OBServatory of Compassionate Use of IVOsidenib in France for Patients With Acute Myeloid Leukemia
1 other identifier
observational
250
1 country
21
Brief Summary
Mutations in IDH genes are found in numerous cancers and more specifically in acute myeloid leukemia (AML). These mutations target specific amino acids, at positions 140 or 172 of IDH2, and 132 of IDH1. Mutant IDH proteins acquire an abnormal enzymatic activity allowing them to convert α-ketoglutarate (αKG) into D-2 hydroxyglutarate (D-2HG), an oncometabolite which massively accumulates in IDH-mutated cells. At high levels, D-2HG behaves as a competitive inhibitor of αKG and affects the activity of Fe(II)/αKG-dependent dioxygenases. This enzymatic family is involved in a broad spectrum of pathways such as demethylation of histone (JHDM histone demethylases) or DNA (methylcytosine hydroxylases of the TET family). As a result, IDH-mutated cells show altered survival, motility, invasiveness and cell differentiation. In AML, IDH1 mutations might be present in 10-15% at diagnosis Ivosidenib (IVO) a first-in-class, oral, irreversible inhibitor of mutant IDH1 has shown clinical activity as a single agent in studies involving patients with IDH1 mutated relapsed or refractory (R/R) AML and in front line settings. In phase II clinical trials, IVO yielded 30-35% of complete response rates both in frontline and R/R settings, with long lasting responses. Based on these results, the FDA (Food and Drug Agency) gave its approval for newly-diagnosed AML IDH1mut patients who are ≥ 75 years old or who have comorbidities and in R/R. However, European Medicines Agency (EMA)'s did not approved IVO due to lack of evidences to support the application. Agios Netherlands B.V. (the company that previously own the drug before Servier Laboratories) withdrew its EMA application. Nevertheless, IVO has been available in France through a compassionate use program (CUP), since February 2020 for R/R patients and March 2022 for first line treatment. In this multicentric retrospective study, sponsor aim to evaluate the efficacy and safety of Ivo in two cohorts of IDH1mut AML patients treated within the CUP. The first cohort will concern patients treated in first line setting and the second cohort those treated in R/R disease. Results might provide new insights regarding IVO in real life settings and support signs of efficacy. This could provide new data for the haematologist community and for another appliance to grant EMA approval of IVO in the setting of R/R IDH1mut AML.
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
21 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
April 17, 2024
CompletedFirst Posted
Study publicly available on registry
April 22, 2024
CompletedStudy Start
First participant enrolled
July 31, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 30, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
June 1, 2026
ExpectedNovember 26, 2025
November 1, 2025
1.1 years
April 17, 2024
November 25, 2025
Conditions
Outcome Measures
Primary Outcomes (1)
characterize the Overall survival (OS) in the both cohort : 1st line and Relapsed/Refractory (R/R)
defined as the time from date of initiation of Ivosidebib to date of death due to any cause. Patients still alive or lost to follow up will be censored at the time they were last known to be alive
6 months
Secondary Outcomes (5)
characterize the composite response rate (CRc) at any time during follow-up, for the both cohort : 1st line and Relapsed/Refractory (R/R)
6 months
characterize the Event Free Survival (EFS) in both cohorts : 1st line and Relapsed/Refractory (R/R)
6 months
characterize the incidence and relatedness of serious adverse events (SAE), for patients treated by Ivosidenib, for both cohorts : 1st line and Relapsed/Refractory (R/R)
6 months
describe the management of treatment by Ivosidenib in both cohorts : 1st line and Relapsed/Refractory (R/R)
6 months
describe the management of treatment by Ivosidenib in both cohorts : 1st line and Relapsed/Refractory (R/R)
6 months
Study Arms (2)
AML in 1st line at inclusion
AML in R/R at inclusion
Eligibility Criteria
Patients of 18 years old or more, with newly diagnosed or relapsed or refractory IDH1 mutated AML according to ELN 2022 and treated with IVO thanks to the CUP between the period 01/01/2017 to 01/08/2023, treated in French AML FILO or ALFA centers
You may qualify if:
- Patient with IDH1 R132 mutated with newly diagnosed or Relapsed or Refractory (R/R) acute myeloid leukemia
- Patient treated within French compassionate access program that have started the treatment between 01/01/2017 to 01/08/2023
- patient treated by Ivosidenib received either as a monotherapy or in combination with other AML therapy (i.e. azacytidine, venetoclax)
- Patient not included within IDH inhibitor clinical trial.
You may not qualify if:
- Patients who expressed their opposition to entered in the study
- Patients who received IVO through a trial
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (21)
Amiens CHU
Amiens, France
Angers CHU
Angers, France
Bayonne CH
Bayonne, France
Besançon CHU
Besançon, France
CHU Estaing
Clermont-Ferrand, France
Créteil CHU HENRI MONDOR
Créteil, France
DUNKERQUE-Hôpital Alexandra Lepève
Dunkirk, 59385, France
Grenoble CHU
Grenoble, France
Le Mans CH
Le Mans, France
Lyon sud CHU
Lyon, France
Marseille IPC
Marseille, France
Meaux CH de l'Est francilien
Meaux, France
Montpellier - Chu Saint Eloi
Montpellier, France
Mulhouse Chu
Mulhouse, 68100, France
Nantes CHU
Nantes, France
Nice CHU
Nice, France
Orléans CHU
Orléans, 45000, France
Paris Saint Louis
Paris, France
Bordeaux CHU
Pessac, France
ICANS - Institut de cancérologie de strasbourg europe
Strasbourg, France
Toulouse - IUCT Oncopole - Service d'Hématologie
Toulouse, France
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Pierre PETERLIN, Dr
French Innovative Leukemia Organisation
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- CROSS SECTIONAL
- Target Duration
- 6 Months
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 17, 2024
First Posted
April 22, 2024
Study Start
July 31, 2024
Primary Completion
August 30, 2025
Study Completion (Estimated)
June 1, 2026
Last Updated
November 26, 2025
Record last verified: 2025-11
Data Sharing
- IPD Sharing
- Will not share