Neurofibromatosis type 1
MONDO:0018975A clinically heterogeneous, neurocutaneous genetic disorder characterized by cafe-au-lait spots, iris Lisch nodules, axillary and inguinal freckling, and multiple neurofibromas.
Also known as: neurofibromatosis, NF1, Neurofibromatosis 1, Nf1-Microdeletion syndrome, neurofibromatosis 1, neurofibromatosis type 1, neurofibromatosis type i, neurofibromatosis, type 1
107 clinical trials for this condition and its sub-types, 73 tagged with Neurofibromatosis type 1 itself.
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Sub-types of Neurofibromatosis type 1
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Neurofibromatosis, familial spinal 0 trials
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Can a Two-Drug combo stop a rare, aggressive nerve tumor?
Disease control Not yet recruitingThis phase 2 trial is testing whether the combination of avutometinib and defactinib can stop or shrink malignant peripheral nerve sheath tumors (MPNST) that have returned or cannot be removed with surgery. The study enrolls people over 12 years old with relapsed, refractory, met…
Phase 2 • Sponsor: Girish Dhall, MD • Aim: Disease control
Last updated Aug 06, 2026 00:00 UTC
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New drug combo aims to tackle tough brain tumors in NF1 patients
Disease control Not yet recruitingThis phase 2 trial tests the drug mirdametinib, alone or with radiation, in people with NF1-related high-grade gliomas (aggressive brain tumors). It includes three groups: those with recurrent sporadic glioblastoma, newly diagnosed NF1-associated glioblastoma, and other NF1-relat…
Phase 2 • Sponsor: University of Alabama at Birmingham • Aim: Disease control
Last updated Jun 27, 2026 12:06 UTC
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Patients and staff team up to redesign hospital clinics for sensory needs
Knowledge-focused Not yet recruitingThis project at King's College London asks how hospital outpatient departments can work better for people with neurofibromatosis who also have sensory challenges. About 30 patients, carers, and staff from Guy's and St Thomas' NHS Foundation Trust take part in interviews, observat…
Sponsor: King's College London • Aim: Knowledge-focused
Last updated Sep 16, 2026 00:00 UTC
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Can a simple walk test spot motor trouble in kids with NF1?
Knowledge-focused Not yet recruitingThis study looks at how young children with neurofibromatosis type 1 (NF1) walk, to see if simple walking tests can help doctors spot and predict movement problems early. Researchers will measure walking speed and other skills in 56 children under 6 years old. The goal is to crea…
Sponsor: NYU Langone Health • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:30 UTC