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

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

    • Bangkok
      • Bangkoknoi, Bangkok, Thailand, 10700
        • Siriraj Clinical Research Center, Siriraj Hospital

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.

    1. 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
    2. Patients in whom the corneal surface of the eye(s) (subject to the investigational therapy) is(are) entirely enveloped by conjunctival tissue
    3. Patients of both genders aged ≥20 years when they give their informed consent
    4. 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:

  1. Patients who are or may be pregnant, and patients who are breast feeding
  2. Patients with infections (HBV, HCV, HIV, HTLV-1)
  3. Patients with extreme hypolacrimation resistant to therapy
  4. 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
  5. Patients whose score of Performance Status (PS*) is 3 or more
  6. 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.
The subjects will have their oral mucosal tissues retrieved for cell sheet preparation. The mucosal cell sheet will be transplanted over the affected cornea.

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.
1 year after transplantation

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.
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

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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