Targeted drug takes aim at NF1-Mutated cancers

NCT ID NCT04439318

First seen Jun 24, 2026 · Last updated Jul 17, 2026 · Updated 3 times

Summary

This phase II trial is testing the drug trametinib in 50 people with advanced cancers that have a specific genetic change called an NF1 mutation. Trametinib blocks certain proteins that help cancer cells grow. The goal is to see if the drug can shrink tumors or stop them from getting worse.

What this could mean

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

Active substance
trametinib (a targeted cancer drug, also known as Mekinist)
What this could lead to
If it works, this could point toward a treatment option for people with advanced cancers that have an NF1 mutation.
What could go wrong
This is a small, early-phase trial with only 50 participants, so results may not apply to everyone. The drug may not shrink tumors or could cause side effects like heart rhythm problems.

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

  • ECOG-ACRIN Cancer Research Group

    Philadelphia, Pennsylvania, 19103, United States

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