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New pill targets rare mutation in blood cancers

NCT ID NCT02074839

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

First seen Jun 20, 2026 · Last updated Jun 23, 2026 · Updated 1 time

Summary

This early-stage trial tests an oral drug called AG-120 in people with advanced blood cancers, like 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 drug daily until their cancer worsens or side effects become too severe.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• 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    WITHDRAWN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

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 drug that targets cancer cells with a specific IDH1 mutation)

What this could lead to

If successful, this could lead to a new targeted treatment option for people with certain blood cancers that have an IDH1 mutation.

What could go wrong

This is an early Phase 1 trial, so the drug may not work or may have side effects. It is only for people with a specific genetic mutation, so it won't help everyone.

Conditions

The condition(s) this trial relates to.

Hematologic Neoplasms Leukemia, Myeloid, Acute 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