Safety Evaluation of LSCD101 Transplantation for Limbal Stem Cell Deficiency

Study Overview
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Brief Summary
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Clinical Study Objective: To evaluate the tolerability and safety after transplantation of LSCD101 in patients with intractable limbal stem cell deficiency Study Method: Subjects who finally meet the inclusion/exclusion criteria results of the subject eligibility evaluation will receive transplantation of the investigational product. Adverse drug reaction will be confirmed during 24 weeks after transplantation.

Official Title
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Phase I Study to Evaluate Safety of LSCD101(Cultured Autologous Limbal Epithelial Cell Sheet) Transplantation for Limbal Stem Cell Deficiency

Conditions
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Limbus Corneae, Limbus Corneae Insufficiency Syndrome

Intervention / Treatment
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* Drug: LSCD101 (Cultured Autologous Limbal Epithelial Cell Sheet) transplantation


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Men and women aged 19 or older Diagnosed with intractable limbal stem cell deficiency in single eye, with severity in which corneal neovascularization invades more than two quadrants No improvement of indication by known treatment (non-surgical/surgical) method according to investigator's judgment BCVA 20/40 or less at screening in the eye with the limbal stem cell deficiency Voluntarily agreed to participate in this study and signed a consent form Exclusion Criteria: Inappropriate for harvesting limbal epithelial cells for the production of limbal epithelial cell sheet. Difficulty in reconstruction of the corneal epithelium because the eye is not closed due to eyelid function problems in the eye with the limbal stem cell deficiency. Severe dry eye with problem with tear secretion in both eyes (value of Schirmer I test without anesthesia < 2 mm/5 min at screening) Acute ocular surface inflammation of both corneas at Visit 2 Malignant tumor history (except in case of no recurrence more than 5 years after surgery) Uncontrolled comorbidities such as moderate to severe infections and bleeding Positive for virus infection (HBV, HCV, HIV, CMV, HTLVⅠ/Ⅱ, syphilis) Uncontrolled diabetes (HbA1c ≥9.0%) Uncontrolled hypertension (SBP > 140 mmHg or DBP > 90 mmHg despite taking antihypertensives at screening) Uncontrolled glaucoma (IOP > 21 mmHg despite taking glaucoma medication) Pregnant or lactating women Women with childbearing potential, those who do not agree to contraception by medically accepted contraceptive methods during clinical trials Participation in another clinical study within 4 weeks Subjects judged by the investigator to be inappropriate to participate in the clinical study

Ages Eligible for Study
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Minimum Age: 19 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: LSCD101 (Cultured Autologous Limbal Epithelial Cell Sheet) transplantation<br>LSCD101 (Cultured Autologous Limbal Epithelial Cell Sheet) is transplanted in the lesion. | Drug: LSCD101 (Cultured Autologous Limbal Epithelial Cell Sheet) transplantation<br>* LSCD101 (Cultured Autologous Limbal Epithelial Cell Sheet) is transplanted in the lesion.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Rate of Adverse event | Adverse event | From limbal tissue collection until 24 weeks after transplantation | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Rate of normal/abnormal results in clinical laboratory test | Comparisons between pre-/post- transplantation are summarized and presented | Before treatment, 1 week, 2 weeks, 4 weeks, 12 weeks, 24 weeks after transplantation | 
| Blood pressure(systolic/diastolic) at each visit | Comparisons between pre-/post- transplantation are summarized and presented. | Before treatment, 1 day, 1 week, 2 weeks, 4 weeks, 12 weeks, 24 weeks after transplantation | 
| Pulse rate at each visit | Comparisons between pre-/post- transplantation are summarized and presented. | Before treatment, 1 day, 1 week, 2 weeks, 4 weeks, 12 weeks, 24 weeks after transplantation | 
| Rate of normal/abnormal results in Electrocardiogram | Comparisons between pre-/post- transplantation are summarized and presented. | Before treatment, 4 weeks, 12 weeks, 24 weeks after transplantation | 
| Rate of normal/abnormal results in physical examination at each visit | Comparisons between pre-/post- transplantation are summarized and presented. | Before treatment, 4 weeks, 12 weeks, 24 weeks after transplantation | 
| Rate of eye infection and inflammation | Eye infection and inflammation test (slit lamp microscopy) | Before treatment, 1 week, 2 weeks, 4 weeks, 12 weeks, 24 weeks after transplantation | 
| Intraocular pressure test | Intraocular pressure test | Before treatment, 4 weeks, 12 weeks, 24 weeks after transplantation |