Neoplasms

Nervous System Diseases

Diseases and Abnormalities at or Before Birth

All Conditions

Symptoms and General Pathology

Skin and Connective Tissue Diseases

Rare Diseases

Neurofibromatoses

Neurofibromatosis 1

Neurofibroma

Neurofibroma, Plexiform

Nerve Sheath Neoplasms

Neoplasms, Nerve Tissue

Neoplasms by Histologic Type

Syndrome

Neoplastic Syndromes, Hereditary

Neurocutaneous Syndromes

Heredodegenerative Disorders, Nervous System

Neurodegenerative Diseases

Genetic Diseases, Inborn

Peripheral Nervous System Diseases

Neuromuscular Diseases

Nervous System Neoplasms

Peripheral Nervous System Neoplasms

Neurofibromatosis

Neurofibromatosis Type 1

Selumetinib oral capsules (10 mg and 25 mg)

Selumetinib

Placebo oral capsules for Selumetinib masking (10 mg and 25 mg)

Placebo

Alice P. Chen, MD

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.

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)

Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal.

All request will be evaluated as per the AZ disclosure commitment:

https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.

Adverse events are defined according to Common Terminology Criteria for Adverse Events (CTCAE) v5.0

Selumetinib and N-desmethyl selumetinib plasma concentrations assessment

ORR will be defined as the proportion of patients who have a confirmed complete response or confirmed partial response as determined by ICR per REiNS criteria

Difference in mean change from baseline in chronic target PN pain intensity score between Arm A and Arm B, obtained using an NRS-11 scale to assess pain intensity of a target plexiform neurofibroma

DoR will be defined as the time from the date of first documented response (which is subsequently confirmed) until progression by ICR per REiNS criteria or death due to any cause

PFS will be defined as the time from first selumetinib dose until date of disease progression by ICR per REiNS criteria or death due to any cause

TTP is defined as the time from the date of first selumetinib dose until date of disease progression by ICR per REiNS criteria

TTR is defined as the time from date of first selumetinib dose until the date of objective response by ICR per REiNS criteria

Difference in best percentage change from baseline in target PN volume by ICR per REiNS criteria

Difference in change from baseline between Arm A and Arm B

Merck Sharp & Dohme LLC

AstraZeneca

Participants who started selumetinib and discontinued selumetinib before enrollment (the "discontinued" cohort).

Participants who started selumetinib before enrollment and are continuing to receive selumetinib at the time of enrollment (the "continuing" cohort).

Participants who intend to initiate selumetinib within approximately 3 months after enrollment (the "initiating" cohort).

Alexion Pharmaceuticals, Inc.

The purpose of this study is to understand treatment patterns and assess long-term effectiveness and safety outcomes associated with selumetinib treatment as well as to explore clinical and non-clinical factors affecting those outcomes in participants with neurofibromatosis type 1 (NF1) and plexiform neurofibromas (PNs) who were aged 2 to 18 years at the time selumetinib was started in a real-world setting.

This is a US observational registry of pediatric NF1 PN participants treated with selumetinib.

US Selumetinib Registry

Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

Skeletal manifestations may include changes in the angle of spine curvature.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas	NCT04924608	Neurofibromatos... Plexiform Neuro...	Selumetinib Placebo	18 Years -	AstraZeneca
US Selumetinib Registry	NCT05683678	Neurofibromatos... Plexiform Neuro...		2 Years - 18 Years	Alexion Pharmaceuticals, Inc.
US Selumetinib Registry	NCT05683678	Neurofibromatos... Plexiform Neuro...		2 Years - 18 Years	Alexion Pharmaceuticals, Inc.

Spots Global Cancer Trial Database for pns

Every month we try and update this database with for pns cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Spots Global Cancer Trial Database for pns

Every month we try and update this database with for pns cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for pns cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.