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

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 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


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

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000034226


Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name