A Study Of Implantation Of Retinal Pigment Epithelium In Subjects With Acute Wet Age Related Macular Degeneration
Study Details
Study Description
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
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 1 |
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
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: 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 |
Biological: PF-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.
|
Outcome Measures
Primary Outcome Measures
- 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 Outcome Measures
- 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.
- 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
- 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 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.
- 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.
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Male and /or post-menopausal female subjects aged 60 years or above.
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Diagnosis of wet Age-related Macular Degeneration (AMD) plus rapid recent vision decline
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An informed consent document signed and dated by the subject or a legal representative.
Exclusion Criteria:
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Pregnant females; breastfeeding females; and females of childbearing potential.
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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.
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Current or previous significant other ocular disease in the study eye, as determined by the investigator.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Moorfields Eye Hospital NHS Foundation Trust | London | United Kingdom | EC1V 2PD | |
| 2 | Moorfields Eye Hospital NHS Foundation Trust | London | United Kingdom | EC1V 2PD |
Sponsors and Collaborators
- Moorfields Eye Hospital NHS Foundation Trust
- University College, London
Investigators
- Study Director: Moorfields, Moorfields Eye Hospital NHS Foundation Trust
Study Documents (Full-Text)
None provided.More Information
Additional Information:
Publications
None provided.- B4711001
- 2011-005493-37