Stem Cell Therapy for Outer Retinal Degenerations

Federal University of São Paulo

Status and phase: Completed; Phase 2; Phase 1

Conditions: Age Related Macular Degeneration; Exudative Age-related Macular Degeneration; Stargardt's Disease

Treatments: Procedure: injection of hESC-RPE in suspension; Procedure: injection hESC-RPE seeded in a substrate

Study type: Interventional

Funder types: Other

Identifiers

NCT02903576

12018712.5.0000.5505

Details and patient eligibility

About

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 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.

Enrollment: 15 patients

Sex: All

Ages: 18 to 90 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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15 participants in 2 patient groups

injection of hESC-RPE in suspension

Active Comparator group

Description:

6 patients will receive cell suspension injections on the sub retinal space prior to the surgeries, to access safety

Treatment:

Procedure: injection of hESC-RPE in suspension

injection hESC-RPE seeded in a substrate

Active Comparator group

Description:

15 patients will receive a sub-retinal implantation of a polymeric scaffold seeded hesc- RPE in monolayer

Treatment:

Procedure: injection hESC-RPE seeded in a substrate