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New pill targets rare gene mutation in Hard-to-Treat blood cancers

NCT ID NCT02074839

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 study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Updated 1 time

Summary

This early-stage trial tests an oral drug called AG-120 in people with advanced blood cancers, like acute myeloid leukemia, that have a specific IDH1 mutation. The study aims to find the safest dose and see if the drug can control the disease. About 291 participants will take the pill daily until their disease gets worse or side effects become too severe.

What this could mean

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

Active substance
AG-120 (a pill that targets cancer cells with a specific IDH1 mutation)
What this could lead to
If it works, this could point toward a new treatment option for people with certain blood cancers that have not responded to other therapies.
What could go wrong
This is an early phase 1 trial, so the drug may not work or could cause unexpected side effects. It is only for people with a specific gene mutation, so it would not help everyone.

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 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 291 people

The number the study aims to enrol. It can still change while the study runs.

Started

Mar 2014

Expected to finish

Dec 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.

Key Inclusion Criteria: * Subject must be ≥18 years of age. * Subjects must have documented IDH1 R132 gene-mutated advanced hematologic malignancy based on local or central evaluation. * Subjects must be amenable to serial bone marrow biopsies, peripheral blood sampling, and urine sampling during the study. * Subjects must have ECOG PS of 0 to 2. * Platelet count ≥20,000/µL (Transfusions to achieve this level are allowed). * Subjects must have adequate hepatic function as evidenced by: Aspartate aminotransferase (AST), alanine aminotransferase (ALT), and alkaline phosphatase (ALP) ≤3.0 × ULN, unless considered due to leukemic disease and serum total bilirubin ≤1.5 x upper limit of normal (ULN), unless considered due to Gilbert's disease or leukemic disease * Subjects must have adequate renal function as evidenced by a serum creatinine ≤2.0 × ULN or creatinine clearance \>40mL/min based on Cockroft-Gault glomerular filtration rate (GFR) * Subjects must be recovered from any clinically relevant toxic effects of any prior surgery, radiotherapy, or other therapy intended for the treatment of cancer. * Female subjects with reproductive potential must have a negative serum pregnancy test within 7 days prior to the start of therapy and on the first day of study drug administration. Key Exclusion Criteria: * Subjects who have undergone hematopoietic stem cell transplant (HSCT) within 60 days of the first dose of AG-120, or subjects on immunosuppressive therapy post HSCT at the time of screening, or with clinically significant graft-versus-host disease (GVHD). (The use of a stable dose of oral steroids post HSCT and/or topical for ongoing skin GVHD is permitted.) * Subjects who received systemic anticancer therapy or radiotherapy \<14 days prior to their first day of study drug administration. (Hydroxyurea is allowed prior to enrollment and after the start of AG-120). * Subjects who received an investigational agent \<14 days prior to their first day of study drug administration. * Subjects who are pregnant or breastfeeding. * Subjects with an active severe infection or with an unexplained fever \>38.5°C during screening visits or on their first day of study drug administration (at the discretion of the Investigator, subjects with tumor fever may be enrolled). * Subjects with New York Heart Association (NYHA) Class III or IV congestive heart failure or LVEF \<40% by echocardiogram (ECHO) or multi-gated acquisition (MUGA) scan within approximately 28 days of C1D1. * Subjects with a history of myocardial infarction within the last 6 months of screening. * Subjects with a known unstable or uncontrolled angina pectoris. * Subjects with a known history of severe and/or uncontrolled ventricular arrhythmias. * Subjects with known unstable or uncontrolled angina pectoris. * Subjects with heart-rate corrected QT (QTc) interval ≥450 ms or other factors that increase the risk of QT prolongation or arrhythmic events. * Patients taking medications that are known to prolong the QT interval * Subjects with known infection with human immunodeficiency virus (HIV) or active hepatitis B or C. * Subjects with clinical symptoms suggesting active central nervous system (CNS) leukemia or known CNS leukemia. Evaluation of cerebrospinal fluid is only required if there is a clinical suspicion of CNS involvement by leukemia during screening. * Subjects with immediately life-threatening, severe complications of leukemia such as uncontrolled bleeding, pneumonia with hypoxia or shock, and/or disseminated intravascular coagulation.

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Conditions

The condition(s) this trial relates to.

acute myeloid leukemia hematopoietic and lymphoid system neoplasm myelodysplastic syndrome Myelodysplastic Syndromes Recurrence

As listed by the trial registrant

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

Myelodysplastic syndromes Other IDH1-mutated positive hematologic malignancies Relapsed or refractory acute myeloid leukemia (AML) Untreated AML

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    6 sites in 2 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Central Lyon Sud

    RECRUITING

    Pierre-Bénite, 69310, France

  • City of Hope

    ACTIVE_NOT_RECRUITING

    Duarte, California, 91010, United States

  • Cleveland Clinic

    WITHDRAWN

    Cleveland, Ohio, 44124, United States

  • Cornell Cancer Center

    TERMINATED

    New York, New York, 10065, United States

  • Dana Farber Cancer Institute

    TERMINATED

    Boston, Massachusetts, 02215, United States

  • Duke Cancer Center

    TERMINATED

    Durham, North Carolina, 27705, United States

  • Emory University

    ACTIVE_NOT_RECRUITING

    Atlanta, Georgia, 30322, United States

  • Hopital Haut-Leveque

    RECRUITING

    Pessac, 33600, France

  • Hopital La Timone

    TERMINATED

    Marseille, France

  • Institute Gustave Roussly (IGR)

    RECRUITING

    Villejuif, 94800, France

  • John Hopkins Cancer Center

    ACTIVE_NOT_RECRUITING

    Baltimore, Maryland, 21287, United States

  • Karmanos Cancer Center

    TERMINATED

    Detroit, Michigan, 48201, United States

  • MD Anderson Cancer Center

    RECRUITING

    Houston, Texas, 77030, United States

  • Massachusetts General Hospital

    TERMINATED

    Boston, Massachusetts, 02214, United States

  • Mayo Clinic-AZ

    TERMINATED

    Phoenix, Arizona, 85259, United States

  • Mayo Clinic-Jacksonville

    TERMINATED

    Jacksonville, Florida, 32224, United States

  • Medical University of South Carolina

    RECRUITING

    Charleston, South Carolina, 29425, United States

  • Memorial Sloan Kettering Cancer Center

    ACTIVE_NOT_RECRUITING

    New York, New York, 10021, United States

  • Moffit Cancer Center

    ACTIVE_NOT_RECRUITING

    Tampa, Florida, 33612, United States

  • Northwestern University Medical Hospital

    TERMINATED

    Chicago, Illinois, 60611, United States

  • Ohio State University

    TERMINATED

    Columbus, Ohio, 43210, United States

  • Oregon Health and Science University

    TERMINATED

    Portland, Oregon, 97239, United States

  • Sarah Cannon Research Institute

    TERMINATED

    Nashville, Tennessee, 37203, United States

  • UT Southwestern Medical Center

    RECRUITING

    Dallas, Texas, 75390, United States

  • University of Alabama at Birmingham

    ACTIVE_NOT_RECRUITING

    Birmingham, Alabama, 35294, United States

  • University of California-Los Angeles

    TERMINATED

    Los Angeles, California, 90095, United States

  • University of California-San Francisco

    TERMINATED

    San Francisco, California, 94143, United States

  • University of Colorado Denver

    TERMINATED

    Aurora, Colorado, 80045, United States

  • University of Miami

    TERMINATED

    Miami, Florida, 33136, United States

  • Washington University

    TERMINATED

    St Louis, Missouri, 63110, United States

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