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

AML, Adult Clinical Trial

— VENAZA  

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06263387
• Other study ID #: FILObsLAM_VENAZA
• Status: Not yet recruiting
• Start date: June 20, 2024
• Est. completion date: March 30, 2025

Study information

• Verified date: June 2024
• Source: French Innovative Leukemia Organisation
• Contact: Ariane MINEUR
• Phone: +33 (0)5 57 62 31 08
• Email: ariane.mineur[@]chu-bordeaux.fr
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 230
• Est. completion date: March 30, 2025
• Est. primary completion date: December 31, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• AML, Adult
• Leukemia, Myeloid, Acute

Locations

Country	Name	City	State
France	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	vandoeuvre les Nancy
France	Versailles CH	Versailles
France	Villejuif IGR	Villejuif

Sponsors (2)

Lead Sponsor	Collaborator
French Innovative Leukemia Organisation	Acute Leukemia French Association

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	characterize the overall response rate, in patients treated by Venetoclax/Azacitidine (VEN/AZA), in real life cohort	Rate of complete remission and complete remission with incomplete hematologic recovery (CR/CRi),; Rate of morphological leukemia free state (MLFS); 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)
Primary	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)
Primary	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	characterize the incidence and relatedness of serious adverse events (SAE), in patients treated by Venetoclax/Azacitidine (VEN/AZA), in real life	description of grade 3/4 SAE and death according to CTCAE v5.	18 months (since Cycle 1 Day 1)
Secondary	describe the management of Venetoclax/Azacitidine (VEN/AZA) in a real -life cohort of patient.	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)