Study of Subretinal Implantation of Human Embryonic Stem Cell-Derived RPE Cells in Advanced Dry AMD
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.
• 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.
• 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.
• To assess the feasibility of measuring the change in area of geographic atrophy over time using spectral domain optical coherence tomography or fundus autofluorescence.
Dry Macular Degeneration
|Study Design:||Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
|Official Title:||A Phase I/IIa Safety Study of Subretinal Implantation of CPCB-RPE1 (Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells Seeded on a Polymeric Substrate) in Subjects With Advanced, Dry Age-Related Macular Degeneration (AMD)|
- Frequency and Severity of Treatment-Related Adverse Events [Safety and Tolerability] [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]Comparison of product, procedure and immunosuppression related adverse events in the implanted eye to those experienced in the non-treated eye
- Visual Acuity [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]Comparison of VA changes in the treated eye versus baseline and versus the non-treated eye
- Visual Field [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]Comparison of visual field changes in the treated eye versus baseline and versus the non-treated eye
- Photoreceptor Electrical Responses [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]Comparison of multifocal electroretinogram changes in the treated eye versus baseline and versus the non-treated eye
|Study Start Date:||October 2015|
|Estimated Study Completion Date:||September 2022|
|Estimated Primary Completion Date:||September 2018 (Final data collection date for primary outcome measure)|
Experimental: CPCB-RPE1 treatment
Subretinal implantation of CPCB-RPE1 in dry AMD patients
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.
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 ≤ 20/400. 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 cohort patients will have significant central vision loss with BCVA of the eye to be implanted of ≤ 20/100 (but better than 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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02590692
|United States, California|
|Retina-Vitreous Associates Medical Group||Recruiting|
|Beverly Hills, California, United States, 90211|
|Contact: Daniel Bandary 310-289-2478 ext 3 firstname.lastname@example.org|
|Principal Investigator: Firas Rahhal, MD|
|USC Keck School of Medicine / Eye Institute||Recruiting|
|Los Angeles, California, United States, 90033|
|Contact: Yoon H Kim, BSc 323-865-6935 YoonHee.Kim@med.usc.edu|
|Contact: Nelson J Aguilar, BA 323-442-6490 email@example.com|
|Principal Investigator: Amir H Kashani, MD PhD|
|Study Director:||Peter Kondor, Ph.D.||Regenerative Patch Technologies|