UMIN-CTR Clinical Trial

Unique ID issued by UMIN UMIN000012819
Receipt number R000014978
Scientific Title Clinical Study on Cultivated Autologous Oral Mucosal Epithelial Sheet Transplantation for Treating Patients with Intractable Keratoconjunctival Disease
Date of disclosure of the study information 2014/01/16
Last modified on 2017/08/09 11:15:02

* This page includes information on clinical trials registered in UMIN clinical trial registed system.
* We don't aim to advertise certain products or treatments


Basic information

Public title

Clinical Study on Cultivated Autologous Oral Mucosal Epithelial Sheet Transplantation for Treating Patients with Intractable Keratoconjunctival Disease

Acronym

Cultivated Autologous Oral Mucosal Epithelial Sheet Transplantation

Scientific Title

Clinical Study on Cultivated Autologous Oral Mucosal Epithelial Sheet Transplantation for Treating Patients with Intractable Keratoconjunctival Disease

Scientific Title:Acronym

Cultivated Autologous Oral Mucosal Epithelial Sheet Transplantation

Region

Japan


Condition

Condition

Patients with keratoconjunctival disease
(Stevens-Johnson syndrome, ocular pemphigoid, or severe thermal/chemical injury)

Classification by specialty

Ophthalmology

Classification by malignancy

Others

Genomic information

NO


Objectives

Narrative objectives1

The aims of this clinical study on cultivated autologous oral mucosal epithelial sheet transplantation for patients with intractable keratoconjunctival disease (hereinafter referred to as the "present study") are as follows: 1) to check the safety of cultivated autologous oral mucosal epithelial sheet transplantation for patients with severe stem cell deficiency who are afflicted with the most severe degree of an intractable keratoconjunctival disease which is difficult to treat using any method, 2) to improve abnormalities on the ocular surface (corneal opacity, epithelial defects, and ocular surface adhesion), and 3) to investigate the efficacy of ocular surface restoration, which is further categorized into corneal restoration and conjunctival restoration.

Basic objectives2

Safety,Efficacy

Basic objectives -Others


Trial characteristics_1

Exploratory

Trial characteristics_2

Pragmatic

Developmental phase

Phase I,II


Assessment

Primary outcomes

1)Change in distance vision (5 meters) from before transplantation to postoperative Week 24.
2)Change in total epithelial abnormality score (out of the total ophthalmological findings A score, the total score for epithelial defects, conjunctival invasion, and vascular invasion) from before transplantation to postoperative Week 24.
3)Change in conjunctival sac adhesion score (total of scores for upper and lower) within ophthalmological findings A from before transplantation to postoperative Week 24.

The purpose of transplantation is either improvement of vision, repairing epithelial defects, or detaching ocular surface adhesion. The primary endpoints are the scores for changes in vision, changes in total epithelial abnormality scores, and conjunctival sac adhesion score for each purpose.

Key secondary outcomes

<Objective findings>
Each item in ophthalmology findings A and B.
<Safety Evaluation>
Incidence rates and severities of adverse events.


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

Device,equipment Maneuver

Interventions/Control_1

Corneal reconstruction (improvement of vision and epithelial repair)
Conjunctival sac reconstruction (adhesion separation)

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

15 years-old <=

Age-upper limit

85 years-old >

Gender

Male and Female

Key inclusion criteria

1) Being diagnosed with intractable keratoconjunctival disease due to the following diseases: a) Stevens Johnson syndrome, b) ocular pemphigoid, or c) severe thermal/chemical injury).
2) Afflicted with a bilateral disease with no availability of a therapeutic option that utilizes autologous tissue transplantation.
3) Being 15 years of age or older and younger than 86 years of age at the time of obtaining informed consent.
4) Patients undergoing transplantation for the purpose of recovering vision, improving epithelial defects, or improving ocular surface adhesion. In addition, scores and values for each purpose at the time of registration are as follows:
Improvement of vision: Patients with decimal visual acuity of less than 0.1.
Improvement of epithelial defects: Patients with a total score value for epithelial defects, conjunctival invasion, and vascular invasion of 3 or higher. These are the three ophthalmological findings scores stipulated in the present study.
Detachment of ocular surface adhesion: Patients with 50% or greater adhesion either on the upper or lower conjunctival sac.
5) Patients or their representatives (both the patient and their representative if the patient is between 15 and 20 years of age) from whom written informed consent is obtained.

Key exclusion criteria

1) Having an active corneal infection.
2) Having glaucoma with insufficient ocular pressure control.
3) Patients diagnosed as being complicated with, or carriers of, any of the following diseases: HBV, HCV, HIV, HTLV, or syphilis.
4) Diabetic patients with insufficient glucose control.
5) Patients who are hypersensitive to cyclosporine or steroids.
6) Pregnant women, female patients who have expressed their desire to become pregnant during the course of the study, or those who are currently breastfeeding a child.
7) Patients for whom participation in the present study is believed to be inappropriate due to personal complications or other reasons.
8) Patients who have been diagnosed with any of the following intractable keratoconjunctival diseases: severe recurring pterygium, aniridia, cicatricial pemphigoid, and stem cell deficiency due to drug toxicity or due to an unknown cause.
9) Patients complicated with systemic autoimmune diseases.
10) Patients with visual impairment which is strongly suspected to be affected by other causes.
11) Patients who have already undergone the present protocol treatment.
12) Patients who are currently using or are planning to use anti-cancer agents.
13) Patients with a past history (or those complicated with) cardiac diseases, or cerebrovascular disorders.
14) Patients with impaired eyelid closure due to eyelid function disorders if the purpose of treatment is corneal reconstruction (the purpose of recovering vision or repairing the epithelium).
15) Patients for whom it has been determined that it is difficult to collect the oral cavity mucosal tissue which is required for creating the test material for the present study.
16) Patients for whom it has been determined by the principal investigator or other researchers that there will be complicating issues with the patient participating in the study for other reasons.

Target sample size

30


Research contact person

Name of lead principal investigator

1st name
Middle name
Last name Shigeru Kinoshita, M.D., Ph.D.

Organization

Kyoto Prefectural University of Medicine

Division name

Department of Ophthalmology

Zip code


Address

465 Kajii-cho, Hirokoji-agaru, Kawaramachi-dori, Kamigyo-ku, Kyoto, Japan

TEL

075-251-5577

Email

shigeruk@koto.kpu-m.ac.jp


Public contact

Name of contact person

1st name
Middle name
Last name Chie Sotozono, M.D., Ph.D.

Organization

Kyoto Prefectural University of Medicine

Division name

Department of Ophthalmology

Zip code


Address

465 Kajii-cho, Hirokoji-agaru, Kawaramachi-dori, Kamigyo-ku, Kyoto, Japan

TEL

075-251-5577

Homepage URL


Email

csotozon@koto.kpu-m.ac.jp


Sponsor or person

Institute

Kyoto Prefectural University of Medicine

Institute

Department

Personal name



Funding Source

Organization

Ministry of Health, Labour and Welfare

Organization

Division

Category of Funding Organization

Japanese Governmental office

Nationality of Funding Organization



Other related organizations

Co-sponsor

Foundation for Biomedical Research and Innovation

Name of secondary funder(s)

Ministry of Education, Culture, Sports, Science and Technology


IRB Contact (For public release)

Organization


Address


Tel


Email



Secondary IDs

Secondary IDs

YES

Study ID_1

TRIOPH1007

Org. issuing International ID_1

Translational Research Informatics Center, Foundation for Biomedical Research and Innovation

Study ID_2


Org. issuing International ID_2


IND to MHLW



Institutions

Institutions

京都府立医科大学病院(京都府)
(公財)先端医療振興財団先端医療センター病院(兵庫県)


Other administrative information

Date of disclosure of the study information

2014 Year 01 Month 16 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


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

2013 Year 09 Month 13 Day

Date of IRB


Anticipated trial start date

2014 Year 01 Month 23 Day

Last follow-up date

2017 Year 03 Month 31 Day

Date of closure to data entry


Date trial data considered complete


Date analysis concluded



Other

Other related information



Management information

Registered date

2014 Year 01 Month 10 Day

Last modified on

2017 Year 08 Month 09 Day



Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000014978


Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name