Phase 1 trial of retinal pigment epithelium replacement in subjects with wet age-related macular degeneration in whom there is rapidly progressing vision loss
Phase 1, open-label, safety and feasibility study of implantation of PF-05206388 (human embryonic stem cell derived retinal pigment epithelium) in subjects with wet age related macular degeneration and rapid vision loss
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
9
PF-05206388 will be provided as a Retinal Pigment Epithelium living tissue equivalent for intraocular use in the form of a monolayer of Retinal Pigmented Epithelial (RPE) cells immobilized on a polyester membrane. The membrane is approximately 6 mm x 3 mm and will contain a confluent layer of RPE cells, at a nominal dose of 17 mm2. The implant is intended to be life-long.
Moorfields Eye Hospital NHS Foundation Trust
London, United Kingdom
Incidence and severity of adverse events.
The number of Adverse Events (AE) and Serious Adverse Events (SAE) noted during the study and an assessment of whether they are trial product related
Time frame: 52 weeks
Change in baseline in ETDRS best corrected visual acuity (BCVA) - Proportion of subjects with an improvement of 15 letters or more at Week 24.
The number of patients with a difference between the baseline BCVA and BCVA at 24 weeks in ETDRS letters, where the differences is 15 letters or more, as a percentage of the total number of cases.
Time frame: 24 weeks
Change in baseline in ETDRS best corrected visual acuity (BCVA) - Proportion of subjects with an improvement of 15 letters or more
The number of patients with a difference between the baseline BCVA and BCVA at weeks 1,2,4,8, 12,16, 36, 52 in ETDRS letters, where the differences is 15 letters or more, as a percentage of the total number of cases.
Time frame: Weeks 1,2,4,8, 12,16, 36, 52
Mean change of best corrected visual acuity (BCVA) from baseline by study visit.
The mean difference between the baseline BCVA and final BCVA in ETDRS letters for all cases
Time frame: 52 weeks
Position of PF-05206388 by serial biomicroscopic evaluation.
Measurement of movement in millimetre and rotation in degrees measure relative to baseline, at day 2 and weeks 1, 2, 4, 8, 10, 12, 16, 24, 36 and 52
Time frame: Day 2 and Weeks 1, 2, 4, 8, 10, 12, 16, 24, 36, 52
Position and presence of pigmented RPE cells by serial fundus photography
The subjective reporting of area of pigmentation as a % with cross reference to the OCT at weeks 2, 4, 8, 10, 12, 16, 24, 36 and 52
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Time frame: Weeks 2, 4, 8, 10, 12, 16, 24, 36, 52
Mean change from baseline in contrast sensitivity by Pelli Robson test
The mean difference between the baseline BCVA and final BCVA in Pelli Robson letters read, across all subjects
Time frame: Weeks 24, 52
Change in liver and renal function by blood tests and liver ultrasound .
Record of any abnormalities in liver and renal function on blood testing and any abnormalities detected on the liver ultrasound.
Time frame: Weeks 24 and 52
Change in leakage or perfusion in normal fundal vasculature and presence of abnormal vasculature by fundus fluorescein angiography.
Assessment and noting of abnormalities on Funded fluorescine angiography at weeks 4, 8, 12, 24 and 52.
Time frame: Weeks 4, 8, 12, 24 and 52
Change in central 30 degree of visual function by Humphrey Field test.
Recording and reporting of any changes on the central 30 degree field on the automated Humphrey Field test at weeks 4, 8, 12, 24 and 52
Time frame: Weeks 4, 8, 12, 24 and 52
Change in thickness of RPE layer by B-mode orbital ultrasound.
Recording of any changes in thickness of RPE layer by B-mode orbital ultrasound carried out by the ocular oncologist or medical physicist at weeks 4, 8, 16, 24, 36, 52.
Time frame: Weeks 4, 8, 16, 24, 36, 52