Targeted drug trial aims at rare cancer mutations
NCT ID NCT04439357
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.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
-
4 people
The number who actually took part.
- Started
-
Feb 2016
- Expected to finish
-
Jan 2027
An estimate. End dates often move.
- Lead sponsor
-
A government research agency
The lead sponsor is the US National Institutes of Health.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 years and older
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Patients must have met applicable eligibility criteria in the Master MATCH Protocol prior to registration to treatment subprotocol * Patients must have GNAQ or GNA11 mutations, or another aberration, as determined via the MATCH Master Protocol * Patients must have an electrocardiogram (ECG) within 8 weeks prior to treatment assignment and must have NONE of the following cardiac criteria: * Clinically important abnormalities in rhythm, conduction or morphology of resting ECG (e.g. complete left bundle branch block, third degree heart block) * Treatment-refractory hypertension defined as a blood pressure of systolic \> 140 mmHg and/or diastolic \> 90 mmHg which cannot be controlled by anti-hypertensive therapy * Patients must have an echocardiogram (ECHO) or a nuclear study (multigated acquisition scan \[MUGA\] or First Pass) within 4 weeks prior to registration to treatment and must not have a left ventricular ejection fraction (LVEF) \< the institutional lower limit of normal (LLN). If the LLN is not defined at a site, the LVEF must be \> 50% for the patient to be eligible * Patients who previously received monoclonal antibody therapy (e.g., ipilimumab, nivolumab, pembrolizumab and others) must have stopped the prior therapy for 8 or more weeks before starting on trametinib * Patients with glioblastoma must have histologically or radiographically confirmed recurrent or progressive World Health Organization (WHO) grade 4 glioma (glioblastoma). * NOTE: All baseline and post-baseline disease assessments must be performed using contrast-enhanced cranial magnetic resonance imaging (MRI) or contrast-enhanced computed tomography (CT) for subjects who cannot have MRI performed Exclusion Criteria: * Patients with a history of interstitial lung disease or pneumonitis are excluded * Patients must not have known hypersensitivity to trametinib or compounds of similar chemical or biologic composition or to dimethyl sulfoxide (DMSO) * Patients must not have a history or current evidence/risk of retinal vein occlusion (RVO). An eye exam is required at baseline * Patients who previously received prior treatment with other MEK inhibitors (including, but not limited to, trametinib, binimetinib, cobimetinib, selumetinib, RO4987655 \[CH4987655\], GDC-0623 and pimasertib) will be excluded * Patients with uveal melanoma are excluded
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Advanced lymphoma are added.
Genom att skicka in godkänner du våra Användarvillkor
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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a trio of repurposed drugs shrink recurrent brain tumors?
- Can an existing drug boost bevacizumab against recurrent brain cancer?
- One extra radiation dose after standard brain cancer therapy: can it boost the immune system?
- Radioactive tracer lights up Oxygen-Starved brain tumors
- Can a common virus vaccine help fight brain cancer?
- Radioactive tiles target brain tumors in frail patients