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A Study of Implantation of Retinal Pigment Epithelium in Subjects with Acute Wet Age Related Macular Degeneration

Phase 1
Completed
Conditions
Age Related Macular Degeneration
Interventions
Registration Number
NCT01691261
Lead Sponsor
Moorfields Eye Hospital NHS Foundation Trust
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

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
9
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.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
TreatmentPF-05206388PF-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
Primary Outcome Measures
NameTimeMethod
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.

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

Secondary Outcome Measures
NameTimeMethod
Position and presence of pigmented RPE cells by serial fundus photographyWeeks 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 testWeeks 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.

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

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.

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

Change in baseline in ETDRS best corrected visual acuity (BCVA) - Proportion of subjects with an improvement of 15 letters or moreWeeks 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 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

Trial Locations

Locations (1)

Moorfields Eye Hospital NHS Foundation Trust

🇬🇧

London, United Kingdom

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