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First-in-human clinical research of iPS derived corneal epithelial cell sheet transplantation for patients with limbal stem-cell deficiency (First-in-human clinical research of iPS derived corneal epithelial cell sheet transplantation for patients with limbal stem-cell deficiency)

First-in-human clinical research of iPS derived corneal epithelial cell sheet transplantation for patients with limbal stem-cell deficiency (First-in-human clinical research of iPS derived corneal epithelial cell sheet transplantation for patients with limbal stem-cell deficiency)

Takehara Tetsuo

Dec. 13, 2021

4

1st series (HLA-incompatible subjects with immunosuppressive agents administered) 1) 44-year-old female (idiopathic limbal stem-cell deficiency) 2) 66-year-old male (ocular pemphigoid) 2nd series (HLA-incompatible subjects without immunosuppressive agents administered) 3) 72-year-old male (idiopathic limbal stem-cell deficiency) 4) 39-year-old female (toxic epidermal necrolysis: a severe form of Stevens-Johnson syndrome)

Two patients enrolled in the 1st series (HLA-incompatible and immunosuppressive agents administered) underwent iCEPS transplantation and completed the specified observation period. Twenty-four weeks after the second transplant, an interim evaluation was conducted and it was decided that the 2nd series would be administered to HLA-incompatible subjects without immunosuppressive agents. Two patients were enrolled in the second series, underwent iCEPS transplantation, and completed the specified observation period.

There was no disease, disability, death or infectious diseases suspected to be caused by iCEPS transplantation.

In all 4 subjects in the 1st and 2nd series, regarding the primary endpoint (safety), no rejection or neoplastic lesions were observed, and no other serious adverse events or diseases were observed. Regarding the secondary endpoint (efficacy), the degree of limbal stem-cell dificiency was improved compared to the preoperative level. Improvement was also observed in other parameters.

There was no safety issue of the primary endpoint, including immune rejection and the development of neoplastic lesions, in four subjects following iCEPS transplantation. All the iCEPS transplantations were successful, with or without immunosuppressive agents, despite transplantation into HLA-incompatible recipients. The degree of limbal stem cell deficiency was improved postoperatively in all the subjects with or without administration of immunosuppressants.

Mar. 31, 2024

https://jrct.niph.go.jp/latest-detail/jRCTa050190084

Nishida Kohji

Osaka University Graduate School of Medicine

2-2, Yamada-oka, Suita, Osaka

ips_cornea@ophthal.med.osaka-u.ac.jp

Soma Takeshi

Osaka University Graduate School of Medicine

2-2, Yamada-oka, Suita, Osaka

+81-6-6879-3451

ips_cornea@ophthal.med.osaka-u.ac.jp

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

1) Limbal stem-cell deficiency patients who have been classified as LSCD Stage IIB, IIC, or III
2) Aged 20 years old and over, and written informed consent has been obtained from the patient.

1) Contraindication to antimicrobials, steroid drugs, and anesthetics used in the trial
2) Allergy to antibiotics of penicillin, streptomycin, or animal (cattle, pigs, or rodent animal)
3) Test positive for Hepatitis B virus, Hepatitis C virus, Human Immunodeficiency Virus

Both

limbal stem-cell deficiency

Transplantation of iPS derived corneal epithelial cell sheet (iCEPS)

Safety(Adverse events)

Efficacy
1) LSCD staging, 2) corneal epithelial defect, 3) subjective symptom, 4) visual acuity, 5) QOL, 6) corneal opacity, 7) corneal neovascularization, 8) symblepharon

Complete

+81-6-6210-8293

Jan. 10, 2019