UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	A Clinical Study to Evaluate the Efficacy and Safety of JR-031 in Patients with Epidermolysis Bullosa
Title of the study (Brief title)	A Clinical Study to Evaluate the Efficacy and Safety of JR-031 in Patients with Epidermolysis Bullosa
Region	Japan

Condition
Condition	Epidermolysis Bullosa
Classification by specialty	Dermatology
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	To evaluate efficacy and safety of JR-031 in patients with Epidermolysis Bullosa
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2	Explanatory
Developmental phase	Phase II

Assessment
Primary outcomes	Rate of patients number who show 50% or more improvement in the size of cutaneous ulcer after 48 week treatment
Key secondary outcomes	1) Changes from baseline in the size of cutaneous ulcer after 1,2,4,8,16,24,32,40,48 week treatment 2) Rate of patients number who show 50% or more improvement in the size of cutaneous ulcer after 1,2,4,8,16,24,32,40 week treatment

Base
Study type	Interventional

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

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Other
Interventions/Control_1	JR-031 is subcutaneously administered at the dose of 0.5 million cells /250 micro L /site every two centimeters around cutaneous ulcers. The maximum number of injection sites is 40 and the maximum dose is 20 million cells /man.
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	70 years-old >
Gender	Male and Female
Key inclusion criteria	1) Patients diagnosed as Epidermolysis Bullosa 2) Patients who have skin ulcers which last more than 12 months 3) Patients who is 6 years old or elder and younger than 70 years old
Key exclusion criteria	1) Patients who received a therapy with mesenchymal stem cell derived from human bone marrow or TEMUCELL HS injection. 2) Patients who are suffering from serious mental diseases. 3) Patients who are suffering from cancer or who have histories of suffering from cancer within 5 years prior to enrollment. 4) Patients who are pregnant or probably pregnant. 5) Patients who do not agree to take effective contraceptive measures during the study period. 6) Patients who have entered other clinical trials within 6 months prior to enrollment.
Target sample size	6

Research contact person
Name of lead principal investigator	Katsuto Tamai
Organization	Graduate School of Medicine, Osaka University
Division name	Department of Stem Cell Therapy Science
Address	2-2, Yamadaoka, Suita, Osaka 565-0871, Japan
TEL	06-6210-8396
Email	tamai@gts.med.osaka-u.ac.jp

Public contact
Name of contact person	Katsuto Tamai
Organization	Graduate School of Medicine, Osaka University
Division name	Department of Stem Cell Therapy Science
Address	2-2, Yamadaoka, Suita, Osaka 565-0871, Japan
TEL	06-6210-8396
Homepage URL
Email	tamai@gts.med.osaka-u.ac.jp

Sponsor
Institute	Osaka University
Institute
Department

Funding Source
Organization	Japan Agency for Medical Research and Development
Organization
Division
Category of Funding Organization	Japanese Governmental office
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	2017 Year 03 Month 24 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2017 Year 02 Month 28 Day
Anticipated trial start date	2017 Year 04 Month 01 Day
Last follow-up date	2018 Year 06 Month 28 Day
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	2017 Year 03 Month 22 Day
Last modified on	2018 Year 08 Month 01 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000030601