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A single-arm, unblinded phase 2 trial of allogeneic iPS cell-derived corneal epithelial cell sheet REM-01 transplantation in subjects with limbal stem cell deficiency

Basic Information

Recruitment status	Recruiting
Health condition(s) or Problem(s) studied	Limbal stem cell deficiency
Date of first enrollment	10/04/2026
Target sample size	12
Countries of recruitment
Study type	Interventional
Intervention(s)	For each eye, an iPS cell-derived corneal epithelial cell sheet is transplanted onto the corneal surface

Outcome(s)

Primary Outcome

Success rate of corneal epithelial reconstruction at 52 weeks after REM-01 transplantation (%)

Secondary Outcome

Efficacy 1) Severity of limbal stem cell deficiency up to 52 weeks post-REM-01 transplantation 2) Corrected visual acuity up to 52 weeks post-REM-01 transplantation 3) Severity of corneal opacity up to 52 weeks post-REM-01 transplantation 4) Severity of corneal neovascularization up to 52 weeks post-REM-01 transplantation 5) Severity of symblepharon up to 52 weeks post-REM-01 transplantation 6) Corneal epithelial defects up to 52 weeks post-REM-01 transplantation Safety 1) Key safety endpoints: rejection, neoplastic lesions 2) Frequency and severity of adverse events 3) Frequency of complications

Key inclusion & exclusion criteria

Age minimum	>= 16age old
Age maximum	Not applicable
Gender
Include criteria	1) Patients diagnosed with Stage IIB, IIC, or III of linbal stem cell deficiency according to the severity classification. 2) Patients who have provided written consent to participate in this clinical trial, either by themselves or their legal representative. 3) Patients aged 16 years or older at the time of obtaining consent.
Exclude criteria	1) Patients with contraindications to the antibacterial agents, steroids, immunosuppressants, or anesthetics used in this clinical trial. 2) Patients with a history of allergy to cattle, mice, or pigs. 3) Patients with a history of malignant tumors within the past 5 years, or patients suspected of having malignant tumors. 4) Patients with glaucoma whose intraocular pressure is poorly controlled. 5) Patients with diabetes whose blood glucose is poorly controlled. 6) Pregnant women, breastfeeding women, patients who may be pregnant, or patients who wish to become pregnant during the clinical trial period. 7) Patients who have participated in, are currently participating in, or are scheduled to participate in clinical trials of other pharmaceuticals, regenerative medicine products, or medical devices within 16 weeks prior to the scheduled transplant. 8) Patients scheduled to undergo intraocular surgery during the clinical trial period. 9) Patients who have complications, infections, etc. in the transplanted eye or systemically, and who the principal investigator or co-investigator determines cannot be adequately evaluated (patients who are positive for HBs antigen, HBc antibody, or HBs antibody are considered unsuitable. However, this does not apply to hepatitis B vaccine recipients who are positive for HBs antibody, negative for HBs antigen, and negative for HBc antibody). 10) Other patients whom the principal investigator or co-investigator deems unsuitable.