Study Evaluating the Efficacy and Safety of the Addition of Ivosidenib to Oral Azacitidine (Onureg®) in Patients Over 55 With Acute Myeloid Leukemia (AML) and IDH1 Mutation, in Complete Remission After Intensive Chemotherapy.

NCT07463768 | Not Yet Recruiting Phase 2 DMC

• 1 other identifier: FILO-AML-01-MIVONU
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 20

Brief Summary

After remission post-induction and consolidation, maintenance therapy by an ivosidenib and oral azacitidine combination is susceptible to improve the prevention of AML relapse, which remains a major issue in the study population. We assume that the combination of ivosidenib with oral azacitidine will not be less well tolerated than in combination with the subcutaneous form, therapeutic regimen authorized until progression or toxicity. Ivosidenib and Onureg®, being already authorized treatments, it has been decided to use the classic administration schedules and dosages in combination. The primary objective of the study is to evaluate relapse free survival (RFS) at 24 months in patients receiving oral azacitidine with ivosidenib.

Conditions

Acute Myeloid Leukemia

Keywords

acute myeloid leukemia and IDH1 mutation; complete remission after intensive chemotherapy; ivosidenib and oral azacitidine

Outcome Measures

Primary Outcomes (1)

• RFS 24 months

  relapse-free-survival (RFS) at 24 months in patients receiving oral azacitidine with ivosidenib

  24 months

Secondary Outcomes (4)

• RFS 12 months

  12 months

• Overall survival

  24 months

• EFS MRD neg

  24 months

• TEAEs

  24 months

Study Arms (1)

ivosidenib + Oral azacitidine

EXPERIMENTAL

cf intervention

Drug: Ivosidenib + oral azacitidine

Interventions

Ivosidenib + oral azacitidine DRUG

Subjects will be administered treatment over 28-day cycles until progression or another reason (unacceptable toxicity, patient choice to discontinue study treatment, sponsor decision, HSCT at any time, patients who require the use of any of the excluded therapies). The treatment is the association of ivosidenib with oral azacitidine. * The investigational medicinal product is ivosidenib. Two tablets (500 mg) will be administered as a single dose, once daily, during 28 days-cycles until discontinuation. * The auxiliary treatment is oral azacitidine. Oral AZA 300 mg will be given once-daily for 14 days of repeated 28-day treatment cycles. For all patients, oral AZA will be interrupted systematically after day 14 of each 28-days cycle.

ivosidenib + Oral azacitidine

Eligibility Criteria

• Age: 55 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female ≥ 55 years of age at the time of signing informed consent
• Patients with confirmation of newly diagnosed AML by 2022 WHO criteria
• Presence of IDH1 R132 mutation at AML diagnosis
• Achievement CR or CRi following induction therapy by intensive chemotherapy (according to ELN 2022, appendix 2), within 17 weeks prior to enrollment.
• Received at least 2 consolidations :
• with intermediate dose of cytarabine (IDAC)
• or with standard dose cytarabine and idarubicin (5+1)
• Patients who are not candidate for Allo-HSCT
• Adequate baseline organ function defined by the following criteria:
• Estimated Glomerular Filtration Rate (eGFR) ≥ 30 ml/min (using CKD-EPI).
• aspartate aminotransferase (AST) ≤ 2.5 × ULN
• alanine aminotransferase (ALT) ≤ 2.5× ULN
• bilirubin ≤ 1.5 × ULN
• ECOG \< 3 (appendix 1)
• Absence of any psychological, familial, sociological, or geographical conditions potentially hampering compliance with the study protocol and follow-up schedule

You may not qualify if:

• Acute promyelocytic leukemia (FAB M3) with t(15;17) or its molecular equivalents (PML::RARA)
• AML associated with t(9;22) or molecular evidence of such a translocation
• Prior BM or hematopoietic stem cell transplantation
• CR/CRi following treatment with hypomethylating agents
• Proven central nervous system leukemia
• Candidate for Allo-HSCT at screening
• Diagnosis of malignant disease within the previous 12 months (excluding MDS or CMML, basal cell carcinoma of the skin without complications, "in- situ" carcinoma of the cervix or breast, or other local malignancy excised or irradiated with a high probability of cure)
• Abnormal cardiac status with any of the following:
• Unstable angina
• Myocardial infarction within the last 6 months
• Significant cardiac arrhythmia
• New York Heart Association (NYHA) class 3 or 4 congestive heart failure
• Congenital long QT syndrome, familial history of sudden death or polymorphic ventricular arrhythmias and QT/QTc interval \> 500 msec regardless of the correction method.
• For subject with 450 ≤ QTc ≤ 500 ms, practitioners should thoroughly reassess the benefit/risk of initiating ivosidenib. In case QTc interval prolongation is between 480 msec and 500 msec, initiation of treatment with ivosidenib should remain exceptional and be accompanied by close monitoring. This issue will be discussed with coordinating investigator.
• Uncontrolled systemic fungal, bacterial or viral infection (defined as ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics or other treatment)

Sponsors & Collaborators

• French Innovative Leukemia Organisation: lead
• Servier Affaires Médicales: collaborator

Study Sites (20)

CHU Angers

Angers, 49933, France

Location

CH côte Basque

Bayonne, 64109, France

Location

CHU Besançon

Besançon, 25030, France

Location

Hopital d'Instruction des Armées Percy

Clamart, 92140, France

Location

CHU Henri Mondor

Créteil, 94000, France

Location

CHU Grenoble

Grenoble, 38043, France

Location

Chru de Lille - Hopital Claude Huriez

Lille, 59037, France

Location

CHU de Limoges

Limoges, 87000, France

Location

CHU de Nantes

Nantes, 44093, France

Location

CHU de Nîmes - Institut de Cancérologie du Gard

Nîmes, 30029, France

Location

Hôpital Saint Louis

Paris, 75010, France

Location

CH de Perpignan

Perpignan, 66046, France

Location

Hôpital Haut-Lévêque

Pessac, 33604, France

Location

Hôpital Lyon-Sud

Pierre-Bénite, 69310, France

Location

Chu de Rennes - Hopital Pontchaillou

Rennes, 35033, France

Location

Centre Henri Becquerel

Rouen, 76038, France

Location

CHU de TOULOUSE - IUCT ONCOPOLE

Toulouse, 31059, France

Location

Chru Bretonneau

Tours, 37044, France

Location

CHRU Nancy

Vandœuvre-lès-Nancy, 54500, France

Location

Gustave Roussy

Villejuif, 94800, France

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

ivosidenib; Azacitidine

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Study Officials

• Arnaud PIGNEUX

  University Hospital, Bordeaux

  PRINCIPAL INVESTIGATOR

• Stephane DE BOTTON

  Gustave Roussy, Cancer Campus, Grand Paris

  PRINCIPAL INVESTIGATOR

• Maël HEIBLIG

  Centre Hospitalier Lyon Sud

  PRINCIPAL INVESTIGATOR

• Yohan DESBROSSES

  CHRU Jean Minjoz Besançon

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Cindy FENOLL

CONTACT

+33607065843; c.fenoll@filo-leucemie.org

Ariane MINEUR

CONTACT

+ 33557623108; ariane.mineur@chu-bordeaux.fr

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 18, 2026
• First Posted: March 11, 2026
• Study Start (Estimated): September 1, 2026
• Primary Completion (Estimated): September 1, 2030
• Study Completion (Estimated): September 1, 2030
• Last Updated: March 11, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

FR (20)