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Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas | Clinical Trials | Clareo Health

ACTIVE_NOT_RECRUITINGPHASE3

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

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)

NCT04924608•AstraZeneca

View on ClinicalTrials.gov

Summary

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

Detailed Description

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.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

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 (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

Locations (34)

Research Site

Gainesville, Florida, United States

Research Site

Rockville, Maryland, United States

Research Site

St Louis, Missouri, United States

Research Site

Commack, New York, United States

Research Site

Melbourne, Australia

Research Site

St Leonards, Australia

Research Site

Porto Alegre, Brazil

Research Site

Ribeirão Preto, Brazil

Research Site

São Paulo, Brazil

Research Site

Toronto, Ontario, Canada

Key Dates

Start Date

November 19, 2021

Primary Completion Date

August 5, 2024

Completion Date

February 15, 2029

Last Updated

January 12, 2026

Enrollment

145

ACTUAL participants

Conditions

Neurofibromatosis 1Plexiform Neurofibroma (PN)

Interventions

Selumetinib

DRUG

Placebo

OTHER

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: January 12, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial

Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical Neurofibromas

A Decentralized Clinical Trial to Promote Evidence-Based Care for Underserved Patients With Neurofibromatosis 1

Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II

Assessing the Efficacy of Repeat, Monthly Treatments of Deoxycholate for NF1 Associated Cutaneous Neurofibromas (cNFs)

Multi-parametric Biomarker Development to Predict Malignant Conversion in Patients With Neurofibromatosis Type 1