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

NCT02415218 | Completed Phase 1 DMC

• 1 other identifier: 662/2555(EC2)
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

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.

Conditions

Limbal Stem-cell Deficiency

Outcome Measures

Primary Outcomes (1)

• Evaluation of conjunctivalization and corneal epithelial defect

  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 Outcomes (1)

• Corrected visual acuity (efficacy)

  1 year

Study Arms (1)

Mucosal cell sheet transplantation

EXPERIMENTAL

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

Interventions

Mucosal cell sheet transplantation BIOLOGICAL

The subjects will have their oral mucosal tissues retrieved for cell sheet preparation. The mucosal cell sheet will be transplanted over the affected cornea.

Mucosal cell sheet transplantation

Eligibility Criteria

• Age: 20 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Siriraj Hospital: lead
• Osaka University: collaborator

Study Sites (1)

Siriraj Clinical Research Center, Siriraj Hospital

Bangkoknoi, Bangkok, 10700, Thailand

Location

Related Publications (1)

• Booranapong W, Kosrirukvongs P, Duangsa-Ard S, Kasetsinsombat K, Sa-Ngiamsuntorn K, Wongkajornsilp A. Transplantation of autologous cultivated oral mucosal epithelial sheets for limbal stem cell deficiency at Siriraj Hospital: a case series. J Med Case Rep. 2022 Aug 4;16(1):298. doi: 10.1186/s13256-022-03502-8.

  PMID: 35922868 DERIVED

MeSH Terms

Conditions

Limbal Stem Cell Deficiency

Condition Hierarchy (Ancestors)

Corneal Diseases; Eye Diseases

Study Officials

• Adisak Wongkajornsilp, M.D., Ph.D.

  Mahidol University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: M.D., Ph.D.

Study Record Dates

• First Submitted: April 8, 2015
• First Posted: April 14, 2015
• Study Start: April 17, 2013
• Primary Completion: September 3, 2016
• Study Completion: April 16, 2017
• Last Updated: October 11, 2019

Record last verified: 2019-10

Locations

TH (1)