Safety Evaluation of LSCD101 Transplantation for Limbal Stem Cell Deficiency

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

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.

Study Overview

• Status: Completed
• Conditions: Limbus Corneae Insufficiency Syndrome; Limbus Corneae
• Intervention / Treatment: Drug: LSCD101 (Cultured Autologous Limbal Epithelial Cell Sheet) transplantation

• Study Type: Interventional
• Enrollment (Actual): 6
• Phase: Phase 1

Contacts and Locations

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of, 04168
    • CliPS

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 19 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: LSCD101 (Cultured Autologous Limbal Epithelial Cell Sheet) transplantation; LSCD101 (Cultured Autologous Limbal Epithelial Cell Sheet) is transplanted in the lesion.	Drug: LSCD101 (Cultured Autologous Limbal Epithelial Cell Sheet) transplantation; LSCD101 (Cultured Autologous Limbal Epithelial Cell Sheet) is transplanted in the lesion.

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: CliPS Co., Ltd
• Investigators: Principal Investigator: SoHyang Chung, MD,Ph.D, The Catholic University of Korea

Study record dates

Study Major Dates

• Study Start (Actual): January 31, 2020
• Primary Completion (Actual): January 14, 2021
• Study Completion (Actual): January 14, 2021

Study Registration Dates

• First Submitted: January 22, 2021
• First Submitted That Met QC Criteria: February 24, 2021
• First Posted (Actual): February 26, 2021

Study Record Updates

• Last Update Posted (Actual): February 26, 2021
• Last Update Submitted That Met QC Criteria: February 24, 2021
• Last Verified: January 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Corneal Diseases
• Other Study ID Numbers: LSCD101_P1

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No