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.

NCT ID: NCT05907057

Last Updated: 2025-04-11

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

245 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-06-14

Study Completion Date

2026-12-15

Brief Summary

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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).

Detailed Description

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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.

Conditions

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Acute Myeloid Leukemia (AML)

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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.

Group Type EXPERIMENTAL

Ivosidenib 500mg Oral Tablet

Intervention Type DRUG

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

Azacitidine

Intervention Type DRUG

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.

Interventions

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Ivosidenib 500mg Oral Tablet

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

Intervention Type 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.

Intervention Type DRUG

Eligibility Criteria

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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)
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Servier Affaires Médicales

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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AKH - Medizinische Universität Wien

Vienna, , Austria

Site Status COMPLETED

Klinikum Wels-Grieskirchen GmbH

Wels, , Austria

Site Status COMPLETED

Institut Paoli Calmettes

Marseille, Bouches-du-Rhône, France

Site Status RECRUITING

CHU CAEN - Hôpital de la Côte de Nacre

Caen, Calvados, France

Site Status RECRUITING

CHU de Toulouse pt

Toulouse, Haute Garonne, France

Site Status RECRUITING

CHU Rennes - Hopital Pontchaillou

Rennes, Ille et Vilaine, France

Site Status NOT_YET_RECRUITING

CHU Angers - Hôpital Hôtel Dieu

Angers, Liore, France

Site Status NOT_YET_RECRUITING

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

Brescia, Brescia, Italy

Site Status RECRUITING

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

Meldola, Forli-Cesena, Italy

Site Status RECRUITING

IRCCS Ospedale Policlinico San Martino

Genova, Genova, Italy

Site Status RECRUITING

Azienda Ospedaliera di Perugia Ospedale S. Maria della Misericordia

Perugia, Perugia, Italy

Site Status RECRUITING

Meander Medisch Centrum

Amersfoort, , Netherlands

Site Status RECRUITING

Amsterdam UMC

Amsterdam, , Netherlands

Site Status RECRUITING

Rijnstate

Arnhem, , Netherlands

Site Status NOT_YET_RECRUITING

Universitair Medisch Centrum Groningen

Groningen, , Netherlands

Site Status RECRUITING

Countries

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Austria France Italy Netherlands

Central Contacts

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Servier Affaires Médicales

Role: CONTACT

+33 1 55 72 60 00

Other Identifiers

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2022-501709-11

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

DIM-95031-006

Identifier Type: -

Identifier Source: org_study_id

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