Plexiform Neurofibromas

• CLINICAL_TRIALS research · 2026-04-24
  Clinical Trial Update: Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)

  This clinical trial update describes the development and validation of patient-reported outcome measures for Neurofibromatosis type 1 and pl…

• CLINICAL_TRIALS trial_result · 2026-03-05
  Clinical Trial Update: MEK 1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas [Phase 2]

  Phase 2 trial update for the MEK inhibitor selumetinib in adults with Neurofibromatosis Type 1 and inoperable plexiform neurofibromas; the s…

• FDA approval · 2025-09-10
  FDA Approved: KOSELUGO — Original Application (AstraZeneca Pharmaceuticals LP)

  FDA approved KOSELUGO (selumetinib) as an original application for pediatric patients with neurofibromatosis type 1 and inoperable plexiform…

• CLINICAL_TRIALS other · 2025-04-04
  Clinical Trial Update: US Selumetinib Registry

  The US Selumetinib registry trial for Neurofibromatosis Type 1 with plexiform neurofibromas has been terminated.

• CLINICAL_TRIALS trial_result · 2023-05-24
  Clinical Trial Update: Cabozantinib for Plexiform Neurofibromas (PN) in Subjects With NF1 in Children and Adults [Phase 2]

  Phase 2 trial evaluating cabozantinib for plexiform neurofibromas in NF1 patients (children and adults) has completed with results posted in…

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• CLINICAL_TRIALS trial_result · 2021-12-06
  Clinical Trial Update: Acceptance and Commitment Training for Adolescents and Young Adults With Neurofibromatosis Type 1, Plexiform Neurofibromas, and Chronic Pain [Phase 3]

  Phase 3 trial evaluating Acceptance and Commitment Training for adolescents and young adults with Neurofibromatosis Type 1 and plexiform neu…

• CLINICAL_TRIALS trial_result · 2021-07-14
  Clinical Trial Update: Study to Investigate Safety, Pharmacokinetic (PK), Pharmacodynamic (PD) and Clinical Activity of Trametinib in Subjects With Cancer or Plexiform Neurofibromas and Trametinib in Combination With Dabrafenib in Subjects With Cancers Harboring V600 Mutations [Phase 1, Phase 2]

  Phase 1/2 study evaluating trametinib alone or with dabrafenib in cancers with V600 mutations or plexiform neurofibromas; completed with res…