UMIN-CTR Clinical Trial

Public title

Multicenter Investigator-initiated clinical trial using cultivated autologous oral mucosal epithelial cell sheet (COMET01) transplantation for patients with limbal stem-cell deficiency

Acronym

Multicenter Investigator-initiated clinical trial using COMET01 for patients with limbal stem-cell deficiency

Scientific Title

Multicenter Investigator-initiated clinical trial using cultivated autologous oral mucosal epithelial cell sheet (COMET01) transplantation for patients with limbal stem-cell deficiency

Scientific Title:Acronym

Multicenter Investigator-initiated clinical trial using COMET01 for patients with limbal stem-cell deficiency

Region

Japan

Condition

limbal stem-cell deficiency

Classification by specialty

Ophthalmology

Classification by malignancy

Others

Genomic information

NO

Narrative objectives1

To evaluate the efficacy and safety of cultivated autologous oral mucosal epithelial cell sheet (COMET01) transplantation.

Basic objectives2

Efficacy

Basic objectives -Others

Trial characteristics_1

Trial characteristics_2

Developmental phase

Primary outcomes

success rate of corneal epithelium reconstruction 52 weeks after COMET01 transplantation

Key secondary outcomes

Efficacy
1) case number of each LSCD staging 52 weeks after COMET01 transplantation
2) subjective symptom
3) visual acuity
4) QOL
5) corneal opacity
6) corneal neovascularization
7) symblepharon
8) additional therapy for visual improvement 52 weeks after COMET01 transplantation or at the date of termination
9) LSCD staging, subjective symptom, visual acuity, corneal opacity, corneal neovascularization, and symblepharon of fellow eye

Safety
1) Safety endpoint
i. SPK
ii. corneal epithelial defect
iii. keratinization
iv. conjunctival injection
v. infectious keratitis
vi. endophthalmitis
2) Adverse events and Product-related adverse events
3) Severe product-related adverse events

Study type

Interventional

Basic design

Single arm

Randomization

Non-randomized

Randomization unit

Blinding

Open -no one is blinded

Control

Uncontrolled

Stratification

Dynamic allocation

Institution consideration

Blocking

Concealment

No. of arms

1

Purpose of intervention

Treatment

Type of intervention

Device,equipment

Interventions/Control_1

Removal of oral mucosal tissue, COMET01 transplantation

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

Age-lower limit

20

years-old

<=

Age-upper limit

Not applicable

Gender

Male and Female

Key inclusion criteria

1) Limbal stem-cell deficiency patients who are classified as LSCD Stage III
2) Patients who don't have extensive oral mucosal scarring and have healthy oral mucosal tissue for harvest.
3) Aged 20 years old and over

Key exclusion criteria

1) contraindication to antimicrobials, steroid drugs, and anesthetics used in the trial
2) allergy to antibiotics of penicillin, kanamycin, streptomycin, amphotericin B; Medical history of allergy to penicillin antibiotic and aminoglycoside antibiotic
3) medical history of allergy to animal (cattle, mice, or pigs)
4) malignant tumor within 5 years before registration, or doubtful malignant tumor
5) glaucoma with uncontrollable IOP
6) diabetes with uncontrollable glycemic control
7) eye with central visual field defect
8) extremely severe dry eye
9) planned intraocular surgery during the trial
10) eye with severe eyelid defect
11) systemic complications unsuitable for entry in this trial
12) pregnant/lactating women, might be pregnant women; wish to become pregnant women
13) another trial participant within 16 weeks before COMET01 transplantation, planning to participate in another trial during this trial
14) unsuitable for entry in this trial judged by principal investigator or sub-investigators

Target sample size

6

Name of lead principal investigator

1st name
Middle name
Last name	Kohji Nishida

Organization

Osaka University Graduate School of Medicine

Division name

Department of Ophthalmology

Zip code

Address

2-2, Yamada-oka, Suita, Osaka

TEL

+81-6-6879-3451

Email

knishida@ophthal.med.osaka-u.ac.jp

Name of contact person

1st name
Middle name
Last name	Yoshinori Oie

Organization

Osaka University Graduate School of Medicine

Division name

Department of Ophthalmology

Zip code

Address

2-2, Yamada-oka, Suita, Osaka

TEL

+81-6-6879-3456

Homepage URL

Email

yoie@ophthal.med.osaka-u.ac.jp

Institute

Department of Ophthalmology, Osaka University Graduate School of Medicine

Institute

Department

Personal name

Organization

Japan Agency for Medical Research and Development

Organization

Division

Category of Funding Organization

Other

Nationality of Funding Organization

Japan

Co-sponsor

Name of secondary funder(s)

Organization

Address

Tel

Email

Secondary IDs

NO

Study ID_1

Org. issuing International ID_1

Study ID_2

Org. issuing International ID_2

IND to MHLW

Institutions

大阪大学医学部附属病院（大阪府）
東北大学病院（宮城県）
東京大学医学部附属病院（東京都）

Date of disclosure of the study information

2015

Year

08

Month

13

Day

URL releasing protocol

Publication of results

Unpublished

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

Recruitment status

Completed

Date of protocol fixation

2015

Year

07

Month

01

Day

Date of IRB

Anticipated trial start date

2015

Year

08

Month

14

Day

Last follow-up date

Date of closure to data entry

Date trial data considered complete

Date analysis concluded

Other related information

Registered date

2015

Year

08

Month

13

Day

Last modified on

2018

Year

02

Month

15

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000021510