Clinical trial NCT06263387: Results From a French Temporary Utilization Authorization of First-line Acute Myeloid Leukemia (AML) Patients Ineligible for Intensive Chemotherapy (IC), Treated With Venetoclax Azacitidine
Official title: 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
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: AML, Adult
Lead sponsor: French Innovative Leukemia Organisation
Sponsor class: OTHER
Collaborators: name: Acute Leukemia French Association
First posted: 2024-02-16
Enrollment: 230
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
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

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.


Eligibility criteria: 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

Primary outcomes:
measure: characterize the overall response rate, in patients treated by Venetoclax/Azacitidine (VEN/AZA), in real life cohort
timeFrame: 18 months (since Cycle 1 Day 1)
measure: characterize the overal survival (OS) in patients treated by Venetoclax/Azacitidine (VEN/AZA), in real life cohort
timeFrame: 18 months (since Cycle 1 Day 1)
measure: characterize the event free survival (EFS) in patients treated by Venetoclax/Azacitidine (VEN/AZA), in real life cohort
timeFrame: 18 months (since Cycle 1 Day 1)
Secondary outcomes:
measure: characterize the incidence and relatedness of serious adverse events (SAE), in patients treated by Venetoclax/Azacitidine (VEN/AZA), in real life
timeFrame: 18 months (since Cycle 1 Day 1)
measure: describe the management of Venetoclax/Azacitidine (VEN/AZA) in a real -life cohort of patient.
timeFrame: 18 months (since Cycle 1 Day 1)
Other outcomes:
Locations:
city: Amiens
country: France
facility: Amiens CHU
city: Angers
country: France
facility: Angers CHU
city: Avignon
country: France
facility: Avignon CH
city: Bayonne
country: France
facility: Bayonne CH
city: Besançon
country: France
facility: Besançon CHU
city: Brest
country: France
facility: Brest CHU
city: Caen
country: France
facility: Caen CHU
city: Cergy-Pontoise
country: France
facility: CERGY PONTOISE - CH René Dubos
city: Clamart
country: France
facility: Hôpital d'Instruction des Armées PERCY
city: Clermont-Ferrand
country: France
facility: CHU Estaing
city: Corbeil-Essonnes
country: France
facility: Corbeil-Essonnes - Ch Sud Francilien
city: Créteil
country: France
facility: Créteil CHU HENRI MONDOR
city: Dijon
country: France
facility: Dijon CHU
city: Grenoble
country: France
facility: Grenoble CHU
city: Le Mans
country: France
facility: Le Mans CH
city: Lille
country: France
facility: CHU Lille
city: Limoges
country: France
facility: Limoges CHU
city: Lyon
country: France
facility: Lyon sud CHU
city: Marseille
country: France
facility: Marseille IPC
city: Meaux
country: France
facility: Meaux CH de l'Est francilien
city: Metz
country: France
facility: METZ-THIONVILLE CHR- Hôpital de Mercy
city: Montpellier
country: France
facility: Montpellier - Chu Saint Eloi
city: Nantes
country: France
facility: Nantes CHU
city: Nice
country: France
facility: Nice CHU
city: Nîmes
country: France
facility: Nimes CHU
city: Paris
country: France
facility: Paris La Pitié salpetrière
city: Paris
country: France
facility: Paris Necker
city: Paris
country: France
facility: Paris Saint Louis
city: Pessac
country: France
facility: Bordeaux CHU
city: Reims
country: France
facility: Reims CHU
city: Rennes
country: France
facility: Rennes CHU
city: Roubaix
country: France
facility: roubaix CH
city: Rouen
country: France
facility: Centre de Lutte Contre le Cancer H. Becquerel
city: Saint-Priest-en-Jarez
country: France
facility: Institut de Cancérologie Lucien Neuwirth
city: Saint-Quentin
country: France
facility: Saint Quentin CH
city: Strasbourg
country: France
facility: ICANS - Institut de cancérologie de strasbourg europe
city: Toulouse
country: France
facility: Toulouse - IUCT Oncopole - Service d'Hématologie
city: Tours
country: France
facility: Tours CHU
city: Troyes
country: France
facility: Troyes CH
city: vandoeuvre les Nancy
country: France
facility: Nancy CHU
city: Versailles
country: France
facility: Versailles CH
city: Villejuif
country: France
facility: Villejuif IGR
Study officials:
affiliation: French Innovative Leukemia Organisation
name: Sylvain GARCIAZ, MD-PhD
role: PRINCIPAL_INVESTIGATOR
References: