AMDNCT03372746
Generation of Induced Pluripotent Stem (iPS) Cell Lines From Skin Fibroblast Cells of Participants With Age-Related Macular Degeneration
An observational study of AMD, run by National Eye Institute (NEI) at 14 sites in United States.
Trial summary
Background: Age-related macular degeneration (AMD) is the leading cause of blindness in the United States. Currently, there is no safe way to obtain cells from the eye to study. But researchers now can turn other types of cells, like skin or blood, into induced pluripotent stem (iPS) cells. Read more about this study
- Who is it for?
- Ages 50 years and older
- Conditions the study is for
- AMD
- How many people
- 187 participants
- Where
- 14 sitesUnited States
- Study dates
- 23 May 2018to 21 Mar 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: 100 participants across multiple sites with AMD from the original cohort of study participants will be enrolled in the AREDS2 who are returning for a 10 year in-clinic study visit and have donated DNA in the AREDS2 study
Who may be able to join
- To be eligible, the following inclusion criteria must be met, where applicable.
- AREDS2 participants who have provided DNA samples. A list will be generated based upon the results, picking the top GWAS results from this cohort.
- Participant must understand and sign the protocol s informed consent document.
- Participant is able to provide 20 ml blood sample.
Who cannot join
- A participant is not eligible if any of the following exclusion criteria are present.
- 1. Participant is unable to comply with study procedures.
Trial locations and contacts
14 sites in 1 country.
14 sites in 1 country
United States14 sites
Emory University Eye Center
Atlanta, Georgia, 30322
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
Retina-Vitreous Associates Med Group
Beverly Hills, California, 90211
Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, 02114-3096
Charlotte Eye Ear Nose & Throat Associates
Charlotte, North Carolina, 28210
Texas Retina Associates
Dallas, Texas, 75231
Vision Research ROPARD Foundation/Associated Retinal Consultants, Grand Rapids
Grand Rapids, Michigan, 49546
Retina Vitreous Associates of Kentucky
Lexington, Kentucky, 40509
University of Wisconsin-Madison
Madison, Wisconsin, 53705
Bascom Palmer Eye Institute
Miami, Florida, 33136
Vision Research ROPARD Foundation/Associated Retinal Consultants, Novi
Novi, Michigan, 48375
Casey Eye Institute
Portland, Oregon, 97210
Elman Retina Group, P.A.
Rosedale, Maryland, 21237
John Moran Eye Center, University of Utah
Salt Lake City, Utah, 84132
About this study
Official title: Generation of Induced Pluripotent Stem (iPS) Cell Lines From Skin Fibroblast Cells of Participants With Age-Related Macular Degeneration: An Ancillary Study to the Age-Related Eye Disease Study-2 (AREDS2)
Background:
Age-related macular degeneration (AMD) is the leading cause of blindness in the United States. Currently, there is no safe way to obtain cells from the eye to study. But researchers now can turn other types of cells, like skin or blood, into induced pluripotent stem (iPS) cells. These can be grown in a lab and turned into other types of cells, like cells from the eye. This will allow researchers to understand and treat diseases of the eye such as AMD.
Read more
Objectives:
To establish a bank of samples that can be changed into other cell types, such as eye cells, to better understand diseases such as AMD. Also to test drugs in order to treat various eye diseases.
Eligibility:
People who provided DNA samples in another protocol (07-EI-0025)
Design:
Participants will be screened with their data from the previous protocol. Participants with select genetic variants will be chosen and contacted via phone.
Participants will have a punch skin biopsy. The skin will be washed. A numbing medication will be injected. A small piece of skin will be removed with a biopsy tool. The site will be covered with a dressing. They will receive instructions on how to care for the area. They will have follow-up visits if needed for clinical care for the area.
Participants may be asked to return if their first sample did not provide enough cells for the lab.
Participants sample will be developed into eye cells. The cells will be used to understand diseases and test new drugs.
Read the detailed description
Objective: This ancillary study will establish a repository of biospecimens to generate induced pluripotent stem (iPS) cells that can be differentiated into ocular cell types, to be used for study of molecular mechanisms of and development of treatments for age-related macular degeneration (AMD). This repository will allow the cells to be used to perform high throughput drug screens to identify novel potential therapeutic compounds. Although research involving multiple different ocular cell types from these patients may be performed, the vast majority of the work will be centered on the retinal pigment epithelium (RPE) and neural retina. RPE and/or neural retinal cells generated from the iPS cells of participants with AMD will be used to analyze molecular mechanisms involved in disease initiation and progression.
Study Population: We plan to recruit 100 participants across multiple sites with AMD from the original cohort of study participants enrolled in the AREDS2 who are returning for a 10 year in-clinic study visit and have donated DNA in the AREDS2 study. Up to two participants will be enrolled at NEI. Participants with the highest genetic burden as well as those with rare variants will be included in the population.
Design: A 520 blood sample will be collected from 350 participants with specific genetic variants that are identified prior to the start of the study. Up to 60 participants will be enrolled at NEI. All of these participants were previously enrolled in the AREDS2 protocol (07-EI-0025) and they are returning for a 10-year in-clinic study visit for further phenotyping and for assessing the long-term effects of the ARESD2 supplements. Collected samples will be used to analyze molecular mechanisms involved in disease initiation and progression. In addition, the iPS cell-derived ocular cells may be used to perform high throughput (HTP) drug screens aimed at suppressing the molecular phenotypes of the disease and to identify potential therapeutic agents for these diseases. This study will typically require only one visit by each participant.
Outcome Measures: The primary outcome is to develop a repository for iPS cells for investigators involved in vision research. Secondary outcomes include the assessment of potential therapies for the treatment of age related macular disorder (AMD). There are no specific participant-based clinical outcomes for this protocol. Participants will, in general, be seen only once for this protocol, as this is an ancillary study to the main study.
- Conditions
- AMD
- Keywords
- Biopsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Participants across multiple sites with AMD from the original cohort of study participants enrolled in the AREDS2 |
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary outcome is to develop a repository for iPS cells for investigators involved in vision research.
Measured: Ongoing
Secondary outcomes (1)
Secondary outcomes include the assessment of potential therapies for the treatment of age related macular disorder (AMD).
Measured: Ongoing
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Dec 2017
- Study start23 May 2018
- Primary completion21 Mar 2019
- Study completion21 Mar 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AMDStudies 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
- 187 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Eye Institute (NEI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Emily Y Chew, M.D., Principal investigator, National Eye Institute (NEI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03372746
- Sponsor's protocol ID
- 180027
- Other IDs
- 18-EI-0027
- Status last verified
- Mar 2019
- First submitted
- 13 Dec 2017
- Last updated
- 5 Apr 2019
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Apr 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Generation of Induced Pluripotent Stem (iPS) Cell Lines From Skin Fibroblast Cells of Participants With Age-Related Macular Degeneration: An Ancillary Study to the Age-Related Eye Disease Study-2 (AREDS2)
- ClinicalTrials.gov ID
- NCT03372746
- Acronym / sponsor ID
- 180027
- Conditions
- AMD
- Keywords
- Biopsy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 187 participants (actual)
- Lead sponsor
- National Eye Institute (NEI)US National Institutes of Health
- Study start
- 23 May 2018 (actual)
- Primary completion
- 21 Mar 2019 (actual)
- Study completion
- 21 Mar 2019 (actual)
- First posted
- 14 Dec 2017 (actual)
- 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.
Background:
Age-related macular degeneration (AMD) is the leading cause of blindness in the United States. Currently, there is no safe way to obtain cells from the eye to study. But researchers now can turn other types of cells, like skin or blood, into induced pluripotent stem (iPS) cells. These can be grown in a lab and turned into other types of cells, like cells from the eye. This will allow researchers to understand and treat diseases of the eye such as AMD.
Objectives:
To establish a bank of samples that can be changed into other cell types, such as eye cells, to better understand diseases such as AMD. Also to test drugs in order to treat various eye diseases.
Eligibility:
People who provided DNA samples in another protocol (07-EI-0025)
Design:
Participants will be screened with their data from the previous protocol. Participants with select genetic variants will be chosen and contacted via phone.
Participants will have a punch skin biopsy. The skin will be washed. A numbing medication will be injected. A small piece of skin will be removed with a biopsy tool. The site will be covered with a dressing. They will receive instructions on how to care for the area. They will have follow-up visits if needed for clinical care for the area.
Participants may be asked to return if their first sample did not provide enough cells for the lab.
Participants sample will be developed into eye cells. The cells will be used to understand diseases and test new drugs.
Detailed description
Objective: This ancillary study will establish a repository of biospecimens to generate induced pluripotent stem (iPS) cells that can be differentiated into ocular cell types, to be used for study of molecular mechanisms of and development of treatments for age-related macular degeneration (AMD). This repository will allow the cells to be used to perform high throughput drug screens to identify novel potential therapeutic compounds. Although research involving multiple different ocular cell types from these patients may be performed, the vast majority of the work will be centered on the retinal pigment epithelium (RPE) and neural retina. RPE and/or neural retinal cells generated from the iPS cells of participants with AMD will be used to analyze molecular mechanisms involved in disease initiation and progression.
Study Population: We plan to recruit 100 participants across multiple sites with AMD from the original cohort of study participants enrolled in the AREDS2 who are returning for a 10 year in-clinic study visit and have donated DNA in the AREDS2 study. Up to two participants will be enrolled at NEI. Participants with the highest genetic burden as well as those with rare variants will be included in the population.
Design: A 520 blood sample will be collected from 350 participants with specific genetic variants that are identified prior to the start of the study. Up to 60 participants will be enrolled at NEI. All of these participants were previously enrolled in the AREDS2 protocol (07-EI-0025) and they are returning for a 10-year in-clinic study visit for further phenotyping and for assessing the long-term effects of the ARESD2 supplements. Collected samples will be used to analyze molecular mechanisms involved in disease initiation and progression. In addition, the iPS cell-derived ocular cells may be used to perform high throughput (HTP) drug screens aimed at suppressing the molecular phenotypes of the disease and to identify potential therapeutic agents for these diseases. This study will typically require only one visit by each participant.
Outcome Measures: The primary outcome is to develop a repository for iPS cells for investigators involved in vision research. Secondary outcomes include the assessment of potential therapies for the treatment of age related macular disorder (AMD). There are no specific participant-based clinical outcomes for this protocol. Participants will, in general, be seen only once for this protocol, as this is an ancillary study to the main study.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | – | –Participants across multiple sites with AMD from the original cohort of study participants enrolled in the AREDS2 |
Who may qualify
- Ages
- 50 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- 100 participants across multiple sites with AMD from the original cohort of study participants will be enrolled in the AREDS2 who are returning for a 10 year in-clinic study visit and have donated DNA in the AREDS2 study
Key inclusion criteria (4 listed)
- To be eligible, the following inclusion criteria must be met, where applicable.
- AREDS2 participants who have provided DNA samples. A list will be generated based upon the results, picking the top GWAS results from this cohort.
- Participant must understand and sign the protocol s informed consent document.
- Participant is able to provide 20 ml blood sample.
Key exclusion criteria (2 listed)
- A participant is not eligible if any of the following exclusion criteria are present.
- 1. Participant is unable to comply with study procedures.
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
The primary outcome is to develop a repository for iPS cells for investigators involved in vision research.
Time frame: Ongoing
1 secondary outcome
Secondary outcomes include the assessment of potential therapies for the treatment of age related macular disorder (AMD).
Time frame: Ongoing
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 5 Apr 2019.
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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.
Emily Y Chew, M.D. principal investigator
National Eye Institute (NEI)
14 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of AMD, run by National Eye Institute (NEI) at 14 sites in United States.
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 4 inclusion and 2 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 5 Apr 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
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?
The primary outcome is to develop a repository for iPS cells for investigators involved in vision research. (time frame: Ongoing). 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 5 Apr 2019; AzTrials last refreshed its copy on 6 Nov 2026.