Suspended

KOMETSelumetinib for Symptomatic, Inoperable Plexiform Neurofibromas in Adults with NF1

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Study Aim

This study aims to evaluate the effectiveness of Selumetinib in reducing the size of symptomatic, inoperable plexiform neurofibromas in adults with NF1, as measured by volumetric MRI analysis.

What is being tested

Selumetinib

+ Placebo

DrugOther
Who is being recruted

Congenital, Hereditary, and Neonatal Diseases and Abnormalities+16

+ Neoplasms

+ Neoplasms by Histologic Type

Over 18 Years
+5 Eligibility Criteria
See all eligibility criteria
How is the trial designed

Treatment Study

Placebo-ControlledPhase 3
Interventional
Study Start: November 2021
See protocol details

Summary

Principal SponsorAstraZeneca
Last updated: March 24, 2026
Sourced from a government-validated database.Claim as a partner

Study start date: November 19, 2021

Actual date on which the first participant was enrolled.

This study focuses on adult participants who have a condition called Neurofibromatosis Type 1 (NF1) and are dealing with symptomatic, inoperable plexiform neurofibromas (PN). The main goal is to evaluate the effectiveness and safety of a drug named Selumetinib compared to a placebo. This research is important as it aims to find a potential treatment for this specific condition, which currently has limited treatment options. During the study, participants are randomly assigned to receive either Selumetinib or a placebo. The progress is monitored using volumetric MRI scans. The primary outcome being measured is the rate of confirmed partial and complete response by the end of Cycle 16. This response rate is defined as the proportion of participants who experience a significant decrease in the volume of their target PN or its disappearance. An increase in the volume of the target PN by 20% or more compared to the baseline may indicate disease progression.

Official TitleA 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
Principal SponsorAstraZeneca
Last updated: March 24, 2026
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details

145 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

Over 18 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Congenital, Hereditary, and Neonatal Diseases and AbnormalitiesNeoplasmsNeoplasms by Histologic TypeNeoplasms, Nerve TissueNeoplastic Syndromes, HereditaryNervous System DiseasesNervous System NeoplasmsNeurofibromaNeurofibromatosis 1Neuromuscular DiseasesPeripheral Nervous System DiseasesPeripheral Nervous System NeoplasmsNeurofibromatosesNerve Sheath NeoplasmsNeurofibroma, PlexiformNeurodegenerative DiseasesHeredodegenerative Disorders, Nervous SystemNeurocutaneous SyndromesGenetic Diseases, Inborn

Criteria

5 exclusion criteria prevent from participating
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
Show More Criteria

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives

2 intervention groups are designated in this study

50% chance of being blinded to the placebo group

Treatment Groups

Group I

Experimental
Selumetinib

Group II

Placebo
Placebo

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 34 locations

Suspended

Research Site

Gainesville, United StatesOpen Research Site in Google Maps
Suspended

Research Site

Rockville, United States
Suspended

Research Site

St Louis, United States
Suspended

Research Site

Commack, United States
Suspended34 Study Centers