Clinical trial · Observational
Results From a French Temporary Utilization Authorization of First-line Acute Myeloid Leukemia (AML) Patients Ineligible for Intensive Chemotherapy (IC), Treated With Venetoclax Azacitidine
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
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| AML, Adult | Adult Acute Myeloid Leukemia | ALIAS | 0.90 |
Interventions
Interventions (0)
Data not yet available
Design
Arms and outcomes
Arms (0)
[]Primary outcomes (3)
- 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)
- description
- 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
- 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)
- description
- rate of OS according to the 2022 European LeukemiaNet (ELN)
- 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)
- description
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
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
References
Publications (0)
Data not yet available