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Acute Myeloid Leukemia (AML)Phase 3NCT05907057

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.

A Phase 3 study testing Ivosidenib 500mg Oral Tablet and Azacitidine in people with Acute Myeloid Leukemia (AML), run by Servier Affaires Médicales at 15 sites in 4 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

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… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Acute Myeloid Leukemia (AML)
What is being tested?
Ivosidenib 500mg Oral Tablet and Azacitidine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
245 participants
Where
15 sitesin 4 countries
Study dates
14 Jun 2023to 15 Oct 2027
Phase
Phase 3
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

Who may be able to join

  • 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

Who cannot join

  • 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)

Trial locations and contacts

15 sites in 4 countries. Contact the study team or a site near you to ask about taking part.

Servier Affaires Médicales contact

+33 1 55 72 60 00

scientificinformation@servier.com

15 sites in 4 countries

France5 sites, 5 recruiting

  • CHU Angers - Hôpital Hôtel Dieu

    Angers, Liore, 49100

    Not yet recruiting

  • CHU CAEN - Hôpital de la Côte de Nacre

    Caen, Calvados, 14033

    Recruiting

  • Institut Paoli Calmettes

    Marseille, Bouches-du-Rhône, 13273

    Recruiting

  • CHU Rennes - Hopital Pontchaillou

    Rennes, Ille Et Vilaine, 35033

    Not yet recruiting

  • CHU de Toulouse pt

    Toulouse, Haute Garonne, 31059

    Recruiting

Other trials in France

Italy4 sites, 4 recruiting

  • Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia (Presidio Spedali Civili)

    Brescia, 25123

    Recruiting

  • IRCCS Ospedale Policlinico San Martino

    Genova, 16132

    Recruiting

  • IRCCS Istituto Scientifico Romagnolo Per Lo Studio Dei Tumori "Dino Amadori" - IRST

    Meldola, Forli-Cesena, 47014

    Recruiting

  • Azienda Ospedaliera di Perugia Ospedale S. Maria della Misericordia

    Perugia, 06135

    Recruiting

Other trials in Italy

Netherlands4 sites, 4 recruiting

  • Meander Medisch Centrum

    Amersfoort, 3818 AZ

    Recruiting

  • Amsterdam UMC

    Amsterdam, 1105 AZ

    Recruiting

  • Rijnstate

    Arnhem, 6815 AD

    Not yet recruiting

  • Universitair Medisch Centrum Groningen

    Groningen, 9713 GZ

    Recruiting

Other trials in Netherlands

Austria2 sites

  • AKH - Medizinische Universität Wien

    Vienna, 1090

    Completed

  • Klinikum Wels-Grieskirchen GmbH

    Wels, 4600

    Completed

Other trials in Austria

About this study

Official title: 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).

Read the 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.

Being tested
Ivosidenib 500mg Oral Tablet, Azacitidine
Keywords
IDH1 Mutation AML
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.

  • Ivosidenib 500mg Oral TabletDrug

    Provided as tablets, taken orally as two 250mg tablets once daily.

  • AzacitidineDrug

    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.

Study groups in NCT05907057
Study groupRoleWhat they receive
Open-Label Ivosidenib in combination with AzacitidineExperimentalAll 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.
Receives: Drug: Ivosidenib 500mg Oral Tablet; Drug: Azacitidine

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Adverse Events (AEs)

    Adverse events (AEs) will be graded according to the CTCAE v5.0

    Measured: up to week 116

  2. Number of Serious Adverse Events (SAEs)

    Adverse events (AEs) will be graded according to the CTCAE v5.0

    Measured: up to week 116

  3. Differentiation Syndrome of Grade 2 or higher

    Measured: up to week 116

  4. Number of Adverse Events (AEs) leading to ivosidenib + azacitidine discontinuation

    Measured: up to week 112

  5. Number of Adverse Events (AEs) leading to ivosidenib + azacitidine interruption

    Measured: up to week 112

  6. Number of Adverse Events (AEs) leading to ivosidenib + azacitidine dose reduction

    Measured: up to week 112

  7. Number of Adverse Events (AEs) leading to death

    Measured: up to week 116

  8. Number of clinical laboratory anomalies assessed as Adverse Events (AEs)

    Measured: up to week 116

  9. Number of patients requiring transfusion (platelet and RBC) and the average number of units transfused

    Measured: up to week 116

  10. Rate of infections

    Infection rates will be summarized by classification and will include a count and proportion.

    Measured: up to week 116

  11. QT Prolongation event assessed as Grade 3 or higher

    Measured: up to week 116

Secondary outcomes (11)

  1. Event-free survival (EFS)

    Measured: up to week 116

  2. Proportion of patients who achieve a complete remission (CR)

    Measured: up to week 116

  3. Proportion of patients who achieve complete remission plus complete remission with partial hematologic recovery rate (CR + CRh)

    Measured: up to week 116

  4. Proportion of patients who achieve complete remission plus complete remission with incomplete hematologic recovery rate (CR + CRi)

    Measured: up to week 116

  5. Duration of response (DOR)

    Measured: up to week 116

  6. Time to response (TTR)

    Measured: up to week 116

  7. Overall survival (OS)

    Measured: until study closure

  8. Quality of life (QoL), as measured by Hematologic Malignancy-Patient-Reported Outcome (HM-PRO)

    For patients with a baseline assessment and at least 1 post-baseline assessment that generates a score

    Measured: up to week 116

  9. Quality of life (QoL), as measured by Family Reported Outcome Measure (FROM-16), for caregivers and/or family

    For patients with a baseline assessment and at least 1 post-baseline assessment that generates a score

    Measured: up to week 116

  10. Health economic measures, as assessed by the 5-level EuroQol 5-Dimensions (EQ-5D-5L)

    For patients with a baseline assessment and at least 1 post-baseline assessment that generate a score

    Measured: up to week 116

  11. Average proportion of days at home

    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

    Measured: up to week 116

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted18 Jun 2023
  2. Study start14 Jun 2023
  3. Primary completion15 Oct 2027 (estimated)
  4. Study completion15 Oct 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
245 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05907057
Sponsor's protocol ID
DIM-95031-006
Other IDs
2022-501709-11
Status last verified
May 2026
First submitted
8 Jun 2023
Last updated
8 May 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: 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.

Publications

  • Vyas P, Salek S, Vives S, Recher C, Dohner H, Venditti A, Derrien H, Chatin S, De La Bigne AM, Pelouchova J, Hills R, Nier S. ALIDHE phase 3b study design: ivosidenib + azacitidine in adults with newly diagnosed IDH1 mutant acute myeloid leukemia. Future Oncol. 2025 Dec;21(29):3721-3729. doi: 10.1080/14796694.2025.2567838. Epub 2025 Nov 15.

    PubMed 41241779

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Ivosidenib 500mg Oral Tablet and Azacitidine in people with Acute Myeloid Leukemia (AML), run by Servier Affaires Médicales at 15 sites in 4 countries.

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 6 inclusion and 7 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 8 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 15 sites in France, Italy, Netherlands, Austria. 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.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

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 Servier Affaires Médicales (+33 1 55 72 60 00, scientificinformation@servier.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Adverse Events (AEs) (time frame: up to week 116). The study lists 11 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05907057, which also lists 1 linked publication.

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 8 May 2026.

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