AML, AdultNCT06377579
OBServatory of Compassionate Use of IVOsidenib in France for Patients With Acute Myeloid Leukemia
An observational study of AML, Adult, run by French Innovative Leukemia Organisation at 21 sites in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- AML, Adult
- How many people
- 250 participants
- Where
- 21 sitesFrance
- Study dates
- 31 Jul 2024to 1 Jun 2026
- How long will I take part?
- 6 Months
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: 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
Who may be able to join
- 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.
Who cannot join
- Patients who expressed their opposition to entered in the study
- Patients who received IVO through a trial
Trial locations and contacts
21 sites in 1 country. Contact the study team or a site near you to ask about taking part.
21 sites in 1 country
France21 sites, 21 recruiting
- Not yet recruiting
Amiens CHU
Amiens
Delphine Lebon, Dr, lebon.delphine@chu-amiens.fr
- Not yet recruiting
Angers CHU
Angers
corentin Orvain, Dr, corentin.orvain@chu-angers.fr
- Not yet recruiting
Bayonne CH
Bayonne
Anne Banos, Dr, abanos@ch-cotebasque.fr
- Not yet recruiting
Besançon CHU
Besançon
Yohann Desbrosses, Dr, ydesbrosses@chu-besancon.fr
- Recruiting
CHU Estaing
Clermont-Ferrand
Gaspar Aspas Requena, MD, gaspasrequena@chu-clermontferrand.fr
- Recruiting
Créteil CHU HENRI MONDOR
Créteil
Cécile Pautas, Dr, cecile.pautas@aphp.fr
- Recruiting
DUNKERQUE-Hôpital Alexandra Lepève
Dunkirk, 59385
Adrien DANIEL, Dr, Adrien.daniel@ch-dunkerque.fr
Adrien DANIEL, Dr
- Not yet recruiting
Grenoble CHU
Grenoble
Martin Carré, Dr, MCarre1@chu-grenoble.fr
- Not yet recruiting
Le Mans CH
Le Mans
Kamel Laribi, Dr, klaribi@ch-lemans.fr
- Not yet recruiting
Lyon sud CHU
Lyon
Mael Heiblig, Pr, mael.heiblig@chu-lyon.fr
- Not yet recruiting
Marseille IPC
Marseille
Sylvain GARCIAZ, Dr, GARCIAZS@ipc.unicancer.fr
- Recruiting
Meaux CH de l'Est francilien
Meaux
Jamilé Frayfer, Dr, jfrayfer@ghef.fr
- Not yet recruiting
Montpellier - Chu Saint Eloi
Montpellier
Ludovic Gabellier, Dr, l-gabellier@chu-montpellier.fr
- Recruiting
Mulhouse Chu
Mulhouse, 68100
Mario OJEDA-URIBE, Dr, ojeda-uribem@ghrmsa.fr
Mario ODJEDA-URIBE
- Recruiting
Nantes CHU
Nantes
Pierre Peterlin, Dr, pierre.peterlin@chu-nantes.fr
- Not yet recruiting
Nice CHU
Nice
Thomas Cluzeau, Prof, cluzeau.t@chu-nice.fr
- Not yet recruiting
Orléans CHU
Orléans, 45000
Magda ALEXIS, Dr
magda.alexis@chr-orleans.fr
- Not yet recruiting
Paris Saint Louis
Paris
Emmanuel Raffoux, Prof, emmanuel.raffoux@aphp.fr
- Not yet recruiting
Bordeaux CHU
Pessac
Pierre-Yves Dumas, Prof, pierre-yves.dumas@chu-bordeaux.fr
- Not yet recruiting
ICANS - Institut de cancérologie de strasbourg europe
Strasbourg
Celestine SIMAND, Dr, c.simand@icans.eu
- Not yet recruiting
Toulouse - IUCT Oncopole - Service d'Hématologie
Toulouse
Sarah BERTOLI, Dr, bertoli.sarah@iuct-oncopole.fr
About this study
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.
- Conditions
- AML, Adult
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| AML in 1st line at inclusion | Not described | |
| AML in R/R at inclusion | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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)
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
Measured: 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
Measured: 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
Measured: 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
Measured: 6 months
describe the management of treatment by Ivosidenib in both cohorts : 1st line and Relapsed/Refractory (R/R)
duration of treatment by Ivosidenib
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Apr 2024
- Study start31 Jul 2024
- Primary completion30 Aug 2025
- Study completion1 Jun 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AML, AdultStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 250 participants (estimated)
- Target duration
- 6 Months
Sponsor and investigators
- Lead sponsor
- French Innovative Leukemia Organisation
- Sponsor type
- University, hospital or other
- Collaborators
- Acute Leukemia French Association
- Responsible party
- Sponsor
- Investigators
- Pierre PETERLIN, Dr, Principal investigator, French Innovative Leukemia Organisation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06377579
- Sponsor's protocol ID
- FILObsLAM_IVOOBS
- Status last verified
- Nov 2025
- First submitted
- 17 Apr 2024
- Last updated
- 26 Nov 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- OBServatory of Compassionate Use of IVOsidenib in France for Patients With Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT06377579
- Acronym / sponsor ID
- FILObsLAM_IVOOBS
- Conditions
- AML, Adult
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 250 participants (estimated)
- Lead sponsor
- French Innovative Leukemia OrganisationUniversity, hospital or other
- Collaborators
- Acute Leukemia French Association
- Study start
- 31 Jul 2024 (actual)
- Primary completion
- 30 Aug 2025 (actual)
- Study completion
- 1 Jun 2026 (estimated)
- First posted
- 22 Apr 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Target follow-up
- 6 Months
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| AML in 1st line at inclusion | – | – |
| AML in R/R at inclusion | – | – |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- 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
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (2 listed)
- Patients who expressed their opposition to entered in the study
- Patients who received IVO through a trial
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
characterize the Overall survival (OS) in the both cohort : 1st line and Relapsed/Refractory (R/R)
Time frame: 6 months
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
5 secondary outcomes
characterize the composite response rate (CRc) at any time during follow-up, for the both cohort : 1st line and Relapsed/Refractory (R/R)
Time frame: 6 months
characterize the Event Free Survival (EFS) in both cohorts : 1st line and Relapsed/Refractory (R/R)
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)
Time frame: 6 months
describe the management of treatment by Ivosidenib in both cohorts : 1st line and Relapsed/Refractory (R/R)
Time frame: 6 months
describe the management of treatment by Ivosidenib in both cohorts : 1st line and Relapsed/Refractory (R/R)
Time frame: 6 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Nov 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Pierre PETERLIN, Dr principal investigator
French Innovative Leukemia Organisation
21 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of AML, Adult, run by French Innovative Leukemia Organisation at 21 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 26 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 21 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Ariane MINEUR (+33 (0)5 57 62 31 08, ariane.mineur@chu-bordeaux.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
characterize the Overall survival (OS) in the both cohort : 1st line and Relapsed/Refractory (R/R) (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Nov 2025.