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New hope for NF1 patients: targeted drug shrinks inoperable tumors in early trial

NCT ID NCT04590235

First seen Nov 01, 2025 · Last updated May 23, 2026 · Updated 30 times

Summary

This early-stage study tests the safety and effectiveness of selumetinib, a drug that blocks a key cancer growth signal, in 32 Chinese children and adults with neurofibromatosis type 1 (NF1) who have inoperable plexiform neurofibromas (nerve tumors). Participants take the drug by mouth, and researchers monitor side effects, drug levels in the blood, and tumor shrinkage using MRI. The goal is to see if the drug can control tumor growth, but lifelong management may still be needed.

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Contacts and locations

Locations

  • Research Site

    Shanghai, 200011, China

  • Research Site

    Shanghai, CN-200092, China

Conditions

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