Age Related Macular Degeneration
Conditions
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
Interventions
BIOLOGICALPF-05206388
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.
Sponsors
University College, London
Moorfields Eye Hospital NHS Foundation Trust
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
60 Years to No maximum
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence and severity of adverse events. | 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 |
| Change in baseline in ETDRS best corrected visual acuity (BCVA) - Proportion of subjects with an improvement of 15 letters or more at Week 24. | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Position of PF-05206388 by serial biomicroscopic evaluation. | Day 2 and Weeks 1, 2, 4, 8, 10, 12, 16, 24, 36, 52 | 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 |
| Position and presence of pigmented RPE cells by serial fundus photography | Weeks 2, 4, 8, 10, 12, 16, 24, 36, 52 | 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 |
| Mean change from baseline in contrast sensitivity by Pelli Robson test | Weeks 24, 52 | The mean difference between the baseline BCVA and final BCVA in Pelli Robson letters read, across all subjects |
| 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 | 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. |
| 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 | Assessment and noting of abnormalities on Funded fluorescine angiography at weeks 4, 8, 12, 24 and 52. |
| Change in central 30 degree of visual function by Humphrey Field test. | Weeks 4, 8, 12, 24 and 52 | 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 |
| Change in thickness of RPE layer by B-mode orbital ultrasound. | Weeks 4, 8, 16, 24, 36, 52 | 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. |
| Change in liver and renal function by blood tests and liver ultrasound . | Weeks 24 and 52 | Record of any abnormalities in liver and renal function on blood testing and any abnormalities detected on the liver ultrasound. |
| Mean change of best corrected visual acuity (BCVA) from baseline by study visit. | 52 weeks | The mean difference between the baseline BCVA and final BCVA in ETDRS letters for all cases |
Countries
United Kingdom
Outcome results
None listed