UMIN-CTR Clinical Trial

Basic information
Public title	A clinical study to evaluate efficacy, safety and tolerability of ISN001 in dystrophic epidermolysis bullosa patients.
Acronym	A clinical study to evaluate efficacy, safety and tolerability of ISN001 in dystrophic epidermolysis bullosa patients.
Scientific Title	A clinical study to evaluate efficacy, safety and tolerability of ISN001 in dystrophic epidermolysis bullosa patients.
Scientific Title:Acronym	A clinical study to evaluate efficacy, safety and tolerability of ISN001 in dystrophic epidermolysis bullosa patients.
Region	Japan

Condition
Condition	dystrophic epidermolysis bullosa
Classification by specialty	Dermatology
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	Multi-center, open-clinical trial of human mesenchymal stem cells derived from adipocytes (ALLO-ASC sheet) for the treatment of dystrophic epidermolysis bullosa.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1	Confirmatory
Trial characteristics_2	Explanatory
Developmental phase	Phase I,II

Assessment
Primary outcomes	Improvement in size of cutaneous ulcer at 5 weeks after the ISN001 application completion.
Key secondary outcomes	(1)Improvement of cutaneous ulcer size at every visit (2)Re-epithelization of the lesions treated (3)Over-all clinical efficacy assessment

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	NO
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Other
Interventions/Control_1	ISN001, the sheet containing allogenic adipose derived mesenchymal stromal cells (1000000/sheet), is applied to the lesion(s) once a week up to 8 times
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 who are diagnosed with dystrophic epidermolysis bullosa according to the immunostaining test: (2)Patients with cutaneous ulcer size of 10 - 250cm2 (3)Patients with 50% or fewer changes in cutaneous ulcer size during the screening period
Key exclusion criteria	(1)Patients with the following medical histories. 1)Patients who are being suffered from squamous cell carcinoma except more than one year has passed without relapses following surgical operations. 2)Patients with severe allergic reactions such as shock and/or anaphylaxis clinical symptoms 3)Patients with skin hypersensitivities against topically applied medicines. 4)Patients with allergies against xenogeneic proteins 5)Patients with allergies against penicillin, streptomycin and/or amphotericin B. 2) Patients with the following complications. 1) Severe functional failures in the liver, heart and/or lung. (3)Patients who underwent the following conditions within one year of the day of initiation of this study. 1) Drug abuses 2) Alcohol dependencies (4)Patients who participated in other clinical trials and/or intervention studies within 4 months (120 days) of the day of initiation of this study. (5)Patients who are considered not suitable for the study by principal investigator.
Target sample size	5

Research contact person
Name of lead principal investigator	1st name Middle name Last name Teikichi Aoyagi
Organization	Ishin Pharmaceutical Co. Ltd.,
Division name	Clinical development department
Zip code
Address	2-3-11 Nihonbashi-honcyo, Chuo-ku, Tokyo, Japan.
TEL	03-6262-5243
Email	aoyagi@ishinpharma.co.jp

Public contact
Name of contact person	1st name Middle name Last name Sadao Kikuta
Organization	Ishin Pharmaceutical Co. Ltd.,
Division name	Clinical development department
Zip code
Address	2-3-11 Nihonbashi-honcyo, Chuo-ku, Tokyo, Japan.
TEL	03-6262-5243
Homepage URL
Email	kikuta@ishinpharma.co.jp

Sponsor
Institute	Ishin Pharmaceutical Co. Ltd.
Institute
Department

Funding Source
Organization	Ishin Pharmaceutical Co. Ltd.
Organization
Division
Category of Funding Organization	Profit organization
Nationality of Funding Organization

Other related organizations
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IRB Contact (For public release)
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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	・北海道大学病院（北海道）Hokkaido University Hospital ・東邦大学医療センター大森病院（東京都）Toho University Omori Medical Center ・埼玉医科大学（埼玉県） Saitama Medical University Hospital

Other administrative information
Date of disclosure of the study information	2017 Year 07 Month 25 Day

Related information
URL releasing protocol
Publication of results	Unpublished

Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
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Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
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IPD sharing Plan description

Progress
Recruitment status	Enrolling by invitation
Date of protocol fixation	2017 Year 02 Month 16 Day
Date of IRB
Anticipated trial start date	2017 Year 06 Month 21 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date	2017 Year 07 Month 25 Day
Last modified on	2018 Year 01 Month 23 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000032442

Research Plan
Registered date	File name

Research case data specifications
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Research case data
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