Stem Cell Therapy for Outer Retinal Degenerations

Study Overview
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Brief Summary
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This study is a Phase I/II , open label,non randomized, prospective study to determine the safety of human embryonic stem cell derived Retinal pigmented epithelium (hESC RPE) sub retinal injections versus hESC RPE seeded on a polymeric substrate implanted in the sub retinal space,

Detailed Description
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To determine whether the surgical implantation of a human embryonic stem cell-derived retinal pigmented epithelium (hESC-RPE) monolayer seeded onto a polymeric versus hESC-RPE injections into the sub retinal space is a safe procedure. 6 Patients will receive hESC-RPE cell injections (100000 cells) in the sub retinal space (2 Dry Age-related macular degeneration (AMD), 2 Wet AMD with disciform scar and 2 with Stargardt's disease). Also 5 patients Dry AMD, 5 patients with Wet AMD with disciform scar and 5 patients with Stargardt's disease with receive a subretinal implantation of the hESC-RPE seeded in a monolayer in a polymeric substrate. Patients will be enrolled sequentially, and after the procedure the patients will be followed for 1 year.

Official Title
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Stem Cell Derived Retinal Pigmented Epithelium Implantation in Patients With Outer Retinal Degenerations: Phase I/II Clinical Trial

Conditions
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Age Related Macular Degeneration, Stargardt's Disease, Exudative Age-related Macular Degeneration

Intervention / Treatment
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* Procedure: injection of hESC-RPE in suspension
* Procedure: injection hESC-RPE seeded in a substrate


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients with AMD ( Dry , Wet after failure of treatment, disciform scars) Patients with Stargardt's Disease BCVA on the selected eye: worse than 20/200 Exclusion Criteria: Other ophthalmological diseases( Glaucoma, Diabetic Retinopathy, Previous retinal surgery, uveitis) Systemic diseases with contraindication for surgical procedures with local anaesthesia

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 90 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: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: injection of hESC-RPE in suspension<br>6 patients will receive cell suspension injections on the sub retinal space prior to the surgeries, to access safety | Procedure: injection of hESC-RPE in suspension<br>* The first six patients will receive a sub retinal injection of hESC-RPE in solution after a complete pars plana vitrectomy to access safety of the cell implant alone.<br>* Other names: Human Embryonic Stem Cell - Retinal Pigmented Epithelium;|
| Active Comparator: injection hESC-RPE seeded in a substrate<br>15 patients will receive a sub-retinal implantation of a polymeric scaffold seeded hesc- RPE in monolayer | Procedure: injection hESC-RPE seeded in a substrate<br>* Fifteen patients will receive a sub-retinal implantation of embryonic stem cell derived retinal pigmented epithelium seeded in a polymeric substrate to access safety of substrate seeded with RPE<br>* Other names: Human Embryonic Stem Cell seeded in a polymeric substrate;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of Treatment-Emergent Adverse Events of sub retinal implantation of stem cell derived retinal pigmented epithelium in the sub retinal space. | Incidence of surgical related side effects: Retinal detachment, Ocular inflammation, Increase in intraocular pressure, Infection( endophthalmitis), Loss of vision due to surgical, related complications | 1 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of side effects related to the treatment itself( injection and implantation of sub retinal stem cell related RPE) | Inflammation/rejection Cell migration/differentiation Tumor formation Proliferative vitreoretinopathy/Retinal detachment Implant migration | 1 year |