UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000029962 |
|---|---|
| Receipt number | R000034226 |
| Scientific Title | Clinical trial of mesenchymal stem cell mobilization factor KOI2 in epidermolysis bullosa patients |
| Date of disclosure of the study information | 2017/11/30 |
| Last modified on | 2020/11/19 17:40:24 |
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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
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
kawamura@dmi.med.osaka-u.ac.jp
Sponsor or person
Institute
Osaka University Hospital
Institute
Department
Personal name
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
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
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000034226
