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

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT02464956

Enrollment

Registered

2015-06-08

Start date

2015-12-31

Completion date

2019-02-28

Last updated

2022-04-12

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Age Related Macular Degeneration (AMD)

Brief summary

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.

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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

50 Years to 100 Years

Healthy volunteers

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

Design outcomes

Primary

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

Outcome results