Promising drug may shrink inoperable tumors in kids with rare genetic condition

NCT ID NCT01362803

First seen Sep 30, 2025 · Last updated Apr 28, 2026 · Updated 23 times

Summary

This study tests an experimental drug called selumetinib in children and young adults (ages 3-18) with neurofibromatosis type 1 (NF1) who have nerve tumors that cannot be removed by surgery. The goal is to see if the drug can shrink the tumors or stop them from growing. Participants take the drug twice daily in 28-day cycles and are monitored for side effects and tumor changes.

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Contacts and locations

Locations

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229-3039, United States

  • National Institutes of Health Clinical Center

    Bethesda, Maryland, 20892, United States

Conditions

Explore the condition pages connected to this study.