臨床研究等提出・公開システム

Date of registration	Dec. 20, 2018
Last modified on	April. 02, 2021
Trial ID	jRCT1080224498

Scientific Title	A clinical study of CL2020 in patients with epidermolysis bullosa
Public Title	A clinical study of CL2020 in patients with epidermolysis bullosa

Completion Date or Terminated date	July. 14, 2020
Actual Enrolled Number	5
Baseline Characteristics	Five patients [one male and four female, ages 17-49 (median:22)]
Participant flow
Adverse events	stomach pain (Grade 3), acquired lacrimal stricture (G2), fever (G1), gastroenteritis (G1), upper respiratory tract infection (G1) and paraesthesia of the upper arms (G1). One patient showed paraesthesia within 24 h after infusion and resolved in 14 days, which was considered a possible CL2020-related side-effect.
Primary Outcome Measures	All cases showed mild or self-limiting adverse effects.
Secondary Outcome Measures	The change in the total size of the selected ulcers was -9.98 (95% CI: -17.87 to -2.09) cm2 at Wk04, and statistically significant improvement was found(P = 0.017).
Brief summary	CL2020 administration is a well-tolerated therapy and Muse cells are a potentially promising regenerative medicine for adults with severe EB.
Posting of journal publication
Date of the first journal publication of results	Mar. 15, 2021
URL hyperlink(s) related to results and publications	https://onlinelibrary.wiley.com/doi/10.1111/jdv.17201

Plan to share IPD	No
Plan description
URL link to protocol file(s)
Version of protocol file(s)	version: date:

Contact for Scientific Queries	Name	Fukushima Tatsunobu
	Contact Name for Scientific Queries	Life Science Institute Inc.
	Address	1-1 Marunouchi 1-chome, Chiyoda-ku, Tokyo 100-8251
	Phone Number	+81-3-6748-7200
	E-mail Address	lsii.chiken-inq@eppharmaline.co.jp

Contact for Public Queries	Name	call center
	Contact Name for Public Queries	Life Science Institute Inc.
	Address	1-1 Marunouchi 1-chome, Chiyoda-ku, Tokyo 100-8251
	Phone Number	+81-3-6748-7200
	E-mail Address	lsii.chiken-inq@eppharmaline.co.jp

Recruitment Status	completed

Date of First Enrolment		Dec. 27, 2018
Target Sample Size		5
Study Type		Interventional
Study Design	Study Design	Open label, non-randomized
Study Design	Objective	treatment purpose
Phase		2
Key inclusion & exclusion criteria	Inclusion Criteria	Major inclusion criteria Patients with epidermolysis bullosa who have any intractable ulcers Patients aged 12 to 75 years Female or male patients
	Exclusion Criteria	Major exclusion criteria Patients with any existing or previous malignant tumor Female patients who are or may be pregnant or who are breastfeeding
	Minimum Age	12age old over
	Maximum Age	75age old under
	Gender	Both
Health Condition or Problem Studied		Epidermolysis bullosa
Intervention(s)		investigational material(s) Generic name etc : CL2020 INN of investigational material : - Therapeutic category code : 49- Other agents affecting cellular function Dosage and Administration for Investigational material : Intravenous infusion control material(s) Generic name etc : - INN of investigational material : - Therapeutic category code : Dosage and Administration for Investigational material : -
Health Condition(s) Keyword
Intervention(s) Keyword
Health Condition(s) Code
Intervention(s) Code
Primary Outcome		safety Safety after infusion of CL2020
Secondary Outcome		efficacy efficacy after infusion of CL2020

Primary Sponsor	Life Science Institute, Inc.
Secondary Sponsor	-

Name of Funding Organization	-
Name of Research Funding	-

Name of IRB	Review Board of Toho University Omori Medical Center
Address of IRB	6-11-1,Omori-Nishi,Ota-ku,Tokyo 143-8541,Japan
Phone Number of IRB	-
E-mail Address of IRB	-
Status of IRB Review	approved
Date of Approval by IRB	July. 24, 2018

Secondary ID No.	JapicCTI-184563
Name of Other Registries

Countries / Regions of Recruitment	Japan

History of Changes

No	Publication date
4	April. 02, 2021	(this page)	Changes
3	Aug. 06, 2020	Detail	Changes
2	July. 11, 2019	Detail	Changes
1	Dec. 27, 2018	Detail

List of change history