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

Recruiting

• Conditions: AML, Adult

• Registration Number: NCT06263387
• Lead Sponsor: French Innovative Leukemia Organisation

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-12-14
• Study First Submitted QC: 2024-02-15
• Study First Posted: 2024-02-16
• Status Verified: 2024-07
• Study Start: 2024-07-04
• Last Update Submitted: 2024-07-22
• Last Update Posted: 2024-07-23
• Primary Completion: 2024-12-31
• Study Completion: 2025-03-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 230

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
describe the management of Venetoclax/Azacitidine (VEN/AZA) in a real -life cohort of patient.	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
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)	description of grade 3/4 SAE and death according to CTCAE v5.

Trial Locations

Locations (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

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