Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas

Trial number:
Trial phase:
Study type:
Targeted therapy
Overall status:

Study start date

November, 2021

Scientific title

A Phase III, Multicentre, International Study With a Parallel, Randomised, Double-blind, Placebo-controlled, 2 Arm Design to Assess the Efficacy and Safety of Selumetinib in Adult Participants With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas (KOMET)


A global study to demonstrate the effectiveness of selumetinib in participants with NF1 who have symptomatic, inoperable plexiform neurofibromas.


Adults ≥ 18 years at enrollment with diagnosis of NF1 with symptomatic, inoperable PNAt least one inoperable target PN measurable by volumetric MRI analysis Chronic target PN pain score documented for minimum period during screening period Stable chronic PN pain medication use at enrollment Adequate organ and marrow function


Confirmed or suspected malignant glioma or MPNST (low grade glioma, including optic glioma not requiring systemic therapy or radiation therapy are exempt from this exclusion)History of malignancy except for malignancy treated with curative intent with no known active disease ≥ 5 years before the first dose of study intervention and of low potential risk for recurrence Clinically significant cardiovascular disease, including inherited coronary disease, acute coronary syndrome within 6 months prior to enrollment, uncontrolled angina, symptomatic heart failure, cardiomyopathy, severe valvular heart disease, abnormal LVEF and uncontrolled hypertension Ophthalmological findings/conditions including intraocular pressure > 21 mmHg, RPED/CSR or RVO Prior exposure to MEK inhibitors

Study design

Primary purpose: Treatment, Allocation: Randomized, Intervention model: Parallel Assignment, Masking: Quadruple, Subject masked: Yes, Caregiver masked: Yes, Investigator masked: Yes, Outcomes assessor masked: Yes,


Neurofibromatosis 1, Plexiform Neurofibroma (PN)

Other study ID numbers

D134BC00001; 2020-005607-39

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