Study of Subretinal Implantation of Human Embryonic Stem Cell-Derived RPE Cells in Advanced Dry AMD

General Information

Summary 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.
Description 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.
Clinical trials phase Phases 1/2
Start date (estimated) 2015-10-01
End date (estimated) 2023-06-30
Clinical feature
Label atrophic macular degeneration
Link http://www.ebi.ac.uk/efo/EFO_1001492
Description Dry AMD is most common type of macular degeneration and affects 90% of the people who have the condition. In the dry form, there is a breakdown or thinning of the layer of retinal pigment epithelial cells (RPE) in the macula. No medical or surgical treatment is available for this condition.

Administrative Information

NCT number NCT02590692
ICTRP weblink http://apps.who.int/trialsearch/Trial2.aspx?TrialID=NCT02590692
Other study identifiers
Name RPT-14-01
Source weblink https://clinicaltrials.gov/ct2/show/NCT02590692
Regulatory body approval
Name Food and Drug Administration (FDA)
Country
United States
Public contact
Email inquiries@regenerativepatch.com
Public email inquiries@regenerativepatch.com
First name Jane
Last name Lebkowski
Country
United States

Cell Line

Stem cell lines obtained from

Recruitment

Recruitment Status Active, not recruiting
Estimated number of participants 16