Testing two medications together for recurrent brain tumors in young patients

Plain-English translation of NCT04485559 on ClinicalTrials.gov ↗ · Source last updated August 2026 · Translation generated September 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 whether two medications— and —work better together to treat recurrent brain tumors in children and young adults. The research team wants to find the right dose of this combination, understand what side effects occur, and see how well the treatment works. This is an early-stage trial that will help doctors decide if this combination should be tested more widely.

Brain tumors that come back or continue to grow after initial treatment are very difficult to manage, and current options are limited. This medication combination targets specific pathways involved in tumor growth, and researchers believe using both drugs together might be more effective than treating with one drug alone.

Do you qualify?

You likely qualify if…

you have been diagnosed with a low-grade brain tumor (WHO grade I-II) that has come back or grown after previous treatment, or a high-grade brain tumor (WHO grade III-IV)
you have received prior treatment such as chemotherapy, radiation, or other therapies before joining this trial
you are between infancy and young adulthood (the trial includes pediatric and young adult patients)
you have tumor tissue available for genetic testing, or prior genetic testing results already on file
you have fully recovered from side effects of any previous cancer treatments

You likely don't qualify if…

you have had surgery alone for your brain tumor without any additional treatment
you have severe or serious side effects from previous MEK inhibitors or mTOR inhibitors
you have received chemotherapy within the past three weeks (or six weeks if it was a nitrosourea drug)
you have had radiation to the brain within the past 12 weeks
you have had a bone marrow transplant less than 3–6 months before the trial start date

What participation looks like

You would take the two medications by mouth on a regular schedule—either every day or on an intermittent schedule—with each treatment cycle lasting 28 days. You could receive up to 26 cycles of treatment (about two years) if the tumor responds well and you don't experience unacceptable side effects. You would visit the clinic regularly for blood work, imaging scans (like MRI), and checkups to monitor how you're doing and whether the tumor is shrinking. After treatment ends, you'll have follow-up visits at 30 days, then every three months for a year, then every six months for five years.

AI-generated summary from trial data · Sep 5, 2026 · Not medical advice

Trial locations(17 sites)

United States

University of Alabama at Birmingham, Children's of Alabama, Birmingham, Alabama

Children's Hospital Los Angeles, Los Angeles, California

University of California, San Diego Rady Children's Hospital, San Diego, California

University of California, San Francisco, San Francisco, California

Children's National Medical Center, Washington D.C., District of Columbia