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

July 22, 2024 updated by: French Innovative Leukemia Organisation

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

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.

Study Overview

Status

Recruiting

Conditions

Study Type

Observational

Enrollment (Estimated)

230

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Probability Sample

Study 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

Description

Inclusion Criteria:

  • Adult patients
  • treatment with VEN-AZA for newly diagnosed AML and ineligible to intensive chemotherapy
  • Treatment in the named-patients program (ATU)

Exclusion Criteria:

  • 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
  • Opposition to data collection

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
characterize the overall response rate, in patients treated by Venetoclax/Azacitidine (VEN/AZA), in real life cohort
Time Frame: 18 months (since Cycle 1 Day 1)
  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
18 months (since Cycle 1 Day 1)
characterize the overal survival (OS) in patients treated by Venetoclax/Azacitidine (VEN/AZA), in real life cohort
Time Frame: 18 months (since Cycle 1 Day 1)
rate of OS according to the 2022 European LeukemiaNet (ELN)
18 months (since Cycle 1 Day 1)
characterize the event free survival (EFS) in patients treated by Venetoclax/Azacitidine (VEN/AZA), in real life cohort
Time Frame: 18 months (since Cycle 1 Day 1)
rate of EFS according to the 2022 European LeukemiaNet (ELN)
18 months (since Cycle 1 Day 1)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
characterize the incidence and relatedness of serious adverse events (SAE), in patients treated by Venetoclax/Azacitidine (VEN/AZA), in real life
Time Frame: 18 months (since Cycle 1 Day 1)
description of grade 3/4 SAE and death according to CTCAE v5.
18 months (since Cycle 1 Day 1)
describe the management of Venetoclax/Azacitidine (VEN/AZA) in a real -life cohort of patient.
Time Frame: 18 months (since Cycle 1 Day 1)
daily dose and duration of Venetoclax and Azacitidine description of Venetoclax and Azacitidine dose modification description of Venetoclax and Azacitidine duration
18 months (since Cycle 1 Day 1)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

French Innovative Leukemia Organisation

Collaborators

Acute Leukemia French Association

Investigators

  • Principal Investigator: Sylvain GARCIAZ, MD-PhD, French Innovative Leukemia Organisation

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 4, 2024

Primary Completion (Estimated)

December 31, 2024

Study Completion (Estimated)

March 30, 2025

Study Registration Dates

First Submitted

December 14, 2023

First Submitted That Met QC Criteria

February 15, 2024

First Posted (Actual)

February 16, 2024

Study Record Updates

Last Update Posted (Actual)

July 23, 2024

Last Update Submitted That Met QC Criteria

July 22, 2024

Last Verified

July 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • FILObsLAM_VENAZA

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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