New hope for NF1 patients: drug trial targets painful tumors

NCT ID NCT04924608

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests the drug selumetinib in adults with neurofibromatosis type 1 (NF1) who have painful, inoperable nerve tumors called plexiform neurofibromas. About 145 participants will receive either selumetinib or a placebo to see if the drug can shrink the tumors and reduce pain. The goal is to control the disease and improve quality of life, not to cure it.

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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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Conditions

The condition(s) this trial relates to.

neurofibromatosis type 1 plexiform neurofibroma

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Research Site

    Gainesville, Florida, 32610, United States

  • Research Site

    Rockville, Maryland, 20852, United States

  • Research Site

    St Louis, Missouri, 63156, United States

  • Research Site

    Commack, New York, 11725, United States

  • Research Site

    Melbourne, 3000, Australia

  • Research Site

    St Leonards, 2065, Australia

  • Research Site

    Porto Alegre, 90035-903, Brazil

  • Research Site

    Ribeirão Preto, 14051-140, Brazil

  • Research Site

    São Paulo, 045202-001, Brazil

  • Research Site

    Toronto, Ontario, M5G 2C4, Canada

  • Research Site

    Montreal, Quebec, H4A 3J1, Canada

  • Research Site

    Beijing, 100070, China

  • Research Site

    Beijing, 100730, China

  • Research Site

    Guangzhou, 510060, China

  • Research Site

    Shenyang, 110001, China

  • Research Site

    Créteil, 94000, France

  • Research Site

    Lyon, 69008, France

  • Research Site

    Toulouse, 31059, France

  • Research Site

    Hamburg, 20246, Germany

  • Research Site

    Tübingen, 72076, Germany

  • Research Site

    Würzburg, 97080, Germany

  • Research Site

    Milan, 20133, Italy

  • Research Site

    Naples, 80131, Italy

  • Research Site

    Roma, 00165, Italy

  • Research Site

    Minatoku, 105-8471, Japan

  • Research Site

    Nagoya, 466-8560, Japan

  • Research Site

    Shinjuku-ku, 160-8582, Japan

  • Research Site

    Bydgoszcz, 85-094, Poland

  • Research Site

    Moscow, 115522, Russia

  • Research Site

    Moscow, 125412, Russia

  • Research Site

    Badalona, 08916, Spain

  • Research Site

    Madrid, 28041, Spain

  • Research Site

    London, SE1 9RT, United Kingdom

  • Research Site

    Manchester, M20 4BX, United Kingdom