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Neurofibromatosis type 1

MONDO:0018975

A 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

109 clinical trials for this condition and its sub-types.

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Sub-types

Chromosome 17q11.2 deletion syndrome, 1.4Mb (0) Neurofibromatosis, familial spinal (0) Neurofibromatosis type 1 due to NF1 mutation or intragenic deletion (0)

Broader categories

Disease (618) Nervous system disorder (217) Hereditary disease (172) Inherited disease susceptibility (69) Hereditary neoplastic syndrome (57) Syndromic disease (24) Neurofibromatosis (18) Human disease (14) RASopathy (9) Developmental defect during embryogenesis (7)

Trials to join now! 47 Not yet recruiting 6 Not yet finished but already full! 34 Completed 21 Terminated 1

Experimental cancer drug study halted early

Disease control Terminated

This early-stage study tested a new drug called PF-07284892, alone or with other medicines, in people with advanced solid tumors that had specific genetic changes. The goal was to find the safest dose and check for side effects. The study was stopped early, so results are limited…

Phase: PHASE1 • Sponsor: Pfizer • Aim: Disease control

Last updated Jun 27, 2026 13:02 UTC