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

NCT ID: NCT02415218

Last Updated: 2019-10-11

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 6 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-04-17
• Study Completion Date: 2017-04-16

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.

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.

Conditions

Limbal Stem-cell Deficiency

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

Mucosal cell sheet transplantation

Intervention Type: 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.

Interventions

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.

Intervention Type: BIOLOGICAL

Eligibility 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

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.

• Minimum Eligible Age: 20 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Osaka University

OTHER

Sponsor Role: collaborator

Siriraj Hospital

OTHER

Sponsor Role: lead

Responsible Party

Adisak Wongkajornsilp

M.D., Ph.D.

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Adisak Wongkajornsilp, M.D., Ph.D.

Role: PRINCIPAL_INVESTIGATOR

Mahidol University

Locations

Siriraj Clinical Research Center, Siriraj Hospital

Bangkoknoi, Bangkok, Thailand

Countries

Thailand

References

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.

Reference Type: DERIVED

PMID: 35922868 (View on PubMed)

Other Identifiers

662/2555(EC2)

Identifier Type: -

Identifier Source: org_study_id