KOMET
Research type
Research Study
Full 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 Plexiforn Neurofibromas (KOMET)
IRAS ID
1003834
Contact name
Urvi Savani
Contact email
Sponsor organisation
AstraZeneca AB
Eudract number
2020-005607-39
Research summary
KOMET study is a Phase III, multicentre, international study with a parallel, randomized, double-blind, placebo-controlled, 2-arm design to assess the efficacy and safety of selumetinib in adult participants with NF1 who have symptomatic, inoperable PN.
Rationale: Selumetinib has been shown to be effective in the treatment of paediatric patients with NF1 who have symptomatic, inoperable PN and is approved in the US for the treatment of paediatric patients 2 years of age and above. Plexiform neurofibromas can continue to manifest through late adolescence and early adulthood and often associated with significant clinical symptoms including pain and motor dysfunction. PN shrinkage and spontaneous resolution of symptoms associated with PN has been shown to be extremely unlikely, therefore adults with NF1-PN continue to have a significant unmet need.
Patients 18 years of age and older who have NF-1 with symptomatic, inoperable PN will be randomized 1:1 into study to receive selumetinib or placebo. A single target PN will be selected by the Investigator, and is defined as the clinically most relevant PN, which has to be measurable by volumetric MRI analysis. As PNs are often associated with multiple co-morbidities such as pain and motor dysfunction, these elements will be assessed using various PRO tools. At Cycle 8, any patients on placebo arm will automatically cross over to the selumetinib arm. The study will take place for approximately 3 years.
REC name
South West - Central Bristol Research Ethics Committee
REC reference
21/SW/0112
Date of REC Opinion
19 Oct 2021
REC opinion
Further Information Favourable Opinion