Stem Cell Therapy for Outer Retinal Degenerations
Overview
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,
Full Title of Study: “Stem Cell Derived Retinal Pigmented Epithelium Implantation in Patients With Outer Retinal Degenerations: Phase I/II Clinical Trial”
Study Type
- Study Type: Interventional
- Study Design
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Primary Purpose: Treatment
- Masking: None (Open Label)
- Study Primary Completion Date: January 2019
Detailed Description
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.
Interventions
- Procedure: injection of hESC-RPE in suspension
- 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.
- Procedure: injection hESC-RPE seeded in a substrate
- 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
Arms, Groups and Cohorts
- Active Comparator: injection of hESC-RPE in suspension
- 6 patients will receive cell suspension injections on the sub retinal space prior to the surgeries, to access safety
- Active Comparator: injection hESC-RPE seeded in a substrate
- 15 patients will receive a sub-retinal implantation of a polymeric scaffold seeded hesc- RPE in monolayer
Clinical Trial Outcome Measures
Primary Measures
- Incidence of Treatment-Emergent Adverse Events of sub retinal implantation of stem cell derived retinal pigmented epithelium in the sub retinal space.
- Time Frame: 1 year
- Incidence of surgical related side effects: Retinal detachment, Ocular inflammation, Increase in intraocular pressure, Infection( endophthalmitis), Loss of vision due to surgical, related complications
Secondary Measures
- Incidence of side effects related to the treatment itself( injection and implantation of sub retinal stem cell related RPE)
- Time Frame: 1 year
- Inflammation/rejection Cell migration/differentiation Tumor formation Proliferative vitreoretinopathy/Retinal detachment Implant migration
Participating in This Clinical Trial
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
Gender Eligibility: All
Minimum Age: 18 Years
Maximum Age: 90 Years
Are Healthy Volunteers Accepted: No
Investigator Details
- Lead Sponsor
- Federal University of São Paulo
- Provider of Information About this Clinical Study
- Principal Investigator: Rubens Belfort Jr., Full Professor of Ophthalmology – Federal University of São Paulo
- Overall Official(s)
- Rubens Belfort, MD, Study Chair, Federal University of São Paulo UNIFESP
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