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New drug combo aims to control rare leukemia without harsh chemo

NCT ID NCT03173248

Disease control Sponsor: Institut de Recherches Internationales Servier Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jun 30, 2026 · Updated 3 times

Summary

This Phase 3 trial tests whether adding ivosidenib (a targeted pill) to standard azacitidine helps adults with a specific IDH1-mutated acute myeloid leukemia (AML) who cannot have intensive chemotherapy. About 146 participants receive either the combo or a placebo plus azacitidine. The study measures how long people live without their leukemia worsening or returning.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Ivosidenib (AG-120) and azacitidine
What this could lead to
If it works, this combination could offer a new, less intensive treatment option for people with a specific type of AML who cannot tolerate strong chemotherapy.
What could go wrong
This is a Phase 3 trial, but results are not yet final. The drug may not improve survival or remission rates enough, and side effects like heart rhythm changes or liver problems are possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

146 people

The number who actually took part.

Started

Jun 2017

Expected to finish

Jun 2026

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Be ≥ 18 years of age and meet at least 1 of the following criteria defining ineligibility for intensive induction chemotherapy (IC): ≥ 75 years old, Eastern Cooperative Oncology Group Performance Status (ECOG PS) score of 2, severe cardiac disorder (e.g., congestive heart failure requiring treatment, left ventricular ejection fraction (LVEF), ≤50%, or chronic stable angina), severe pulmonary disorder (e.g., diffusing capacity of the lungs for carbon monoxide ≤65% or forced expiratory volume in 1 second ≤65%), creatinine clearance \<45 mL/minute, bilirubin \>1.5 times the upper limit of normal (ULN) and/or have any other comorbidity that the Investigator judges to be incompatible with intensive IC and must be reviewed and approved by the Medical Monitor before study enrollment. 2. Have previously untreated AML, defined according to World Health Organization (WHO) criteria, with ≥ 20% leukemic blasts in the bone marrow. Participants with extramedullary disease alone (i.e., no detectable bone marrow and no detectable peripheral blood AML) are not eligible for the study. 3. Have an isocitrate dehydrogenase 1 (IDH1) mutation. 4. Have an ECOG PS score of 0 to 2. 5. Have adequate hepatic function. 6. Have adequate renal function. 7. Have agreed to undergo serial blood and bone marrow sampling. 8. Be able to understand and willing to sign an informed consent form (ICF). 9. Be willing to complete Quality of Life assessments during the study 10. If female with reproductive potential, must have a negative serum pregnancy test prior to the start of study therapy. Females of reproductive potential, as well as fertile men and their female partners of reproductive potential, must agree to use 2 effective forms of contraception. Exclusion Criteria: 1. Are candidates for and willing to receive intensive induction chemotherapy (IC) for their AML. 2. Have received any prior treatment for AML with the exception of hydroxyurea. 3. Have received a hypomethylating agent for myelodysplastic syndrome (MDS). 4. Participants who had previously received an experimental agent for MDS may not be randomized until a washout period has elapsed since the last dose of that agent. 5. Have received prior treatment with an IDH1 inhibitor. 6. Have a known hypersensitivity to any of the components of AG-120, matched placebo, or azacitidine. 7. Are female and pregnant or breastfeeding. 8. Have an active, uncontrolled, systemic fungal, bacterial, or viral infection without improvement despite appropriate antibiotics, antiviral therapy, and/or other treatment. 9. Have a prior history of cancer other than MDS or myeloproliferative disorder, unless the participant has been free of the disease for ≥ 1 year prior to the start of study treatment. 10. Have had significant active cardiac disease within 6 months prior to the start of the study treatment. 11. Have any condition that increases the risk of abnormal ECG or cardiac arrhythmia. 12. Have a condition that limits the ingestion or absorption of drugs administered by mouth. 13. Have uncontrolled hypertension (systolic blood pressure \[BP\] \> 180 mmHg or diastolic BP \> 100 mmHg). 14. Have clinical symptoms suggestive of active central nervous system (CNS) leukemia or known CNS leukemia. 15. Have immediate, life-threatening, severe complications of leukemia, such as uncontrolled bleeding, pneumonia with hypoxia or sepsis, and/or disseminated intravascular coagulation. 16. Have any other medical or psychological condition deemed by the Investigator to be likely to interfere with the participant's ability to give informed consent or participate in the study. 17. Are taking medications that are known to prolong the QT interval unless they can be transferred to other medications within ≥5 half-lives prior to dosing, or unless the medications can be properly monitored during the study. (If equivalent medication is not available, heart rate corrected QT interval \[QTc\] will be closely monitored.) 18. Have a known medical history of progressive multifocal leukoencephalopathy.

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Conditions

The condition(s) this trial relates to.

acute myeloid leukemia leukemia therapy related acute myeloid leukemia and myelodysplastic syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

AML arising from myelodysplastic syndrome (MDS) Leukemia, myeloid, acute Newly diagnosed acute myeloid leukemia (AML) Untreated AML

Contacts and locations

Locations

  • ASST Grande Ospedale Metropolitano Niguarda - Presidio Ospedaliero Ospedale Niguarda Ca' Granda

    Milan, 20162, Italy

  • ASST dei Sette Laghi - Ospedale Di Circolo E Fondazione Macchi

    Varese, Lombardy, 21100, Italy

  • Ajou University Hospital

    Suwon, Gyeonggido, 16499, South Korea

  • Azienda Ospedaliera Citta della Salute e della Scienza di Torino

    Torino, 10126, Italy

  • Birmingham Heartlands Hospital

    Birmingham, West Midlands, B9 5SS, United Kingdom

  • CHRU de Brest - Hopital Morvan

    Brest, 29609, France

  • CHRU de Poitiers La Miletrie

    Poitiers, 86021, France

  • CHU de Grenoble

    Grenoble, 38043, France

  • CHUS H. Clinico U. de Santiago

    Santiago de Compostela, A Coruna, 15706, Spain

  • Cancer Care Manitoba

    Winnipeg, Manitoba, R3E 0V9, Canada

  • Centre Hospitalier Le Mans

    Le Mans, Sarthe, 72037, France

  • Centre Hospitalier Lyon Sud

    Pierre-Bénite, Rhone, 69495, France

  • Centre Hospitalier de Versailles CHV Hopital Andre Mignot

    Le Chesnay, 78 157, France

  • Changhua Christian Medical Foundation Changhua Christian Hospital

    Changhua, 500, Taiwan

  • Charite - Universitatsmedizin Berlin

    Berlin, 13353, Germany

  • Chi Mei Medical Center, Liouying

    Tainan, 736, Taiwan

  • China Medical University Hospital

    Taichung, 40447, Taiwan

  • City Clinical Hospital # 40

    Moscow, 129301, Russia

  • EDOG - Institut Claudius Regaud - PPDS

    Toulouse, 31059, France

  • Fakultni nemocnice Ostrava

    Ostrava, Czechia

  • Flinders Medical Centre

    Bedford Park, South Australia, 5042, Australia

  • Fondazione IRCCS Policlinico San Matteo di Pavia

    Pavia, 27100, Italy

  • Groupe Hospitalier Necker Enfants Malades

    Paris, 75015, France

  • Guangdong Provincial People's Hospital

    Guangzhou, 510080, China

  • Henan Cancer Hospital

    Zhengzhou, Henan, 450008, China

  • Hopital Bretonneau

    Tours, Indre-et-Loire, 37044, France

  • Hopital Haut Leveque

    Pessac, Gironde, 33604, France

  • Hopital de Hautepierre

    Strasbourg, 67200, France

  • Hospital Amaral Carvalho

    Jaú, São Paulo, 17210-120, Brazil

  • Hospital Clinic de Barcelona

    Barcelona, 08036, Spain

  • Hospital Clinico Universitario Lozano Blesa

    Zaragoza, 50009, Spain

  • Hospital General Universitario Gregorio Maranon

    Madrid, 28007, Spain

  • Hospital Santa Marcelina

    São Paulo, 08270-070, Brazil

  • Hospital Sao Jose

    São Paulo, 01321-001, Brazil

  • Hospital Sirio Libanes

    São Paulo, 01308-050, Brazil

  • Hospital Universitari i Politecnic La Fe de Valencia

    Valencia, 46026, Spain

  • Hospital Universitario 12 de Octubre

    Madrid, 28041, Spain

  • Hospital Universitario Fundacion Jimenez Diaz

    Madrid, 28040, Spain

  • Hospital Universitario Germans Trias i Pujol

    Badalona, Barcelona, 08916, Spain

  • Hospital Universitario Ramon y Cajal

    Madrid, 28034, Spain

  • Hospital Universitario Son Espases

    Palma de Mallorca, Balearic Islands, 07010, Spain

  • Hospital Universitario Vall d'Hebron - PPDS

    Barcelona, 08035, Spain

  • Hospital Universitario Virgen del Rocio - PPDS

    Seville, 41013, Spain

  • Hospital Universitario de Gran Canaria Doctor Negrin

    Las Palmas de Gran Canaria, Las Palmas, 35010, Spain

  • Hotel Dieu - Nantes

    Nantes, Loire-Atlantique, 44093, France

  • Institut Gustave Roussy

    Villejuif, 94805, France

  • Institut dHematologie de Basse Normandie

    Caen, 14000, France

  • Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences

    Tianjin, 300020, China

  • Instituto Nacional de Cancer

    Rio de Janeiro, 20230-130, Brazil

  • Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran

    México, 14000, Mexico

  • Instytut Hematologii i Transfuzjologii

    Warsaw, Masovian Voivodeship, 02-776, Poland

  • Istituto Scientifico Romagnolo Per Lo Studio E La Cura Dei Tumori IRST - PPDS

    Meldola, 47014, Italy

  • Japanese Red Cross Society Himeji Hospital

    Himeji, 670-8540, Japan

  • Kaluga Regional Clinical Hospital

    Kaluga, 248007, Russia

  • Kaohsiung Medical University Hospital

    Kaohsiung City, 807, Taiwan

  • Kaplan Medical Center

    Rehovot, 7610000, Israel

  • Klinikum Chemnitz gGmbH

    Chemnitz, Saxony, 09113, Germany

  • Kobe City Medical Center General Hospital

    Kobe, Japan

  • Krankenhaus Hietzing mit Neurologischem Zentrum Rosenhugel

    Vienna, 1130, Austria

  • LMU Klinikum der Universitat Munchen

    München, 81377, Germany

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • Matsuyama Red Cross Hospital

    Matsuyama, Ehime, 790-8524, Japan

  • Medizinische Hochschule Hannover

    Hanover, 30625, Germany

  • National Cancer Center

    Goyang-si, Gyeonggido, 10408, South Korea

  • National Taiwan University Hospital

    Taipei, Taiwan

  • Norton Cancer Institute - Suburban

    Louisville, Kentucky, 40207, United States

  • Ospedale Infermi di Rimini

    Rimini, 47900, Italy

  • Ospedale San Raffaele S.r.l. - PPDS

    Milan, 20132, Italy

  • Peking Union Medical College Hospital

    Beijing, China

  • Pusan National University Hospital

    Busan, 602-739, South Korea

  • Rabin Medical Center - PPDS

    Petah Tikva, 49100, Israel

  • Royal Adelaide Hospital

    Adelaide, South Australia, 5000, Australia

  • Royal Prince Alfred Hospital

    Camperdown, New South Wales, 2050, Australia

  • SINACOR

    Culiacán, 80230, Mexico

  • Salzburger Landeskliniken

    Salzburg, 5020, Austria

  • Seoul National University Hospital

    Seoul, 110-744, South Korea

  • Severance Hospital Yonsei University Health System

    Seoul, 03722, South Korea

  • Shamir Medical Center Assaf Harofeh

    Tzrifin, 70300, Israel

  • The First Affiliated Hospital, College of Medicine, Zhejiang University

    Hangzhou, 310003, China

  • Unicamp Universidade Estadual de Campinas

    Campinas, São Paulo, 13083-878, Brazil

  • Universitair Medisch Centrum Groningen

    Nijmegen, 6525 GA, Netherlands

  • Universitatsklinikum Essen

    Essen, North Rhine-Westphalia, 45122, Germany

  • Universitatsklinikum Leipzig

    Leipzig, 04103, Germany

  • Universitatsklinikum Ulm

    Ulm, 89081, Germany

  • University Health Network

    Toronto, Ontario, M5G 2M9, Canada

  • University of Fukui Hospital

    Fukui, 910-1193, Japan

  • Uniwersytecki Szpital Kliniczny im. Jana Mikulicza Radeckiego we Wroclawiu

    Wroclaw, Lower Silesian Voivodeship, 50-367, Poland

  • Uniwersyteckie Centrum Kliniczne

    Gdansk, 80-214, Poland

  • VU Medisch Centrum

    Amsterdam, North Holland, 1081 HV, Netherlands

  • West China Hospital Sichuan University

    Chengdu, Sichuan, 610041, China

More trials for these conditions

Other studies related to the condition(s) this trial covers.