New hope for NF1 patients: drug trial targets painful tumors

NCT ID NCT04924608

Ongoing Disease control Sponsor: AstraZeneca Source: ClinicalTrials.gov ↗

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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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.

NEUROFIBROMATOSIS 1 PLEXIFORM NEUROFIBROMA (PN)

Contacts and locations

Locations

Research Site

Gainesville, Florida, 32610, United States
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Rockville, Maryland, 20852, United States
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St Louis, Missouri, 63156, United States
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Commack, New York, 11725, United States
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Melbourne, 3000, Australia
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St Leonards, 2065, Australia
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Porto Alegre, 90035-903, Brazil
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Ribeirão Preto, 14051-140, Brazil
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São Paulo, 045202-001, Brazil
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Toronto, Ontario, M5G 2C4, Canada
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Montreal, Quebec, H4A 3J1, Canada
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Beijing, 100070, China
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Beijing, 100730, China
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Guangzhou, 510060, China
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Shenyang, 110001, China
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Créteil, 94000, France
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Lyon, 69008, France
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Toulouse, 31059, France
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Hamburg, 20246, Germany
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Tübingen, 72076, Germany
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Würzburg, 97080, Germany
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Milan, 20133, Italy
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Naples, 80131, Italy
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Roma, 00165, Italy
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Minatoku, 105-8471, Japan
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Nagoya, 466-8560, Japan
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Shinjuku-ku, 160-8582, Japan
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Bydgoszcz, 85-094, Poland
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Moscow, 115522, Russia
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Moscow, 125412, Russia
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Badalona, 08916, Spain
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Madrid, 28041, Spain
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London, SE1 9RT, United Kingdom
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Manchester, M20 4BX, United Kingdom

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