Testing a New Oral Medication for Brain and Spinal Cord Tumors

Plain-English translation of NCT04541082 on ClinicalTrials.gov ↗ · Source last updated December 2025 · 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.

This is one of the first times this treatment has been tested in people.

What is this trial?

This is an early-stage study testing a new medication called in patients with brain and spinal cord tumors that have come back after previous treatments. Researchers will gradually increase the dose in small groups of patients to find the safest and most effective dose. The study is designed for adults with many different types of primary brain and spinal cord tumors, including glioblastoma, ependymoma, medulloblastoma, and meningioma.

Many patients with recurring brain and spinal cord tumors run out of standard treatment options. This medication is being tested because early laboratory work suggests it may help fight these difficult-to-treat cancers in a new way.

Do you qualify?

You likely qualify if…

you are 18 years or older
you have been diagnosed with a primary brain or spinal cord tumor that has come back after prior treatment
your tumor has been confirmed by a pathologist (biopsy or surgery)
you have already received standard treatments for your tumor type and have no other standard options available
your overall health and ability to care for yourself is good enough to tolerate the study (a score of 70 or higher on a standard scale)
you are willing to wait at least 14 to 42 days since your last cancer treatment before starting this medication, depending on which treatment you received

You likely don't qualify if…

you have received certain cancer drugs or radiation within the last 2 to 6 weeks (depending on the drug)
you are still experiencing moderate to severe side effects from your previous cancer treatments
you have received this medication as part of a previous clinical trial within certain timeframes
you are unable to swallow oral medication or have severe stomach problems that would prevent absorption
you are pregnant or breastfeeding

What participation looks like

You will take the medication by mouth, and you'll start at a low dose that may be increased over time as the study progresses and safety is confirmed. You'll visit the study center regularly for blood tests, scans, and check-ups to monitor how the medication is working and watch for any side effects. The study is expected to continue for several months or longer, and researchers will carefully track your health throughout.

AI-generated summary from trial data · Jul 3, 2026 · Not medical advice

Trial locations(1 site)

United States

National Institutes of Health, Bethesda, Maryland