Selumetinib for Pediatric Brain Cancer

Palo Alto (17 mi)

Overseen byJason R Fangusaro

Age: < 65

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: National Cancer Institute (NCI)

No Placebo Group

Breakthrough Therapy

Approved in 2 jurisdictions

Trial Summary

What is the purpose of this trial?This phase I/II trial studies the side effects and the best dose of selumetinib and how well it works in treating or re-treating young patients with low grade glioma that has come back (recurrent) or does not respond to treatment (refractory). Selumetinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Eligibility Criteria

This trial is for young patients aged 3-21 with recurrent or refractory low grade glioma, including various types of brain tumors like astrocytoma. Participants must have specific tumor characteristics confirmed by recent tests and be willing to provide tissue samples for genetic testing.

Inclusion Criteria

My kidney function test result is better than 1.5 mg/dL.

My kidney function tests are within the required range.

I am over 18 and my blood pressure is below 140/90 mm Hg.

I am not pregnant or breastfeeding and have a negative pregnancy test.

I can swallow pills.

My kidney function is within the required range for my age.

I've been treated with PBTC-029 or PBTC-029B for at least 12 courses with stable disease or had a good response but treated for less than 12 courses.

My cancer can be measured in two different directions.

My child's blood pressure is within the normal range for their age, height, and gender.

Treatment Details

The trial is examining the effects and optimal dosage of Selumetinib, a drug intended to inhibit tumor growth by blocking enzymes necessary for cell proliferation. It's designed as a phase I/II study to determine side effects and effectiveness in children and young adults.

1Treatment groups

Experimental Treatment

Group I: Treatment (selumetinib)Experimental Treatment2 Interventions

Patients receive selumetinib PO BID on days 1-28. Cycles repeat every 28 days for up to 26 cycles in the absence of disease progression or unacceptable toxicity. Patients who experience a sustained objective response from selumetinib on the phase I or phase II portions of the trial, and who have completed 2 years of treatment and stopped study drug may be enrolled on the re-treatment study after progression/recurrence. Patients in the re-treatment study may continue treatment indefinitely in the absence of disease progression or unacceptable toxicities. Patients undergo blood sample collection on study.

Selumetinib is already approved in United States, European Union for the following indications:

🇺🇸 Approved in United States as Koselugo for: