Production of iPSC Derived RPE Cells for Transplantation in AMD

Feasibility of Production of Induced Pluripotent Stem Cell Derived Retinal Pigment Epithelial Cells Fulfilling Regulatory Requirements for Human Transplantation in Dry Age-related Macular Degeneration

No treatments exist for Age-related Macular Degeneration (AMD) when there is cell loss such as in late wet AMD or dry AMD. The differentiation of RPE cells from patient derived iPSC will offer valuable source of tissue for transplantation in these forms of AMD and may form basis for a future treatment option in terms of cell transplantation.

Study Overview

• Status: Completed
• Conditions: Age Related Macular Degeneration (AMD)

Detailed Description

It has been shown that adult human skin cells or human blood cells can be reprogrammed to become stem cells. This type of stem cell is called an induced Pluripotent Stem Cell (iPSC).It has been shown specifically that this type of stem cell can be produced from adult human skin cells or blood cells can in turn be converted into Retinal Pigment Epithelial (RPE) cells. These RPE cells are a very important cell type in the human retina that are abnormal and ultimately lost in Age-related Macular Degeneration (AMD). Although it has been shown to be possible to make RPE cells from adult human skin, it has not been done to a level to fulfil regulatory requirements for human transplantation in AMD.

This is a feasibility study involving 10 patients. The efficiency of creating an iPSC-Derived RPE cells from a patient's own skin or blood will be examined. A sample size of 10 patients will allow us to potentially consolidate the safety and efficacy of this method in order to create these cells within the context of a future transplantation trial.

• Study Type: Observational
• Enrollment (Actual): 3

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 50 years to 100 years (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Patients affected by Age Related Macular Degeneration (AMD)

Description

Inclusion Criteria:

• Subjects over the age of 18 years
• Subjects with the ability to give informed consent
• Subjects with known AMD (both wet and dry)

Exclusion Criteria:

• Unable to give informed consent
• Subjects unable to give blood sample for medical reasons
• Subjects with coagulopathies
• Subjects who are known to be at risk of keloid scarring

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Successful production of a Retinal Epithelial (RPE) layer that fulfils Regulatory Regulation for transplantation. This will be confirmed with standard laboratory characterisation of RPE and completed toxicity and safety studies on the cell layer.	1 year

Collaborators and Investigators

• Sponsor: Moorfields Eye Hospital NHS Foundation Trust
• Collaborators: Medical Research Council

Study record dates

Study Major Dates

• Study Start (ACTUAL): December 1, 2015
• Primary Completion (ACTUAL): August 1, 2018
• Study Completion (ACTUAL): February 1, 2019

Study Registration Dates

• First Submitted: May 14, 2015
• First Submitted That Met QC Criteria: June 3, 2015
• First Posted (ESTIMATE): June 8, 2015

Study Record Updates

• Last Update Posted (ACTUAL): April 12, 2022
• Last Update Submitted That Met QC Criteria: April 4, 2022
• Last Verified: September 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Retinal Degeneration; Retinal Diseases; Macular Degeneration
• Other Study ID Numbers: DACL1011

Drug and device information, study documents

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