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Kinase Inhibitor

Selumetinib for Pediatric Brain Cancer

Phase 1 & 2
Waitlist Available
Led By Jason R Fangusaro
Research Sponsored by National Cancer Institute (NCI)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
- > 15 years: 1.5 mg/dL
Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 ml/min/1.73m^2 or a serum creatinine based on age as follows (within 14 days of registration and within 7 days of the start of treatment):
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at 1 year
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new drug to treat young patients with brain cancer that has come back or does not respond to treatment. The drug may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
Selumetinib may cause side effects such as fatigue, rash, nausea, vomiting, diarrhea, abdominal pain, visual changes, muscle weakness, heart issues (like hypertension), blood count abnormalities which could lead to increased risk of infections or bleeding.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My kidney function test result is better than 1.5 mg/dL.
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My kidney function tests are within the required range.
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I am over 18 and my blood pressure is below 140/90 mm Hg.
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I am not pregnant or breastfeeding and have a negative pregnancy test.
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I can swallow pills.
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My kidney function is within the required range for my age.
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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.
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My cancer can be measured in two different directions.
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My child's blood pressure is within the normal range for their age, height, and gender.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at 1 year
This trial's timeline: 3 weeks for screening, Varies for treatment, and at 1 year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Disease stabilization rates (re-treatment study)
Maximum tolerated dose and recommended phase 2 dose of selumetinib determined by dose-limiting toxicities (phase I)
Objective response (objective response = complete response + partial response) (re-treatment study)
+1 more
Secondary study objectives
Plasma drug concentrations and pharmacokinetic parameters (Phase I)
Presence or absence of BRAF V600E mutations or BRAF KIAA1549 fusion (phase II)
Progression-free survival (retreatment study)
+1 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

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.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Biospecimen Collection
2004
Completed Phase 3
~2030
Selumetinib
2010
Completed Phase 2
~2080

Find a Location

Who is running the clinical trial?

National Cancer Institute (NCI)Lead Sponsor
13,957 Previous Clinical Trials
41,111,867 Total Patients Enrolled
Jason R FangusaroPrincipal InvestigatorPediatric Brain Tumor Consortium
2 Previous Clinical Trials
319 Total Patients Enrolled

Media Library

Selumetinib (Kinase Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT01089101 — Phase 1 & 2
Low Grade Glioma Research Study Groups: Treatment (selumetinib)
Low Grade Glioma Clinical Trial 2023: Selumetinib Highlights & Side Effects. Trial Name: NCT01089101 — Phase 1 & 2
Selumetinib (Kinase Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01089101 — Phase 1 & 2
~13 spots leftby Dec 2025