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

NCT ID NCT02074839

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

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.

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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

Contacts and locations

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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WITHDRAWN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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