Targeted drug trial aims at rare cancer mutations

NCT ID NCT04439357

Ongoing Disease control Sponsor: National Cancer Institute (NCI) Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jul 17, 2026 · Updated 2 times

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.

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.

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.

ADVANCED LYMPHOMA ADVANCED MALIGNANT SOLID NEOPLASM HEMATOPOIETIC AND LYMPHOID CELL NEOPLASM REFRACTORY LYMPHOMA REFRACTORY MALIGNANT SOLID NEOPLASM REFRACTORY MULTIPLE MYELOMA

Contacts and locations

Locations

ECOG-ACRIN Cancer Research Group

Philadelphia, Pennsylvania, 19103, United States

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