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.
Disclaimer
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the original study
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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.
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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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
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Locations
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ECOG-ACRIN Cancer Research Group
Philadelphia, Pennsylvania, 19103, United States