Limbal Stem-cell DeficiencyPhase 1/2NCT02415218
Transplantation of Autologous Oral Mucosal Epithelial Sheets for Limbal Stem-cell Deficiency
A Phase 1/2 study testing Mucosal cell sheet transplantation in people with Limbal Stem-cell Deficiency, run by Siriraj Hospital at 1 site in Thailand.
Trial 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… Read more about this study
- Who is it for?
- Ages 20 years and older
- Conditions the study is for
- Limbal Stem-cell Deficiency
- What is being tested?
- Mucosal cell sheet transplantation
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteThailand
- Study dates
- 17 Apr 2013to 16 Apr 2017
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 20 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.)
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
Thailand1 site
Siriraj Clinical Research Center, Siriraj Hospital
Bangkoknoi, Bangkok, 10700
About this study
Official title: 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.
Read the 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
- Being tested
- Mucosal cell sheet transplantation
- Medical subject headings
- Limbal Stem Cell Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mucosal cell sheet transplantationBiological
The subjects will have their oral mucosal tissues retrieved for cell sheet preparation. The mucosal cell sheet will be transplanted over the affected cornea.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Biological: Mucosal cell sheet transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 1 year after transplantation
Secondary outcomes (1)
Corrected visual acuity (efficacy)
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.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Apr 2015 (estimated)
- Study start17 Apr 2013
- Primary completion3 Sep 2016
- Study completion16 Apr 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Limbal Stem-cell DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Siriraj Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Osaka University
- Responsible party
- Principal investigator, Adisak Wongkajornsilp, Siriraj Hospital
- Investigators
- Adisak Wongkajornsilp, M.D., Ph.D., Principal investigator, Mahidol University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02415218
- Sponsor's protocol ID
- 662/2555(EC2)
- Status last verified
- Oct 2019
- First submitted
- 8 Apr 2015
- Last updated
- 11 Oct 2019
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 35922868
Study at a glance
From the registry record, last updated by the sponsor on 11 Oct 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study of Transplantation of Autologous Cultivated Oral Mucosal Epithelial Sheets for Limbal Stem-cell Deficiency
- ClinicalTrials.gov ID
- NCT02415218
- Acronym / sponsor ID
- 662/2555(EC2)
- Conditions
- Limbal Stem-cell Deficiency
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Siriraj HospitalUniversity, hospital or other
- Collaborators
- Osaka University
- Study start
- 17 Apr 2013
- Primary completion
- 3 Sep 2016 (actual)
- Study completion
- 16 Apr 2017 (actual)
- First posted
- 14 Apr 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Mucosal cell sheet transplantation | Experimental | Biological: Mucosal cell sheet transplantationThe subjects will have their oral mucosal tissues retrieved for cell sheet preparation. The mucosal cell sheet will be transplanted over the affected cornea. |
Who may qualify
- Ages
- 20 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Mucosal cell sheet transplantationThe 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 |
Primary outcome
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 secondary outcome
Corrected visual acuity (efficacy)
Time frame: 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Oct 2019.
- Derived from this study: PubMed 35922868Booranapong 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.
Related trials for Limbal Stem-cell Deficiency
RecruitingPhase 2
Efficacy of Topical Mesenchymal Stromal Cell Secretome for Ocular Surface Disease
Persistent Corneal Epithelial Defect, Corneal Epithelial Disorders
RecruitingPhase 1
Study to Evaluate the Safety and Efficacy of CSB-001 Ophthalmic Solution 0.1% in Subjects With Limbal Stem Cell Deficiency
Limbal Stem Cell Deficiency
RecruitingObservational study
Multi-Center, Prospective, Non-Interventional Study in Subjects With Limbal Stem Cell Deficiency
Limbal Stem Cell Deficiency
Not yet recruitingObservational study
Clinical Predictors of Visual Outcome in Patients Affected With Ocular Surface Diseases
Neurotrophic Keratopathy, Exposure Keratopathy
Active, not recruitingPhase 1
Stem Cell Therapy for Limbal Stem Cell Deficiency
Limbal Stem-cell Deficiency
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Adisak Wongkajornsilp, M.D., Ph.D. principal investigator
Mahidol University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Mucosal cell sheet transplantation in people with Limbal Stem-cell Deficiency, run by Siriraj Hospital at 1 site in Thailand.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Oct 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Thailand. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluation of conjunctivalization and corneal epithelial defect (time frame: 1 year after transplantation). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02415218, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Oct 2019.