New pill hopes to shrink painful NF1 tumors without surgery

NCT ID NCT06961565

First seen Nov 01, 2025 · Last updated May 13, 2026 · Updated 24 times

Summary

This early-stage study tests a daily pill called PAS-004 in adults with neurofibromatosis type 1 (NF1) who have at least one symptomatic, inoperable nerve tumor (plexiform neurofibroma). The main goals are to find safe doses and understand side effects. The study has two parts: Part A tests different doses, and Part B tests two selected doses for up to 6 months. About 56 participants will take the drug once daily and be monitored with blood tests and MRIs.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Locations

  • Asan Medical Center

    RECRUITING

    Seoul, 05505, South Korea

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Peter MacCallum Cancer Centre

    RECRUITING

    Melbourne, Victoria, 3000, Australia

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Royal North Shore Hospital

    RECRUITING

    St Leonards, New South Wales, 2065, Australia

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Severance Hospital, Yonsei University Health System

    RECRUITING

    Sinchŏn-dong, 6164, South Korea

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • University of Alabama at Birmingham

    RECRUITING

    Birmingham, Alabama, 35294, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

Conditions

Explore the condition pages connected to this study.