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)

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2023-507336-20-00

Acronym

KOMET D134BC00001

Enrollment

Registered

2024-05-21

Start date

2021-12-08

Completion date

Unknown

Last updated

2025-12-17

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Inoperable Plexiform Neurofibromas (PN)., Neurofibromatosis Type 1 (NF1) with Symptomatic

Brief summary

Objective Response Rate (ORR) using volumetric MRI analysis as determined by ICR per REiNS criteria.

Detailed description

Change from baseline in chronic target PN pain intensity. Change from baseline in PlexiQoL total score. Objective Response Rate. Duration of Response. Progression Free Survival. Time to progression. Time to Response. Best percentage change from baseline in target PN vol. Pain palliation, pain medication use, pain interference, physical functioning, health related quality of life PROs&health status. Safety&tolerability. Plasma concentrations&PK parameters of selumetinib&Ndesmethyl selumetinib.

Interventions

DRUGSELUMETINIB

DRUGPlacebo to match Selumetinib capsules

DRUG10 and 25 mg.

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Objective Response Rate (ORR) using volumetric MRI analysis as determined by ICR per REiNS criteria.	—

Secondary

Measure	Time frame
Change from baseline in chronic target PN pain intensity. Change from baseline in PlexiQoL total score. Objective Response Rate. Duration of Response. Progression Free Survival. Time to progression. Time to Response. Best percentage change from baseline in target PN vol. Pain palliation, pain medication use, pain interference, physical functioning, health related quality of life PROs&health status. Safety&tolerability. Plasma concentrations&PK parameters of selumetinib&Ndesmethyl selumetinib.	—

Measure

Time frame

—

Countries

France, Germany, Italy, Poland, Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026