Clinical Study of Subretinal Transplantation of Human Embryo Stem Cell Derived Retinal Pigment Epitheliums in Treatment of Macular Degeneration Diseases

Study Overview
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Brief Summary
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The purpose of this study was to determine the safety and therapeutic effect of sub-retinal transplantation of human embryo stem cell derived retinal pigment epitheliums (hESC-RPE) in patients with macular degeneration diseases, and explore new treatment modalities for macular degeneration diseases (Age-related macular degeneration and Stargardt's macular dystrophy).

Official Title
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Clinical Study of Subretinal Transplantation of Human Embryo Stem Cell Derived Retinal Pigment Epitheliums in Treatment of Macular Degeneration Diseases

Conditions
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Macular Degeneration, Stargardt's Macular Dystrophy

Intervention / Treatment
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* Procedure: Subretinal transplantation


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Aging from 18 to 75 years must have signed informed consent At least one visually impaired eye caused by macular degeneration diseases Can not be effectively treated with conventional therapies Best corrected visual acuity scores between 19 and 73 letter in ETDRs (early treatment diabetic retinopathy ) eye chart , including 19 and 73 (or the equivalent of Snellen eyesight from 20/400 to 20/40) Visual loss caused by macular degeneration diseases Exclusion Criteria: Eyes with concomitant diseases which will interfere the visual improvement of the study Active intraocular inflammation regardless of the grade of severity Active infection (e.g. conjunctivitis, keratitis, scleritis, uveitis, ophthalmia) History of uveitis Severe cataract, glaucoma, retinal blood vessels occlusion, retinal detachment, macular hole, vitreous-macula traction Iris neovascularization Patients who have only one functioning eye, or the best corrected vision of untreated eye scores less than 24 letters in ETDRS chart(corresponding to 20/320 in Snellen chart) History of intraocular surgery Severe systemic diseases: Stroke, coronary heart disease, angina pectoris, renal insufficiency needing dialysis Allergic to sodium fluorescein Uncontrolled hypertension (systolic pressure>140mmHg,or diastolic pressure>90mmHg) Coagulative function disorder System administration of drugs that are toxic to lens, retina, or optic nerve like hydroxychloroquine, phenothiazine, ethambutol, tamoxifen, etc. Involved in other clinical trials of any medicine within 1 month (or within 5 half-life periods) Have maternity plan in 6 months In pregnancy or lactation period.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 75 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: hESC-RPE<br>Subretinal transplantation of Human embryo stem cell derived retinal pigment epitheliums | Procedure: Subretinal transplantation<br>* Transplant hESC-RPE into subretinal space of patients with macular degeneration<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of participants with Treatment-Related Adverse Events [Safety and Tolerability] | Patients with Treatment-Related Adverse Events caused by local rejection of implanted cells or systemic immunosuppression treatment | up to 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Early Treatment Diabetic Retinopathy (ETDR ) letters participants can recognize | Visual acuity is reflected by number of ETDR letters participants can recognize | up to 12 months | 
| Visual Field as examined by Static perimetry | Area and sensitivity of visual field are detected by Static perimetry | up to 12 months | 
| Flash Electroretinogram (FERG) | Retinal electrophysiological function is tested by FERG | up to 12 months | 
| Amplitude and Latency of Flash Visual Evoked Potentials (FVEP) | Optic nerve function as assessed by FVEP | up to 12 months | 
| Multifocal Electroretinogram (MFERG) | Local retinal function as assessed by MFERG | up to 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Human embryo stem cell derived retinal pigment epitheliums, Subretinal transplantation, Wet Age related Macular degeneration, Stargardt's macular dystrophy