Neurofibromatosis and Glioma Therapy using Selumetinib – Phase III

ACNS1831: A Study of the Drugs Selumetinib versus Carboplatin/Vincristine in Patients with Neurofibromatosis and Low-Grade Glioma

Protocol Description

This multi-center study organized by the Children’s Oncology Group investigates whether a drug called selumetinib works as well as the standard treatment for young people with a genetic disorder called neurofibromatosis type-1 (NF1) and a low-grade glioma (LGG), also called NF1-associated LGG. Selumetinib is a drug that works by blocking a protein that lets cancer cells grow without stopping.

Study Level: Phase III

Eligibility Criteria

Subject to certain exclusion criteria, this study is enrolling patients ages 2 through 21 years diagnosed with NF1-associated LGG.
Males and Females: Ages 2 through 21 years


Participants will be randomly selected to receive therapy with selumetinib taken orally as a capsule, or the standard treatment, which uses a combination of the drugs carboplatin and vincristine given intravenously. Therapy with selumetinib takes about 27 months to complete, and the standard therapy takes about 15 months for most participants.
Visits: Number of visits will vary based on study arm and the participant’s response to protocol.
Duration: In addition to the treatment phase, participants will complete research tests for about 5 years after they enter this study.

Status: Open to Enrollment

Source(s) of Support

Children’s Oncology Group (ACNS1831)
National Cancer Institute

Primary Investigator

James Felker, MD

Contact Information

For more information about the study or enrollment, please contact the UPMC Children’s Hospital of Pittsburgh Neuro-Oncology team, at: