An observational study in AML, Adult, sponsored by French Innovative Leukemia Organisation. Recruiting at 21 sites in France. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-11-26.
Sponsored by French Innovative Leukemia Organisation · Observational
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.
2,971 studies on the registry are indexed under Leukemia, Myeloid, Acute; 745 are open to participants now.
This study's planned enrollment of 250 is above the median of 120 across 317 observational studies indexed under Leukemia, Myeloid, Acute.
Browse Leukemia, Myeloid, Acute studies →French Innovative Leukemia Organisation is the lead sponsor of 57 studies on the registry; 10 are open to participants now.
Counted across the registry records on this site, refreshed daily.
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
Exclusion Criteria:
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
Time frame: 6 months
characterize the composite response rate (CRc) at any time during follow-up, for the both cohort : 1st line and Relapsed/Refractory (R/R)
CRc is defined as the sum of Complete remission (CR) + Complete remission with partial hematological recovery (CRh) + Complete remission with incomplete count recovery (CRi) + MLFS, according to ELN 2022 criteria
Time frame: 6 months
characterize the Event Free Survival (EFS) in both cohorts : 1st line and Relapsed/Refractory (R/R)
defined as the time from initiation of Ivosidenib (IVO) to the date of treatment failure, hematologic relapse from Complete remission (CR)/Complete remission with partial hematological recovery (CRh)/ Complete remission with incomplete count recovery (CRi)/ Morphologic leukemia-free state (MLFS) or death from any cause, whichever occurs first; Treatment failure is defined as not achieving either CR, CRh,CRi or MLFS by day 180 from Ivo start
Time frame: 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)
description of grade 3/4 SAE and death according to CTCAE v5
Time frame: 6 months
describe the management of treatment by Ivosidenib in both cohorts : 1st line and Relapsed/Refractory (R/R)
daily dose of Ivosidenib, description of Ivosidenib dose modification
Time frame: 6 months
describe the management of treatment by Ivosidenib in both cohorts : 1st line and Relapsed/Refractory (R/R)
duration of treatment by Ivosidenib
Time frame: 6 months
Plan to share: No
No publications or documents are linked to this record.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
French Innovative Leukemia Organisation