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
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
1 other identifier
observational
230
1 country
42
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Jul 2024
Shorter than P25 for all trials
42 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
December 14, 2023
CompletedFirst Posted
Study publicly available on registry
February 16, 2024
CompletedStudy Start
First participant enrolled
July 4, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 31, 2024
CompletedStudy Completion
Last participant's last visit for all outcomes
March 30, 2025
CompletedJuly 23, 2024
July 1, 2024
6 months
December 14, 2023
July 22, 2024
Conditions
Outcome Measures
Primary Outcomes (3)
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
18 months (since Cycle 1 Day 1)
characterize the overal survival (OS) in patients treated by Venetoclax/Azacitidine (VEN/AZA), in real life cohort
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
rate of EFS according to the 2022 European LeukemiaNet (ELN)
18 months (since Cycle 1 Day 1)
Secondary Outcomes (2)
characterize the incidence and relatedness of serious adverse events (SAE), in patients treated by Venetoclax/Azacitidine (VEN/AZA), in real life
18 months (since Cycle 1 Day 1)
describe the management of Venetoclax/Azacitidine (VEN/AZA) in a real -life cohort of patient.
18 months (since Cycle 1 Day 1)
Eligibility Criteria
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
You may qualify if:
- Adult patients
- treatment with VEN-AZA for newly diagnosed AML and ineligible to intensive chemotherapy
- Treatment in the named-patients program (ATU)
You may not qualify if:
- Treatment with VEN-AZA for previously treated AML
- Opposition to data collection
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (42)
Amiens CHU
Amiens, France
Angers CHU
Angers, France
Avignon CH
Avignon, France
Bayonne CH
Bayonne, France
Besançon CHU
Besançon, France
Brest CHU
Brest, France
Caen CHU
Caen, France
CERGY PONTOISE - CH René Dubos
Cergy-Pontoise, France
Hôpital d'Instruction des Armées PERCY
Clamart, France
CHU Estaing
Clermont-Ferrand, France
Corbeil-Essonnes - Ch Sud Francilien
Corbeil-Essonnes, France
Créteil CHU HENRI MONDOR
Créteil, France
Dijon CHU
Dijon, France
Grenoble CHU
Grenoble, France
Le Mans CH
Le Mans, France
CHU Lille
Lille, France
Limoges CHU
Limoges, France
Lyon sud CHU
Lyon, France
Marseille IPC
Marseille, France
Meaux CH de l'Est francilien
Meaux, France
METZ-THIONVILLE CHR- Hôpital de Mercy
Metz, France
Montpellier - Chu Saint Eloi
Montpellier, France
Nantes CHU
Nantes, France
Nice CHU
Nice, France
Nimes CHU
Nîmes, France
Paris La Pitié salpetrière
Paris, France
Paris Necker
Paris, France
Paris Saint Louis
Paris, France
Bordeaux CHU
Pessac, France
Reims CHU
Reims, France
Rennes CHU
Rennes, France
roubaix CH
Roubaix, France
Centre de Lutte Contre le Cancer H. Becquerel
Rouen, France
Institut de Cancérologie Lucien Neuwirth
Saint-Priest-en-Jarez, France
Saint Quentin CH
Saint-Quentin, France
ICANS - Institut de cancérologie de strasbourg europe
Strasbourg, France
Toulouse - IUCT Oncopole - Service d'Hématologie
Toulouse, France
Tours CHU
Tours, France
Troyes CH
Troyes, France
Nancy CHU
Vandœuvre-lès-Nancy, France
Versailles CH
Versailles, France
Villejuif IGR
Villejuif, France
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Sylvain GARCIAZ, MD-PhD
French Innovative Leukemia Organisation
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- CROSS SECTIONAL
- Target Duration
- 18 Months
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 14, 2023
First Posted
February 16, 2024
Study Start
July 4, 2024
Primary Completion
December 31, 2024
Study Completion
March 30, 2025
Last Updated
July 23, 2024
Record last verified: 2024-07
Data Sharing
- IPD Sharing
- Will not share