New pill targets genetic flaw in tough cancers
NCT ID NCT02073994
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial tested an oral drug called AG-120 in 174 people with advanced solid tumors (including bile duct cancer, cartilage cancer, and brain tumors) that have a specific IDH1 mutation. The study first found a safe dose, then expanded to see how well the drug works. Participants stayed on the drug until their disease got worse or side effects became too much.
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 (an oral drug that targets cancer cells with a specific genetic mutation)
- What this could lead to
- If successful, this could point toward a new treatment option for certain hard-to-treat cancers that have an IDH1 mutation.
- What could go wrong
- This is an early Phase 1 trial, so the main goals are safety and dosing, not proof of effectiveness. The drug may not shrink tumors or may cause side effects.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Study site
Scottsdale, Arizona, 85258, United States
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Study site
Los Angeles, California, 90095, United States
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Study site
Aurora, Colorado, 80045, United States
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Study site
Miami, Florida, 33136, United States
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Study site
Baltimore, Maryland, 21218, United States
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Study site
Boston, Massachusetts, 02215, United States
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Study site
New York, New York, 10065, United States
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Study site
Nashville, Tennessee, 37203, United States
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Study site
Dallas, Texas, 75390, United States
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Study site
Houston, Texas, 77030, United States
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Study site
San Antonio, Texas, 78229, United States
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Study site
Villejuif, 94800, France
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- A new test could reveal how radiation affects the liver
- Can a Tumor's genetic blueprint guide better treatment for kids with brain cancer?
- Can adding lenvatinib to standard care outsmart a tough brain cancer?
- Can a sharper beam of radiation shield the mind during brain tumor treatment?
- Can blood tests reveal how liver and bile duct cancers respond to treatment?
- Can a smart drug home in on Hard-to-Treat GI cancers?