Testing a new pill for rare tumors linked to specific genetic changes

Plain-English translation of NCT04924075 on ClinicalTrials.gov ↗ · Source last updated July 2026 · Translation generated July 2026 · How we translate trials

Phase 2 — Testing in a bigger group (up to a few hundred people) to see if the treatment actually works and is still safe.

What is this trial?

This study is testing a new medication called to see if it can help people with rare tumors—including pheochromocytomas, paragangliomas, pancreatic neuroendocrine tumors, and certain other cancers linked to genetic changes. The medication works by targeting a specific pathway in cells that helps these tumors grow. Researchers want to know if taking this treatment by mouth each day can slow or shrink these tumors.

Many of these rare tumors are hard to treat and can grow quickly. This medication is designed to attack a genetic weakness that many of these tumors share, offering hope for people who have limited treatment options today.

Do you qualify?

You likely qualify if…

you are at least 12 years old (18 or older for some specific tumor types)
you have been diagnosed with an advanced or metastatic rare tumor including pheochromocytoma, paraganglioma, pancreatic neuroendocrine tumor, gastrointestinal stromal tumor, or a solid tumor with a specific genetic change (HIF-2α related)
you have a life expectancy of at least 3 months
you are able to swallow oral medication without difficulty
you have not received certain cancer treatments within the past 4 weeks

You likely don't qualify if…

you have trouble swallowing pills or absorbing oral medications
you have a history of another cancer in the past 2 years
you have low oxygen levels at rest or require supplemental oxygen
you have serious heart disease or have had recent heart surgery or procedures
you are currently taking certain other cancer treatments (except for specific hormone therapies)

What participation looks like

You would take a daily pill of this medication by mouth for as long as it is helping and you are tolerating it well. The research team will monitor you regularly with blood tests and imaging scans to measure whether the treatment is working and check for any side effects. Your participation continues until your tumor stops responding to the treatment or you decide to stop.

AI-generated summary from trial data · Jul 16, 2026 · Not medical advice

Testing a new pill for rare tumors linked to specific genetic changes

What is this trial?

Do you qualify?

What participation looks like

Trial locations