Targeted drug trial aims at rare cancer mutations

NCT ID NCT04439357

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase II trial tests the drug trametinib in people whose advanced cancers have specific genetic changes called GNAQ or GNA11 mutations. The drug works by blocking certain proteins that help cancer cells grow. Researchers want to see if trametinib can shrink these tumors or stop them from growing. Only 4 patients were enrolled, so results are very preliminary.

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, brand names Mekinist, Meqsel, Spexotras)

What this could lead to

If it works, this could point toward a treatment option for cancers with specific genetic mutations (GNAQ or GNA11).

What could go wrong

This is a very small, early-phase study (only 4 participants) and may not show clear benefit. The drug may cause side effects like heart rhythm issues or not shrink tumors as hoped.

Disclaimer Read more

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.

cancer glioblastoma hematopoietic and lymphoid cell neoplasm hematopoietic and lymphoid system neoplasm lymphoma plasma cell myeloma refractory hematologic cancer refractory malignant neoplasm refractory plasma cell neoplasm

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