ClinicalTrials.gov
ClinicalTrials.gov Menu

Stem Cell Therapy for Outer Retinal Degenerations

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02903576
Recruitment Status : Recruiting
First Posted : September 16, 2016
Last Update Posted : August 9, 2017
Sponsor:
Information provided by (Responsible Party):
Rubens Belfort Jr., Federal University of São Paulo

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
Age Related Macular Degeneration Stargardt's Disease Exudative Age-related Macular Degeneration 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 Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 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 : August 2015
Estimated Primary Completion Date : June 2018
Estimated Study Completion Date : June 2019


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 Name: 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 Name: Human Embryonic Stem Cell seeded in a polymeric substrate




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. [ 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 Outcome Measures :
  1. 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



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years to 90 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02903576


Contacts
Contact: Rodrigo AB Fernandes, MD +5511972456473 rodrigo.brant@ophthal.com.br

Locations
Brazil
Federal University of Sao Paulo Recruiting
Sao Paulo, Brazil, 04023-062
Contact: Rodrigo AB Fernandes, MD    +5511972456473    rodrigo.brant@ophthal.com.br   
Sponsors and Collaborators
Federal University of São Paulo
Investigators
Study Chair: Rubens Belfort, MD Federal University of São Paulo (UNIFESP)

Responsible Party: Rubens Belfort Jr., Full Professor of Ophthalmology, Federal University of São Paulo
ClinicalTrials.gov Identifier: NCT02903576     History of Changes
Other Study ID Numbers: 12018712.5.0000.5505
First Posted: September 16, 2016    Key Record Dates
Last Update Posted: August 9, 2017
Last Verified: August 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: scientific publication in a peer review journal

Additional relevant MeSH terms:
Macular Degeneration
Wet Macular Degeneration
Retinal Degeneration
Retinal Diseases
Eye Diseases