Treatment of RP and LCA by Primary RPE Transplantation

Study Overview
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Brief Summary
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Early Phase I Study of the Safety and Preliminary Efficacy of Human primary Retinal Pigment Epithelial (HuRPE) Cells Subretinal Transplantation in Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA) Patients

Official Title
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Treatment of Retinitis Pigmentosa and Leber Congenital Amaurosis by Primary Retinal Pigment Epithelial Cells Transplantation

Conditions
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Leber Congenital Amaurosis, Retinitis Pigmentosa

Intervention / Treatment
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* Biological: Human primary Retinal Pigment Epithelial (HuRPE) cells


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of RP or LCA; Experimental eye with best-corrected visual acuity (BCVA) of no more than 35 letters or visual field of less than 10 degree; Patient understand and sign the consent form. Exclusion Criteria: Blood routine examination is abnormal (hemoglobin<10gm/dL; blood platelet count<100k/mm³; neutrophil count<1000/mm³) Blood biochemistry is abnormal (ALT／AST>1.5; creatinine>1.3mg/dL) Experimental eye has optic nerve atrophy caused by glaucoma Experimental eye has retinal detachment, or has received retinal detachment surgery. Patients with uveitis and other endophthalmitis Patients with other ocular disease affecting vision. Patients have participated in clinical study of ocular or systemic drug use in recent 6 months. Patients with medical history of malignant cancer (except resected basal cell carcinoma and squamous-cell carcinoma). Patients with medical history of myocardial infarction Patient with diabetes Patient with Parkinson disease or Alzheimer's disease Patients are under the treatment of immunosuppressive agent (except intermittent, low-dose corticosteroid treatment). Patients with other medical conditions that restricts the compliance and safety of patients, or affects experimental results.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 70 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
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: low dose group<br>300,000 HuRPE cells | Biological: Human primary Retinal Pigment Epithelial (HuRPE) cells<br>* HuRPE will be transplanted by a board-certified vitreoretinal surgeon, which will be administered into the subretinal space of experimental eye through a standard surgical approach.<br>|
| Experimental: middle dose group<br>500,000 HuRPE cells | Biological: Human primary Retinal Pigment Epithelial (HuRPE) cells<br>* HuRPE will be transplanted by a board-certified vitreoretinal surgeon, which will be administered into the subretinal space of experimental eye through a standard surgical approach.<br>|
| Experimental: high dose group<br>1,000,000 HuRPE cells | Biological: Human primary Retinal Pigment Epithelial (HuRPE) cells<br>* HuRPE will be transplanted by a board-certified vitreoretinal surgeon, which will be administered into the subretinal space of experimental eye through a standard surgical approach.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| best-corrected visual acuity (BCVA) |  | 12 months |