Ignite Creation Date:
2024-05-06 @ 10:53 AM
Last Modification Date:
2024-10-26 @ 12:36 PM
Study NCT ID:
NCT03372746
Status:
COMPLETED
Last Update Posted:
2019-04-05
First Post:
2017-12-13
Brief Title:
Generation of Induced Pluripotent Stem iPS Cell Lines From Skin Fibroblast Cells of Participants With Age-Related Macular Degeneration
Sponsor:
National Eye Institute NEI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
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
Status:
COMPLETED
Status Verified Date:
2019-03
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 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
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 andor 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 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 Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 18-EI-0027 | None | None | None |