Recruiting

Testing a new cancer drug for tumors with specific genetic changes

Plain-English translation of NCT06004245 on ClinicalTrials.gov ↗ · Source last updated July 2026 · Translation generated June 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 is an early-stage study testing whether , a new oral medication, can help treat advanced cancers that have specific genetic characteristics (microsatellite instability or mismatch repair defects). Researchers believe this medication works by targeting a protein called Werner that may help these particular cancers grow. The study will test the medication on its own and also in combination with two established cancer treatments.

Many patients with advanced cancers that have these specific genetic features have limited treatment options after standard therapies stop working. This medication was designed to specifically target the genetic vulnerabilities in these cancers, potentially offering a new option for people who need it most.

Do you qualify?

You likely qualify if…

you have an advanced solid tumor that has been diagnosed with microsatellite instability or mismatch repair defects
your cancer has progressed despite or you couldn't tolerate standard treatments
you are healthy enough to participate, with a good performance status
you have measurable disease visible on imaging scans
you are expected to live at least 12 weeks
you have a tumor tissue sample available for testing

You likely don't qualify if…

you cannot swallow pills or have problems absorbing medications
you have uncontrolled cancer that has spread to your brain or spinal fluid
you have an active, untreated infection or recent serious infection
you have untreated hepatitis B, hepatitis C, or HIV
you have been diagnosed with Werner syndrome or previously received a different Werner-targeting drug

What participation looks like

You would take the medication by mouth daily and visit the clinic regularly for check-ups, blood tests, and imaging scans to monitor how the cancer is responding and how well you're tolerating the treatment. Depending on which study group you're assigned to, you might receive this medication alone or combined with another established cancer drug. The study will involve blood draws, tissue biopsies, and scans to track your progress over time.

AI-generated summary from trial data · Jun 11, 2026 · Not medical advice

Testing a new cancer drug for tumors with specific genetic changes

What is this trial?

Do you qualify?

What participation looks like

Trial locations(38 sites)