Phase 1, Open-label, Safety and Feasibility Study of Implantation of Pf-05206388 (human Embryonic Stem Cell Derived Retinal Pigment Epithelium (rpe) Living Tissue Equivalent) in Subjects with Acute Wet Age Related Macular Degeneration and Recent Rapid Vision Decline
Trial Snapshot
- Phase
- Phase 1
- Status
- Completed
- Enrollment
- 9
- Locations
- 1
- Primary Endpoint
- Change in baseline in ETDRS best corrected visual acuity (BCVA) - Proportion of subjects with an improvement of 15 letters or more at Week 24.
Study Overview
Brief Summary
Phase 1 trial of retinal pigment epithelium replacement in subjects with wet age-related macular degeneration in whom there is rapidly progressing vision loss
Detailed Description
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 Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 60 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Male and /or post-menopausal female subjects aged 60 years or above.
- •Diagnosis of wet Age-related Macular Degeneration (AMD) plus rapid recent vision decline
- •An informed consent document signed and dated by the subject or a legal representative.
Exclusion Criteria
- •Pregnant females; breastfeeding females; and females of childbearing potential.
- •Treatment with an investigational drug within 30 days (or as determined by the local requirement, whichever is longer) or 5 half-lives preceding the first dose of study medication.
- •Current or previous significant other ocular disease in the study eye, as determined by the investigator.
Arms & Interventions
Treatment
PF-05206388 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
Intervention: PF-05206388 (Biological)
Outcomes
Primary Outcomes
Change in baseline in ETDRS best corrected visual acuity (BCVA) - Proportion of subjects with an improvement of 15 letters or more at Week 24.
Time Frame: 24 weeks
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.
Incidence and severity of adverse events.
Time Frame: 52 weeks
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
Secondary Outcomes
- Position and presence of pigmented RPE cells by serial fundus photography(Weeks 2, 4, 8, 10, 12, 16, 24, 36, 52)
- Mean change from baseline in contrast sensitivity by Pelli Robson test(Weeks 24, 52)
- Change in liver and renal function by blood tests and liver ultrasound .(Weeks 24 and 52)
- Change in leakage or perfusion in normal fundal vasculature and presence of abnormal vasculature by fundus fluorescein angiography.(Weeks 4, 8, 12, 24 and 52)
- Position of PF-05206388 by serial biomicroscopic evaluation.(Day 2 and Weeks 1, 2, 4, 8, 10, 12, 16, 24, 36, 52)
- Change in thickness of RPE layer by B-mode orbital ultrasound.(Weeks 4, 8, 16, 24, 36, 52)
- Mean change of best corrected visual acuity (BCVA) from baseline by study visit.(52 weeks)
- Change in baseline in ETDRS best corrected visual acuity (BCVA) - Proportion of subjects with an improvement of 15 letters or more(Weeks 1,2,4,8, 12,16, 36, 52)
- Change in central 30 degree of visual function by Humphrey Field test.(Weeks 4, 8, 12, 24 and 52)