The Phase I/IIa clinical trial is designed to assess the feasibility of delivery and safety of Human Embryonic Stem Cell-Derived RPE Cells on a parylene membrane (CPCB-RPE1) in patients with advanced, dry age-related macular degeneration. Primary Objective: • To test the safety and tolerability of CPCB-RPE1 during and after subretinal implantation in patients with geographic atrophy with evidence of involvement of the central fovea. Secondary Objective: • To assess visual acuity, visual field, and retinal function after CPCB-RPE1 implantation. Implanted and fellow eyes will be compared post-implantation to assess the ability of the implant to prevent disease progression. Exploratory Objectives: • To assess the feasibility of measuring the change in area of geographic atrophy over time using spectral domain optical coherence tomography or fundus autofluorescence.
The study will include two cohorts, each of 10 patients. For the first cohort, the study population will be patients with advanced, dry AMD with evidence of significant geographic atrophy involving the fovea. These patients will have significant central vision loss with best-corrected visual acuity (BCVA) of the eye to be implanted of BCVA of 20/200 or worse. Each of these patients will have substantial RPE and photoreceptor loss. Patients will be screened for overall health status to minimize risks associated with retinal surgery and any subsequent immunosuppression. As the safety and tolerability of CPCB-RPE1 is demonstrated in the first cohort, patients with less advanced disease will be recruited into a second cohort in this Phase I/IIa clinical trial. In this second cohort patients will have significant central vision loss with BCVA of the eye to be implanted of 20/80 or worse, but better than or equal to 20/400 with comparably less damage to the RPE/photoreceptor complex than Cohort 1. These patients will be screened in the same manner for overall health status to minimize risks associated with retinal surgery and any subsequent immunosuppression. Assessments of visual function will be the same as in Cohort 1.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
OTHER
Masking
NONE
Enrollment
16
Patients will receive one CPCB-RPE1 implant of approximately 100,000 differentiated RPE cells attached to a small parylene membrane. The density of cells on the membrane represents the approximate density of RPE cells in the human eye. The membrane size was chosen to cover a substantial portion of the macular region of the retina.
Retinal Consultants of Arizona LTD
Phoenix, Arizona, United States
Retina-Vitreous Associates Medical Group
Beverly Hills, California, United States
USC Keck School of Medicine / Eye Institute
Los Angeles, California, United States
Southern California Desert Retina Consultants
Palm Desert, California, United States
Orange County Retina Medical Group
Santa Ana, California, United States
California Retina Consultants
Santa Barbara, California, United States
Frequency and Severity of Treatment-Related Adverse Events [Safety and Tolerability]
Comparison of product, procedure and immunosuppression related adverse events in the implanted eye to those experienced in the non-treated eye
Time frame: 1 year
Visual Acuity
Comparison of VA changes in the treated eye versus baseline and versus the non-treated eye
Time frame: 1 year
Visual Field
Comparison of visual field changes in the treated eye versus baseline and versus the non-treated eye
Time frame: 1 year
Photoreceptor Electrical Responses
Comparison of multifocal electroretinogram changes in the treated eye versus baseline and versus the non-treated eye
Time frame: 1 year
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