Targeted drug shows promise for rare NF2-Mutated cancers
NCT ID NCT04439331
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase II trial is testing the drug defactinib in 35 patients whose advanced cancers have a specific genetic change called an NF2 mutation. The drug works by blocking a protein that may help these cancer cells grow. Researchers want to see if defactinib 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
- Defactinib (VS-6063), a drug that blocks a protein called FAK, which may help stop cancer cell growth.
- What this could lead to
- If it works, this could point toward a treatment option for cancers with NF2 mutations, which are often hard to treat.
- What could go wrong
- This is a small, early-phase trial with only 35 participants. The drug may not shrink tumors or improve survival, and side effects are possible.
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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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