UMIN-CTR Clinical Trial

Basic information
Public title	Clinical trial of mesenchymal stem cell mobilization factor KOI2 in epidermolysis bullosa patients
Acronym	Clinical trial of KOI2 in epidermolysis bullosa patients
Scientific Title	Clinical trial of mesenchymal stem cell mobilization factor KOI2 in epidermolysis bullosa patients
Scientific Title:Acronym	Clinical trial of KOI2 in epidermolysis bullosa patients
Region	Japan

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

Objectives
Narrative objectives1	Primary objective Evaluation of efficacy of KOI2 in epidermolysis bullosa Secondary objective Evaluation of safety of KOI2 in epidermolysis bullosa
Basic objectives2	Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	Change rate of total area of blisters, erosions and ulcers compared to those of base line
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	Medicine
Interventions/Control_1	KOI2 1mg/kg drip infusion administration for 30 minutes. First week; once per day for four days, second- to forth-week; twice per week (at least once in three to four days).
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	Not applicable
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	Patients understands the protocol and agrees to provide written informed consent to participate in the study on a voluntary basis. For patient under 20-year old, informed consent of voluntary participation should be obtained by a legal guardian. Informed assent is collected, if possible, with a patient more than 7-year old. (2) Patients who are confirmed at the present day or before to satisfy following item number 1, and at least one item out of the following number 2 to 5. 1. Blister, erosion, and/or ulcer formations induced by daily minor trauma have been continued on the skin and oral mucosa from beginning of the birth or from 0~6 year old. 2. Existence of patient(s) suffering from dystrophic epidermolysis bullosa in the family member. 3. Intradermal blister formation right beneath the basement membrane has been confirmed by histological study. 4. Decrease or absence of type VII collagen at the cutaneous basement membrane zone has been confirmed by immunofluorescent study. 5. Mutation of type VII collagene gene (COL7A1) has been identified. (3) Total area of vesicles, erosions and ulcers is more than 20 cm2 at the timing of obtaining informed consent. The size of each vesicle, erosion and/or ulcer is measured by length x width using a vernier caliper (Large size can be measured by a ruler). The sum of the areas for the whole body is calculated, excluding those under 2 mm by length. (4) Patient agrees to take appropriate birth control measures from day 1 through 1 month after the last drug administration (including condom, birth control by female partner, and/or others). (5) A woman of childbearing potential, agrees to use a highly effective method of contraception from screening visit through 1 month after the last drug administration.
Key exclusion criteria	Epidermolysis bullosa other than malnutrition type (2) Urine pregnancy test is positive (female only) at screening visit (3) Breast feeding female patient (4) HIV, HBs, HCV positive at screening (5) A subject who participated in other clinical trials within 90 days and received a clinical trial drug (including a placebo) (6) A subject who is participating in any other clinical trials. (7) A subject whose liver dysfunction data match the predefined exclusion standard (8) A subject who has acute worsening of epidermolysis bullosa (such as tracheal/esophageal stenosis, acute nephritis) (9) Ineligible by other reasons judged by the investigators
Target sample size	8

Research contact person
Name of lead principal investigator	1st name Middle name Last name Atsushi Tanemura
Organization	Osaka University Hospital
Division name	Dermatology
Zip code
Address	2-15, Yamadaoka, Suita-shi, Osaka 565-0871, Japan
TEL	06-6879-3902
Email	tanemura8@hotmail.co.jp

Public contact
Name of contact person	1st name Middle name Last name Yoko Kawamura
Organization	Osaka University Hospital
Division name	Department of Medical Innovation
Zip code
Address	2-2, Yamadaoka, Suita-shi, Osaka 565-0871, Japan
TEL	06-6210-8289
Homepage URL
Email	kawamura@dmi.med.osaka-u.ac.jp

Sponsor
Institute	Osaka University Hospital
Institute
Department

Funding Source
Organization	Japan Agency for Medical Research and Development/Shionogi & Co., Ltd.
Organization
Division
Category of Funding Organization	Profit organization
Nationality of Funding Organization

Other related organizations
Co-sponsor	Toho University Omori Medical Center
Name of secondary funder(s)	Keio University Hospital

IRB Contact (For public release)
Organization
Address
Tel
Email

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 11 Month 30 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	9
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status	Completed
Date of protocol fixation	2017 Year 11 Month 30 Day
Date of IRB	2017 Year 10 Month 24 Day
Anticipated trial start date	2018 Year 01 Month 01 Day
Last follow-up date	2019 Year 09 Month 30 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date	2017 Year 11 Month 14 Day
Last modified on	2020 Year 11 Month 19 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000034226