Testing a new medication for rare childhood brain tumors

Plain-English translation of NCT06838676 on ClinicalTrials.gov ↗ · Source last updated May 2026 · Translation generated June 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 trial is testing whether , an oral medication taken by mouth twice daily, can help treat children and young adults with rare, aggressive brain tumors called diffuse intrinsic pontine gliomas and certain other high-grade gliomas. The study will track whether the medication slows tumor growth or improves outcomes in patients who have newly diagnosed tumors or tumors that have returned or stopped responding to previous treatments. If the medication is working, patients can continue taking it for up to about 2 years.

These rare brain tumors are very difficult to treat and have very poor outcomes with current therapies. Researchers believe this medication may work differently than standard treatments to help control tumor growth in patients who have limited options.

Do you qualify?

You likely qualify if…

you are between 12 months and 39 years old
you have been diagnosed with a diffuse intrinsic pontine glioma (newly diagnosed, or one that has returned or stopped responding to treatment) or a specific type of high-grade glioma with H3K27 changes
you have completed radiation therapy within the required timeframe (if newly diagnosed) or have measurable disease on MRI (if previously treated)
you have a performance level score of 50% or higher, meaning you are well enough to care for yourself and participate in daily activities
you have not received other anti-cancer medications before (for newly diagnosed patients), or you meet specific prior treatment requirements (for previously treated patients)

You likely don't qualify if…

you are younger than 12 months or older than 39 years old
you have received other anti-cancer drugs besides standard radiation, surgery, or certain approved medications (for newly diagnosed patients)
you are too weak or unwell to participate in the study (performance score below 50%)
your tumor type does not match the specific genetic changes the study is looking for
you cannot start the medication within the required time window after completing radiation therapy (for newly diagnosed patients)

What participation looks like

If you join this trial, you will take the medication by mouth twice each day for 28-day treatment cycles. Your doctors will monitor you with regular MRI scans and blood tests to check how you are responding. If the medication is helping and you are tolerating it well, you can continue treatment for up to 26 cycles over approximately 2 years, or until your tumor starts growing again. You will have regular visits with your treatment team to track your progress and manage any side effects.

AI-generated summary from trial data · Jun 19, 2026 · Not medical advice

Trial locations(20 sites)

United States

Children's Hospital Colorado, Aurora, Colorado

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

Nicklaus Children's Hospital, Miami, Florida

Emory University/Children's Healthcare of Atlanta, Atlanta, Georgia

C.S. Mott Children's Hospital, Ann Arbor, Michigan