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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