Testing a new cancer drug for advanced tumors with specific genetic mutations

Plain-English translation of NCT04678648 on ClinicalTrials.gov ↗ · Source last updated June 2026 · Translation generated July 2026 · How we translate trials

Phase 1 — Testing in a small group (usually 20–80 people) to find a safe dose and watch for side effects.

What is this trial?

This study is testing a new cancer medication called RSC-1255, which works by targeting a specific genetic mutation found in many advanced cancers. Researchers want to find the right dose of the medication and learn whether it works better when used alone or combined with another drug called hydroxychloroquine. The trial will involve about 134 patients with advanced cancers that have not responded to standard treatments.

Many patients with advanced cancers have mutations in a gene called RAS, which makes their tumors harder to treat with existing therapies. This trial exists because there are very few treatment options for these patients, and researchers believe this new medication could offer hope by directly targeting the mutated gene.

Do you qualify?

You likely qualify if…

you have been diagnosed with advanced cancer (solid tumor) that has either stopped responding to standard treatments, made you too sick to tolerate standard treatments, or continued to grow despite treatment
your cancer has been confirmed to have a RAS mutation (or you have glioblastoma, regardless of mutation status)
you are 18 years or older
you are in good enough overall health to tolerate a clinical trial, as measured by standard medical performance tests
you have measurable tumors that can be tracked by imaging or lab tests

You likely don't qualify if…

you are currently receiving other cancer treatments at the time you would enroll
you have serious heart disease or abnormal heart rhythm patterns
you have had another cancer (other than your current one) within the past 2 years
you have a history of low potassium levels in your blood that comes and goes
you already have moderate to severe nausea, vomiting, or diarrhea from your cancer before the study starts

What participation looks like

You would take the medication by mouth twice each day. The study has two phases: first, researchers test different doses to find the safest and most effective amount, and then they enroll more patients to confirm how well it works. Depending on which phase you join, you may also try the medication combined with another drug. You would have regular visits for blood tests, imaging scans, and check-ins to see how you're responding and to monitor for side effects.

AI-generated summary from trial data · Jul 6, 2026 · Not medical advice

Trial locations(3 sites)

United States

University of California, Los Angeles (UCLA) Department of Medicine - Hematology/Oncology, Los Angeles, California

Sarah Cannon, SCRI Oncology Partners - Health One, Denver, Colorado

Sarah Cannon, SCRI Oncology Partners, Nashville, Tennessee