UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Feasibility study of the treatment of the refractory skin ulcer by the punch graft from the Natural gene therapy area in epidermolysis bullosa
Title of the study (Brief title)	Punch graft from natural gene therapy area in epidermolysis bullosa
Region	Japan

Condition
Condition	epidermolysis bllosa
Classification by specialty	Dermatology
Classification by malignancy	Others
Genomic information	YES

Objectives
Narrative objectives1	In order to evaluate the efficacy and safety of skin transplantation using punch grafts from "natural gene therapy" area in the bullous epidermolysis patients.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2	Others
Developmental phase	Not applicable

Assessment
Primary outcomes	We evaluate the rate of reepithelialization for the 4th week after the last grafting (%)
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	Maneuver Other
Interventions/Control_1	skin transplantation using punch grafting from natural gene therapy area to refractory ulcer
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	6 years-old <=
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	Patients diagnosed with epidermolysis bullosa (junctional or dystrophic type)and fulfills as follows: 1.Patient aged 6 years old and up. 2.Patient whose responsible gene for epidermolysis bullosa has been identified. 3.Patient with the skin area in which a natural gene therapy occur. 4.Patients with intractable skin ulcers of bigger than 5 cm2 in size which last for more than 2 weeks. 5.Patient who agreed with genetic ecamination of the skin. 6.Patients who agreed with written informed consent after satisfactory explanation.
Key exclusion criteria	1.Patient who has participated in other clinical trials in recent 3 months. 2.Patient who is participating or will participate in other clinical trials. 3. Patients with malignancy except skin malignancy. 4.Patients with pregnancy 5.The patient who may suffer an unjust disadvantage by not participating in this research. 6.Other patients who are evaluated not to be suitable for this research
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	011-706-7387
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	011-706-7387
Homepage URL
Email	yfujita@med.hokudai.ac.jp

Sponsor
Institute	Hokkaido University Hospital
Institute
Department

Funding Source
Organization	Department of Dermatology Hokkaido University Graduate School of Medicine
Organization
Division
Category of Funding Organization	Self funding
Nationality of Funding Organization

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	2014 Year 08 Month 20 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2014 Year 02 Month 03 Day
Anticipated trial start date	2014 Year 09 Month 01 Day
Last follow-up date
Date of closure to data entry
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	2014 Year 08 Month 18 Day
Last modified on	2017 Year 02 Month 20 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000017065