Stem Cell Therapy for Outer Retinal Degenerations

Phase 1

Completed

• Conditions: Exudative Age-related Macular Degeneration; Age Related Macular Degeneration; Stargardt's Disease
• Interventions: Procedure: injection of hESC-RPE in suspension; Procedure: injection hESC-RPE seeded in a substrate

• Registration Number: NCT02903576
• Lead Sponsor: 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,

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 Timeline

• Study Start: 2015-08
• Study First Submitted: 2016-03-07
• Study First Submitted QC: 2016-09-12
• Study First Posted: 2016-09-16
• Status Verified: 2017-08
• Primary Completion: 2019-01
• Study Completion: 2019-06
• Last Update Submitted: 2020-07-20
• Last Update Posted: 2020-07-22

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 15

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
injection of hESC-RPE in suspension	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
injection hESC-RPE seeded in a substrate	injection hESC-RPE seeded in a substrate	15 patients will receive a sub-retinal implantation of a polymeric scaffold seeded hesc- RPE in monolayer

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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

Trial Locations

Locations (1)

Federal University of Sao Paulo; 🇧🇷 Sao Paulo, Brazil