UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Pivotal study about efficiency and safety of JTEC-01 made from revertant mosaicism in epidermolysis bullosa
Title of the study (Brief title)	Pivotal study of J-TEC-01 from revertant mosaicism for epidermolysis bullosa
Region	Japan

Condition
Condition	recessive dystrophic epidermolysis bullosa or junctional epidermolysis bullosa
Classification by specialty	Dermatology
Classification by malignancy	Others
Genomic information	YES

Objectives
Narrative objectives1	We evaluate the rate of epithelialization at 4 week after the grafting of JTEC-01 to the ulcer, which is cultured from revertant mosaicism areas in epidermolysis bullosa.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1	Confirmatory
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	the individual rate of epithelialization (%) at 4 week after the graft
Key secondary outcomes

Base
Study type	Interventional

Study design
Basic design	Single arm
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Device,equipment Other
Interventions/Control_1	transplanting of J-TEC-01 from revertant mosaicism to skin ulcer or erosion.
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit	20 years-old <=
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	(1)patients diagnosed as severe recessive dystrophic epidermolysis bullosa or junctional epidermolysis bullosa (2)patients whose causative mutations are identified in COL7A1, COL17A1, LAMA3, LAMB3 or LAMC3. (3)patients who have one or more ulcers to be grafted with J-TEC-01 (4)patients who clinically have skin area with revertant mosaicism resistant to mechanical stimuli. (5)patients who have competence and from which we can obtain document consent (6)20 years of age or older
Key exclusion criteria	(1)patients who have allergy to particular antibiotics (Penicillin, Kanamycin, Amphotericin B) . (2)patients who have allergy to caw, mouse or pig. (3)patients who participate in other clinical trials within 6 month. (4)patients who have malignant tumor or past history of malignancy within 3years (except cutaneous malignancy). (5)those who are pregnant or nursing mum, or patients who are planning pregnancy during the study. (6)patients whom the investigators judge inappropriate as a subject for the study
Target sample size	3

Research contact person
Name of lead principal investigator	Yasuyuki Fujita
Organization	Hokkaido University Graduate School of Medicine
Division name	Department of Dermatology
Address	North 15, West 7, Kita-ku, Sapporo 060-8638, Japan
TEL	+81-11-706-7387
Email	yfujita@med.hokudai.ac.jp

Public contact
Name of contact person	Yasuyuki Fujita
Organization	Hokkaido University Graduate School of Medicine
Division name	Department of Dermatology
Address	North 15, West 7, Kita-ku, Sapporo 060-8638, Japan
TEL	+81-11-706-7387
Homepage URL
Email	yfujita@med.hokudai.ac.jp

Sponsor
Institute	Hokkaido University Hospital
Institute
Department

Funding Source
Organization	National Institute of Biomedical Innovation
Organization
Division
Category of Funding Organization	Japanese Governmental office
Nationality of Funding Organization	Japan

Other related organizations
Co-sponsor
Name of secondary funｄer(s)

Secondary IDs
Secondary IDs	NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions

Other administrative information
Date of disclosure of the study information	2016 Year 01 Month 26 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2015 Year 12 Month 01 Day
Anticipated trial start date	2016 Year 01 Month 25 Day
Last follow-up date	2017 Year 12 Month 31 Day
Date of closure to data entry	2018 Year 03 Month 31 Day
Date trial data considered complete
Date analysis concluded

Related information
URL releasing protocol
Publication of results	Unpublished
URL releasing results
Results
Other related information

Management information
Registered date	2016 Year 01 Month 26 Day
Last modified on	2018 Year 05 Month 17 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000023350