancies other that AML, including AZA is not an exclusion criteria

HYDROXYCARBAMIDE given for AML is not an exclusion criteria

AZA started before VEN for AML is not an exclusion criteria

Trial population

Adult Patients with de novo AML, ineligible for intensive chemotherapy, which received VENAZA in the ATU program between 02/15/2021 and 06/30/2021

Trial design

Design model

Cohort

Time perspective

Cross-sectional

Treatments tested in this trial

Not listed

Trial groups

No trial groups listed

Trial outcomes

Primary outcomes

characterize the overall response rate, in patients treated by Venetoclax/Azacitidine (VEN/AZA), in real life cohort

1. Rate of complete remission and complete remission with incomplete hematologic recovery (CR/CRi), 2. Rate of morphological leukemia free state (MLFS) 3. Rate of CR/CRi rate with negative minimal residual disease (MRD) assessed by flow cytometry (FCT) and/or molecular techniques if available

Time frame