- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02415218
Transplantation of Autologous Oral Mucosal Epithelial Sheets for Limbal Stem-cell Deficiency
October 10, 2019 updated by: Adisak Wongkajornsilp, Siriraj Hospital
Clinical Study of Transplantation of Autologous Cultivated Oral Mucosal Epithelial Sheets for Limbal Stem-cell Deficiency
The study purpose is to evaluate the efficacy and safety of cultivated oral mucosal epithelial sheet transplantation as a novel therapy for limbal stem cell deficiency, for which no effective approaches has thus far been available, for the purpose of establishing this therapy as an effective strategy for this disease with an aim of improving visual acuity and corneal transparency.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Detailed Description
The efficacy and safety of cultivated oral mucosal epithelial sheet transplantation as a novel therapy for limbal stem-cell deficiency, for which no effective approaches have thus far been available, will be evaluated for the purpose of establishing this therapy as an effective strategy for this disease with an aim of improving visual acuity and corneal transparency.
The primary endpoint will be an area of no conjunctivalization and no corneal epithelial defect at one year, which will be evaluated according to a 6-grade rating scale.
The secondary endpoints will be corrected visual acuity, severity of corneal opacification, extent of corneal neovascularization, expected ocular complications, and all adverse events including abnormal laboratory findings.
The purpose of this research is to evaluate the efficacy of the investigational therapy so as to provide rationales for making this strategy approved as an advanced medical care in the future.
Study Type
Interventional
Enrollment (Actual)
6
Phase
- Phase 2
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Bangkok
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Bangkoknoi, Bangkok, Thailand, 10700
- Siriraj Clinical Research Center, Siriraj Hospital
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
20 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
Among patients with bilateral limbal stem-cell deficiency, those who meet all the criteria listed below when they give their informed consent to participate in this research will be included.
- Patients in whom the eye(s) (subject to the interventional therapy) show(s) loss or dysfunction of corneal epithelial stem cells throughout the total* circumference of the cornea
- Patients in whom the corneal surface of the eye(s) (subject to the investigational therapy) is(are) entirely enveloped by conjunctival tissue
- Patients of both genders aged ≥20 years when they give their informed consent
- Patients who have given their own written consent to participate in this clinical research (The target disease is bilateral limbal stem-cell deficiency, although it is not always necessary that the both eyes of the patient meet the inclusion criteria.)
Exclusion Criteria:
- Patients who are or may be pregnant, and patients who are breast feeding
- Patients with infections (HBV, HCV, HIV, HTLV-1)
- Patients with extreme hypolacrimation resistant to therapy
- Patients from whom oral mucosal specimens cannot be collected because clear scar formation or inflammatory findings are noted in a cell-collection site of the mucosal membrane
- Patients whose score of Performance Status (PS*) is 3 or more
- Any others who are judged by the chief subinvestigator to be ineligible for this clinical research, due to the presence of complications, etc.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Mucosal cell sheet transplantation
The subjects will have their oral mucosal tissues retrieved for cell sheet preparation.
The mucosal cell sheet will be transplanted over the affected cornea.
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The subjects will have their oral mucosal tissues retrieved for cell sheet preparation.
The mucosal cell sheet will be transplanted over the affected cornea.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Evaluation of conjunctivalization and corneal epithelial defect
Time Frame: 1 year after transplantation
|
One year after the transplantation, an area of no conjunctivalization and no corneal epithelial defect will be rated according to the Grading scale described in Table 2, on the basis of which the efficacy of the investigational therapy will be assessed.
Generally, the preoperative condition of ocular surface is rated as "Bad (Grading 0)."
In subjects showing improvement to Grading 2 or greater, the investigational therapy will be assessed as effective.
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1 year after transplantation
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Corrected visual acuity (efficacy)
Time Frame: 1 year
|
Corrected visual acuity will be evaluated by the decimal acuity method and converted to Log MAR values, which will be used to assess the extent of postoperative change in visual acuity.
Note that 0.005 will be given to "counting fingers," 0.002, to "hand motion," and 0.001, to "light perception" 14).
Visual acuity will be measured before transplantation, and 1, 3, and 6 months as well as one year after transplantation.
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1 year
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Collaborators
Investigators
- Principal Investigator: Adisak Wongkajornsilp, M.D., Ph.D., Mahidol University
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
April 17, 2013
Primary Completion (Actual)
September 3, 2016
Study Completion (Actual)
April 16, 2017
Study Registration Dates
First Submitted
April 8, 2015
First Submitted That Met QC Criteria
April 13, 2015
First Posted (Estimate)
April 14, 2015
Study Record Updates
Last Update Posted (Actual)
October 11, 2019
Last Update Submitted That Met QC Criteria
October 10, 2019
Last Verified
October 1, 2019
More Information
Terms related to this study
Other Study ID Numbers
- 662/2555(EC2)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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Clinical Trials on Mucosal cell sheet transplantation
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He Eye HospitalUnknown
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Air Force Military Medical University, ChinaUnknownChronic PeriodontitisChina
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Virginia Commonwealth UniversityTerminatedMalignant Neoplasm | Leukemia, Not Otherwise SpecifiedUnited States
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Mahidol UniversityUnknownOcular Surface DiseaseThailand
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Hospices Civils de LyonRecruitingTotal Bilateral Limbal Cell DeficiencyFrance