Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

Transplantation of Autologous Oral Mucosal Epithelial Sheets for Limbal Stem-cell Deficiency

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02415218
Recruitment Status : Completed
First Posted : April 14, 2015
Last Update Posted : October 11, 2019
Sponsor:
Collaborator:
Osaka University
Information provided by (Responsible Party):
Adisak Wongkajornsilp, Siriraj Hospital

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Limbal Stem-cell Deficiency Biological: Mucosal cell sheet transplantation Phase 1 Phase 2

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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 6 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Clinical Study of Transplantation of Autologous Cultivated Oral Mucosal Epithelial Sheets for Limbal Stem-cell Deficiency
Study Start Date : April 17, 2013
Actual Primary Completion Date : September 3, 2016
Actual Study Completion Date : April 16, 2017

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.
Biological: 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.




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   20 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02415218


Locations
Layout table for location information
Thailand
Siriraj Clinical Research Center, Siriraj Hospital
Bangkoknoi, Bangkok, Thailand, 10700
Sponsors and Collaborators
Siriraj Hospital
Osaka University
Investigators
Layout table for investigator information
Principal Investigator: Adisak Wongkajornsilp, M.D., Ph.D. Mahidol University
Layout table for additonal information
Responsible Party: Adisak Wongkajornsilp, M.D., Ph.D., Siriraj Hospital
ClinicalTrials.gov Identifier: NCT02415218    
Other Study ID Numbers: 662/2555(EC2)
First Posted: April 14, 2015    Key Record Dates
Last Update Posted: October 11, 2019
Last Verified: October 2019