An Open-label Phase 3b Study of Ivosidenib in Combination With Azacitidine in Adult Patients Newly Diagnosed With IDH1m Acute Myeloid Leukemia (AML) Ineligible for Intensive Induction Chemotherapy.

May 6, 2026 updated by: Servier Affaires Médicales

A Single Arm, Open-label Phase 3b Study to Describe the Safety and Tolerability of Ivosidenib in Combination With Azacitidine in Adult Patients Newly Diagnosed With IDH1m Acute Myeloid Leukemia (AML) Ineligible for Intensive Induction Chemotherapy

The purpose of this study is to learn more about the safety and efficacy of ivosidenib taken with azacitidine to treat adult patients with acute myeloid leukemia (AML) who are presenting a gene mutation called IDH1 (isocitrate dehydrogenase1 mutation-positive [IDH1m]) and cannot receive treatment with intensive chemotherapy (IC).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

Participants who are eligible and enroll in the study will attend a study visit on the first day of each 28-day cycle. Study visits will consist of a physical exam, blood work, electrocardiogram (ECG) and other assessments. After treatment discontinuation participants will be contacted every 12 weeks through the end of the study (currently planned for 2026) to assess survival. The study drug, Ivosidenib, will be taken once daily throughout the duration of participation in the study, and Azacitidine will only be administered for 7 days at the beginning of each 28 day cycle. If at any point ivosidenib is made available as a medical prescription at the patient's site, the patient will switch to commercial product and will continue to be followed according to the protocol.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
245

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Austria
      • Vienna, Austria, 1090
        • Completed
        • AKH - Medizinische Universität Wien
      • Wels, Austria, 4600
        • Completed
        • Klinikum Wels-Grieskirchen GmbH
  • France
    • Bouches-du-Rhône
      • Marseille, Bouches-du-Rhône, France, 13273
        • Recruiting
        • Institut Paoli Calmettes
    • Calvados
      • Caen, Calvados, France, 14033
        • Recruiting
        • Chu Caen - Hôpital de La Côte de Nacre
    • Haute Garonne
      • Toulouse, Haute Garonne, France, 31059
        • Recruiting
        • CHU de Toulouse pt
    • Ille Et Vilaine
      • Rennes, Ille Et Vilaine, France, 35033
        • Not yet recruiting
        • CHU Rennes - Hôpital Pontchaillou
    • Liore
      • Angers, Liore, France, 49100
        • Not yet recruiting
        • Chu Angers - Hôpital Hôtel Dieu
  • Italy
      • Brescia, Italy, 25123
        • Recruiting
        • Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia (Presidio Spedali Civili)
      • Genova, Italy, 16132
        • Recruiting
        • IRCCS Ospedale Policlinico San Martino
      • Perugia, Italy, 06135
        • Recruiting
        • Azienda Ospedaliera Di Perugia Ospedale s. Maria Della Misericordia
    • Forli-Cesena
      • Meldola, Forli-Cesena, Italy, 47014
        • Recruiting
        • IRCCS Istituto Scientifico Romagnolo Per Lo Studio Dei Tumori "Dino Amadori" - IRST
  • Netherlands
      • Amersfoort, Netherlands, 3818 AZ
        • Recruiting
        • Meander Medisch Centrum
      • Amsterdam, Netherlands, 1105 AZ
        • Recruiting
        • Amsterdam UMC
      • Arnhem, Netherlands, 6815 AD
        • Not yet recruiting
        • Rijnstate
      • Groningen, Netherlands, 9713 GZ
        • Recruiting
        • Universitair Medisch Centrum Groningen

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Has untreated Acute Myeloid Leukemia (AML)
  • Have a documented IDH1 R132 gene-mutated disease
  • Have at least one of the following making yourself ineligible for intensive chemotherapy (IC): 75 years or older, Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 2, or any comorbidity that the investigator judges to be incompatible with IC including but not limited to severe cardiac or pulmonary disorder, creatinine clearance less than 45 mL/minute, or bilirubin greater than 1.5 times the upper limit of normal
  • Has adequate hepatic (liver) and renal (kidney) function
  • Female participants of reproductive potential must have a negative blood pregnancy test and must use effective contraception during treatment and for at least 6 months following treatment
  • Fertile male participants with female partners of reproductive potential must use effective contraception during treatment and for at least 3 months following treatment

Exclusion Criteria:

  • Has received any prior treatment for AML, with the exception of hydroxyurea or leukapheresis for white blood cell count control
  • Has received prior treatment with an IDH1 inhibitor
  • Is a woman who is pregnant or breastfeeding
  • Has an active, uncontrolled, systemic fungal, bacterial, or viral infection (including human immunodeficiency virus [HIV], active hepatitis B (HBV), or hepatitis C virus [HCV]) without improvement despite appropriate antibiotics, antiviral therapy, and/or other treatment
  • Has had significant active cardiac disease within 6 months prior to the start of study treatment, including Class III or IV congestive heart failure, myocardial infarction (heart attack), unstable angina (chest pain), and/or stroke
  • Has dysphagia (difficulty swallowing), short-gut syndrome, gastroparesis (stomach paralysis), or any other condition that limits the ingestion or gastrointestinal absorption of orally administered drugs
  • Has uncontrolled hypertension (high blood pressure)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Open-Label Ivosidenib in combination with Azacitidine
All participants will receive both Ivosidenib and Azacitidine for a maximum of 28 cycles. Each cycle will be 4 weeks or 28 days long. Ivosidenib will be taken continuously throughout each cycle and Azacitidine will be taken only for 7 days at the beginning of each cycle.
Drug: Ivosidenib 500mg Oral Tablet
Provided as tablets, taken orally as two 250mg tablets once daily.
Drug: Azacitidine
Administered subcutaneously (SC) or intravenously (IV) at a dose of 75mg/m2/day for 7 days, either consecutively on Days 1-7 or discontinuously for Days 1-5 and 8-9 of each cycle. The 7 days of administration will occur at the beginning of every 4 week-long cycle.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Number of Adverse Events (AEs)
Time Frame: up to week 116
Adverse events (AEs) will be graded according to the CTCAE v5.0
up to week 116
Number of Serious Adverse Events (SAEs)
Time Frame: up to week 116
Adverse events (AEs) will be graded according to the CTCAE v5.0
up to week 116
Differentiation Syndrome of Grade 2 or higher
Time Frame: up to week 116
up to week 116
Number of Adverse Events (AEs) leading to ivosidenib + azacitidine discontinuation
Time Frame: up to week 112
up to week 112
Number of Adverse Events (AEs) leading to ivosidenib + azacitidine interruption
Time Frame: up to week 112
up to week 112
Number of Adverse Events (AEs) leading to ivosidenib + azacitidine dose reduction
Time Frame: up to week 112
up to week 112
Number of Adverse Events (AEs) leading to death
Time Frame: up to week 116
up to week 116
Number of clinical laboratory anomalies assessed as Adverse Events (AEs)
Time Frame: up to week 116
up to week 116
Number of patients requiring transfusion (platelet and RBC) and the average number of units transfused
Time Frame: up to week 116
up to week 116
Rate of infections
Time Frame: up to week 116
Infection rates will be summarized by classification and will include a count and proportion.
up to week 116
QT Prolongation event assessed as Grade 3 or higher
Time Frame: up to week 116
up to week 116

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Event-free survival (EFS)
Time Frame: up to week 116
up to week 116
Proportion of patients who achieve a complete remission (CR)
Time Frame: up to week 116
up to week 116
Proportion of patients who achieve complete remission plus complete remission with partial hematologic recovery rate (CR + CRh)
Time Frame: up to week 116
up to week 116
Proportion of patients who achieve complete remission plus complete remission with incomplete hematologic recovery rate (CR + CRi)
Time Frame: up to week 116
up to week 116
Duration of response (DOR)
Time Frame: up to week 116
up to week 116
Time to response (TTR)
Time Frame: up to week 116
up to week 116
Overall survival (OS)
Time Frame: until study closure
until study closure
Quality of life (QoL), as measured by Hematologic Malignancy-Patient-Reported Outcome (HM-PRO)
Time Frame: up to week 116
For patients with a baseline assessment and at least 1 post-baseline assessment that generates a score
up to week 116
Quality of life (QoL), as measured by Family Reported Outcome Measure (FROM-16), for caregivers and/or family
Time Frame: up to week 116
For patients with a baseline assessment and at least 1 post-baseline assessment that generates a score
up to week 116
Health economic measures, as assessed by the 5-level EuroQol 5-Dimensions (EQ-5D-5L)
Time Frame: up to week 116
For patients with a baseline assessment and at least 1 post-baseline assessment that generate a score
up to week 116
Average proportion of days at home
Time Frame: up to week 116
Defined by subtracting the number of care days (days hospitalized or seen in an ED / oncology clinic / infusion center) from the total days of follow-up, divided by total days of follow-up
up to week 116

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Servier Affaires Médicales

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
June 14, 2023

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 15, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
October 15, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
June 8, 2023

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 8, 2023

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 18, 2023

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 8, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 6, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • DIM-95031-006
  • 2022-501709-11 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Qualified scientific and medical researchers can request access to anonymized patient-level and study-level clinical trial data.

Access can be requested for all interventional clinical studies:

  • used for Marketing Authorization (MA) of medicines and new indications approved after 1 January 2014 in the European Economic Area (EEA) or the United States (US).
  • where Servier is the Marketing Authorization Holder (MAH). The date of the first MA of the new medicine (or the new indication) in one of the EEA Member States will be considered for this scope.

In addition, access can be requested for all interventional clinical studies in patients:

  • sponsored by Servier
  • with a first patient enrolled as of 1 January 2004 onwards
  • for New Chemical Entity or New Biological Entity (new pharmaceutical form excluded) for which development has been terminated before any Marketing authorization (MA) approval.

IPD Sharing Time Frame

After Marketing Authorization in EEA or US if the study is used for the approval.

IPD Sharing Access Criteria

Researchers should register on Servier Data Portal and fill in the research proposal form. This form in four parts should be fully documented. The Research Proposal Form will not be reviewed until all mandatory fields are completed.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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