臨床研究等提出・公開システム

Japanese

Date of registration	June. 24, 2021
Last modified on	May. 24, 2023
Trial ID	jRCT2041210036

Scientific Title	A global study to demonstrate the effectiveness of selumetinib in participants with NF1 who have symptomatic, inoperable plexiform neurofibromas. (KOMET)
Public 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 Plexiform Neurofibromas

Contact for Scientific Queries	Name	Kobayashi Hiroshi
	Affiliation	Alexion Pharma GK
	Address	Tamachi Station Tower N, 3-1-1, Shibaura, Minato-ku, Tokyo 108-0023, Japan
	Telephone	+81-3-3457-9559
	E-mail	hiroshi.kobayashi@alexion.com
Contact for Public Queries	Name	Kobayashi Hiroshi
	Affiliation	Alexion Pharma GK
	Address	Tamachi Station Tower N, 3-1-1, Shibaura, Minato-ku, Tokyo 108-0023, Japan
	Telephone	+81-3-3457-9559
	E-mail	hiroshi.kobayashi@alexion.com

Recruitment status	Not Recruiting

Anticipated date of first enrollment		Oct. 19, 2021
Actual date of first enrollment		July. 01, 2022
Target sample size		15
Study Type		Interventional
Study Design	allocation	randomized controlled trial
	masking	double blind
	control	placebo control
	assignment	parallel assignment
	purpose	treatment purpose
Key inclusion & exclusion criteria	Inclusion Criteria	- Adults 18 years or more 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 (optic glioma not requiring chemotherapy 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 or more 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
	Age Minimum	18age old over
	Age Maximum	No limit
	Gender	Both
Health Condition(s) or Problem(s) Studied		Neurofibromatosis 1, Plexiform Neurofibroma (PN)
Intervention(s)		Arm A: selumetinib 25 mg/m2 orally bid Arm B: placebo orally bid
Health Condition(s) Keyword
Intervention(s) Keyword
Health Condition(s) Code
Intervention(s) Code
Primary Outcome(s)		Confirmed Objective Response Rate (ORR) for Arm A [ Time Frame: Approximately 3 years ] 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
Secondary Outcome(s)

Primary Sponsor	Alexion Pharma GK

Source of Monetary Support / Secondary Sponsor
Secondary Sponsor

Source of Monetary Support
Secondary Sponsor

Name of Certified Review Board	Nagoya University Hospital Institutional review board
Address	65 Tsurumai-cho, Showa-ku, Nagoya, Aichi
Telephone	+81-52-744-1958
E-Mail	center@med.nagoya-u.ac.jp
Approval Status	Approval
Date of approval	Oct. 26, 2021

Plan to share IPD	No
Plan description

Secondary ID(s)	NCT04924608
Issuing Authority	ClinicalTrials.gov

Countries of Recruitment（Except Japan）	Australia/Brasil/Canada/China/France/Germany/Italy/Poland/Russia/Spain/USA/UK

History of Changes

No	Publication date
6	May. 24, 2023	(this page)	Changes
5	Oct. 07, 2022	Detail	Changes
4	July. 29, 2022	Detail	Changes
3	July. 14, 2022	Detail	Changes
2	June. 09, 2022	Detail	Changes
1	June. 24, 2021	Detail

List of change history