UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Multicenter Investigator-initiated clinical trial using cultivated autologous oral mucosal epithelial cell sheet (COMET01) transplantation for patients with limbal stem-cell deficiency
Title of the study (Brief title)	Multicenter Investigator-initiated clinical trial using COMET01 for patients with limbal stem-cell deficiency
Region	Japan

Condition
Condition	limbal stem-cell deficiency
Classification by specialty	Ophthalmology
Classification by malignancy	Others
Genomic information	NO

Objectives
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

Assessment
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

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
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

Eligibility
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

Research contact person
Name of lead principal investigator	Kohji Nishida
Organization	Osaka University Graduate School of Medicine
Division name	Department of Ophthalmology
Address	2-2, Yamada-oka, Suita, Osaka
TEL	+81-6-6879-3451
Email	knishida@ophthal.med.osaka-u.ac.jp

Public contact
Name of contact person	Yoshinori Oie
Organization	Osaka University Graduate School of Medicine
Division name	Department of Ophthalmology
Address	2-2, Yamada-oka, Suita, Osaka
TEL	+81-6-6879-3456
Homepage URL
Email	yoie@ophthal.med.osaka-u.ac.jp

Sponsor
Institute	Department of Ophthalmology, Osaka University Graduate School of Medicine
Institute
Department

Funding Source
Organization	Japan Agency for Medical Research and Development
Organization
Division
Category of Funding Organization	Other
Nationality of Funding Organization	Japan

Other related organizations
Co-sponsor
Name of secondary funｄer(s)

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	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）

Other administrative information
Date of disclosure of the study information	2015 Year 08 Month 13 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2015 Year 07 Month 01 Day
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

Related information
URL releasing protocol
Publication of results	Unpublished
URL releasing results
Results
Other related information

Management information
Registered date	2015 Year 08 Month 13 Day
Last modified on	2018 Year 02 Month 15 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000021510