Stem Cell Therapy for Outer Retinal Degenerations

July 20, 2020 updated by: Rubens Belfort Jr., Federal University of São Paulo

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

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,

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

15

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Brazil
      • Sao Paulo, Brazil, 04023-062
        • Federal University of São Paulo

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 90 years (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NON_RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: NONE

Arms and Interventions

Participant Group / 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

What is the study measuring?

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.
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
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)
Time Frame: 1 year
Inflammation/rejection Cell migration/differentiation Tumor formation Proliferative vitreoretinopathy/Retinal detachment Implant migration
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Federal University of São Paulo

Investigators

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

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

August 1, 2015

Primary Completion (ACTUAL)

January 1, 2019

Study Completion (ACTUAL)

June 1, 2019

Study Registration Dates

First Submitted

March 7, 2016

First Submitted That Met QC Criteria

September 12, 2016

First Posted (ESTIMATE)

September 16, 2016

Study Record Updates

Last Update Posted (ACTUAL)

July 22, 2020

Last Update Submitted That Met QC Criteria

July 20, 2020

Last Verified

August 1, 2017

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 12018712.5.0000.5505

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

scientific publication in a peer review journal

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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