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

  • End date
    Nov 22, 2024
  • participants needed
  • sponsor
Updated on 1 December 2021


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


This is a randomized, double-blind, placebo-controlled, 2 arm multicentre, global Phase III study to assess the efficacy and safety of selumetinib compared with placebo in adult participants with NF1 who have symptomatic, inoperable PN.

Condition Neurofibromatosis, Plexiform Neurofibroma, von Recklinghausen's Disease, Peripheral Neuropathy
Treatment Placebo, Selumetinib
Clinical Study IdentifierNCT04924608
Last Modified on1 December 2021


Yes No Not Sure

Inclusion Criteria

Adults 18 years at enrollment with diagnosis of NF1 with symptomatic, inoperable PN
At 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

Exclusion Criteria

Confirmed or suspected malignant glioma or MPNST (optic glioma not requiring chemotherapy 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
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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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