A Study Of Implantation Of Retinal Pigment Epithelium In Subjects With Acute Wet Age Related Macular Degeneration

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

Phase 1 trial of retinal pigment epithelium replacement in subjects with wet age-related macular degeneration in whom there is rapidly progressing vision loss

Study Overview

• Status: Recruiting
• Conditions: Age Related Macular Degeneration
• Intervention / Treatment: Biological: PF-05206388

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 Type: Interventional
• Enrollment (Anticipated): 10
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Tania West; Phone Number: 2036 02072533411; Email: moorfields.resadmin@nhs.net
• Study Contact Backup: Name: Daniela Narvaez; Phone Number: 2036 02072533411; Email: moorfields.resadmin@nhs.net

Study Locations

• United Kingdom
  • London, United Kingdom, EC1V 2PD
    • Recruiting
    • Moorfields Eye Hospital NHS Foundation Trust
    • Contact: Moorfields R&D; Phone Number: 2937 020 7253 3411; Email: moorfields.resadmin@nhs.net

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 60 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	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.	24 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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	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	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	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	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.	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.	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	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.	Weeks 4, 8, 16, 24, 36, 52

Collaborators and Investigators

• Sponsor: Moorfields Eye Hospital NHS Foundation Trust
• Collaborators: University College, London
• Investigators: Study Director: Moorfields, Moorfields Eye Hospital NHS Foundation Trust

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): October 14, 2021
• Primary Completion (Anticipated): October 10, 2023
• Study Completion (Anticipated): March 14, 2024

Study Registration Dates

• First Submitted: September 19, 2012
• First Submitted That Met QC Criteria: September 19, 2012
• First Posted (Estimate): September 24, 2012

Study Record Updates

• Last Update Posted (Actual): August 22, 2022
• Last Update Submitted That Met QC Criteria: August 18, 2022
• Last Verified: August 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Retinal Degeneration; Retinal Diseases; Macular Degeneration
• Other Study ID Numbers: B4711001; 2011-005493-37 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No
• IPD Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No