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Stem Cell Therapy for Outer Retinal Degenerations

Sponsor
Federal University of São Paulo (Other)
Overall Status
Completed
CT.gov ID
NCT02903576
Collaborator
(none)
15
Enrollment
1
Location
2
Arms
46
Duration (Months)
0.3
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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,

Condition or Disease Intervention/Treatment Phase
  • Procedure: injection of hESC-RPE in suspension
  • Procedure: injection hESC-RPE seeded in a substrate
 Phase 1/Phase 2

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.

Study Design

Study Type:
Interventional
Actual Enrollment :
15 participants
Allocation:
Non-Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Stem Cell Derived Retinal Pigmented Epithelium Implantation in Patients With Outer Retinal Degenerations: Phase I/II Clinical Trial
Study Start Date :
Aug 1, 2015
Actual Primary Completion Date :
Jan 1, 2019
Actual Study Completion Date :
Jun 1, 2019

Arms and Interventions

Arm Intervention/Treatment
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

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.
Other Names:
  • Human Embryonic Stem Cell - Retinal Pigmented Epithelium

    		•
    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

    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
    Other Names:
  • Human Embryonic Stem Cell seeded in a polymeric substrate

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Incidence of Treatment-Emergent Adverse Events of sub retinal implantation of stem cell derived retinal pigmented epithelium in the sub retinal space. [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 Outcome Measures

    1. Incidence of side effects related to the treatment itself( injection and implantation of sub retinal stem cell related RPE) [1 year]

      Inflammation/rejection Cell migration/differentiation Tumor formation Proliferative vitreoretinopathy/Retinal detachment Implant migration

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years to 90 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Federal University of Sao Paulo Sao Paulo Brazil 04023-062

    Sponsors and Collaborators

    • Federal University of São Paulo

    Investigators

    • Study Chair: Rubens Belfort, MD, Federal University of São Paulo UNIFESP

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Rubens Belfort Jr., Full Professor of Ophthalmology, Federal University of São Paulo
    ClinicalTrials.gov Identifier:
    NCT02903576
    Other Study ID Numbers:
    • 12018712.5.0000.5505
    First Posted:
    Sep 16, 2016
    Last Update Posted:
    Jul 22, 2020
    Last Verified:
    Aug 1, 2017
    Individual Participant Data (IPD) Sharing Statement:
    Yes
    Plan to Share IPD:
    Yes
    Additional relevant MeSH terms:
    Macular Degeneration
    Retinal Degeneration
    Stargardt Disease

    Study Results

    No Results Posted as of Jul 22, 2020