hRPC is a cell therapy for retinitis pigmentosa. This is a first-in-human, dose escalation study in which participants with retinitis pigmentosa will receive a single subretinal injection of hRPC cells in one eye to evaluate safety and tolerability. Participants will be followed for two years to evaluate the safety and tolerability of hRPC Additional testing will seek to establish any preliminary efficacy from hRPC.
This is a first-in-human open label phase I/II dose-escalation study in which participants with retinitis pigmentosa will receive a single uni-ocular subretinal implantation of one of three doses of hRPC. Treated eyes will be carefully monitored for any ocular or systemic adverse events for 2 years. Testing will comprise a series of detailed ophthalmic examinations and imaging together with blood testing and systemic evaluations, as necessary. Ophthalmic testing will also be evaluated for any preliminary efficacy signal.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
29
Participants will undergo vitrectomy surgery and subretinal implantation of hRPC in the study eye.
Retinal Research Institute
Phoenix, Arizona, United States
Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, United States
Oregon Health and Science University
Portland, Oregon, United States
Institut de la Màcula
Barcelona, Spain
Safety over the six months after treatment as assessed by the incidence of treatment emergent adverse events (TEAEs) and changes from baseline in other safety parameters.
Safety measures will be assessed by review of important events, including but not limited to inflammation, complications of the surgical procedure and worsening of vision.
Time frame: 6 months
Safety (Visual function measure: change in visual acuity)
A summary of the ETDRS +/- BRVT BCVA letter score and the change from baseline to end of study in the treated eye presented by treatment group.
Time frame: 24 months
Safety (Visual function measure: change in visual field: Goldmann visual field, microperimetry and FST)
A summary of the perimetry and change from baseline to end of study in the treated eye presented by treatment group.
Time frame: 24 months
Safety (Change in retinal sensitivity in the area overlying the implanted hRPC as compared with untreated retina)
ERG results and change from baseline to end of study summarized descriptively and presented by treatment group.
Time frame: 24 months
Safety (Anatomical endpoint relating to retinal function in implant location - Color Fundus Photography)
A qualitative description of the change in retinal appearance of treated and untreated retinal area in the treated eye from baseline to end of study presented by treatment group.
Time frame: 24 months
Safety (Anatomical endpoint relating to retinal function in implant location - Fundus autofluorescence)
A qualitative description of the change in retinal appearance of treated and untreated retinal area in the treated eye from baseline to end of study presented by treatment group.
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Oxford Eye Hospital
Oxford, United Kingdom
Time frame: 24 months
Safety (Anatomical endpoint relating to retinal function in implant location - Spectral domain-OCT)
Summary of the overall retinal thickness, outer nuclear layer thickness and ellipsoid zone measurement and change from baseline to the end of study of treated retina compared with untreated retina in the treated eye by treatment group.
Time frame: 24 months