Age Related Macular Degeneration (AMD)NCT02464956
Production of iPSC Derived RPE Cells for Transplantation in AMD
An observational study of Age Related Macular Degeneration (AMD), run by Moorfields Eye Hospital NHS Foundation Trust.
Trial 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… Read more about this study
- Who is it for?
- Ages 50 to 100 years
- Conditions the study is for
- Age Related Macular Degeneration (AMD)
- How many people
- 3 participants
- Where
- No sites listed yet
- Study dates
- Dec 2015to Feb 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 50 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients affected by Age Related Macular Degeneration (AMD)
Who may be able to join
- Subjects over the age of 18 years
- Subjects with the ability to give informed consent
- Subjects with known AMD (both wet and dry)
Who cannot join
- 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
About this study
Official title: 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.
Read the 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.
- Conditions
- Age Related Macular Degeneration (AMD)
- Medical subject headings
- Macular Degeneration
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2015 (estimated)
- Study startDec 2015
- Primary completionAug 2018
- Study completionFeb 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Age Related Macular Degeneration (AMD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Moorfields Eye Hospital NHS Foundation Trust
- Sponsor type
- University, hospital or other
- Collaborators
- Medical Research Council
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02464956
- Sponsor's protocol ID
- DACL1011
- Status last verified
- Sep 2021
- First submitted
- 14 May 2015
- Last updated
- 12 Apr 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT02464956
- Acronym / sponsor ID
- DACL1011
- Conditions
- Age Related Macular Degeneration (AMD)
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Moorfields Eye Hospital NHS Foundation TrustUniversity, hospital or other
- Collaborators
- Medical Research Council
- Study start
- Dec 2015 (actual)
- Primary completion
- Aug 2018 (actual)
- Study completion
- Feb 2019 (actual)
- First posted
- 8 Jun 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 50 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients affected by Age Related Macular Degeneration (AMD)
Key inclusion criteria (3 listed)
- Subjects over the age of 18 years
- Subjects with the ability to give informed consent
- Subjects with known AMD (both wet and dry)
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
Time frame: 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Apr 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
An observational study of Age Related Macular Degeneration (AMD), run by Moorfields Eye Hospital NHS Foundation Trust.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Apr 2022. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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. (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Apr 2022.