Ignite Creation Date:
2024-05-06 @ 1:24 AM
Last Modification Date:
2024-10-26 @ 11:04 AM
Study NCT ID:
NCT01808729
Status:
COMPLETED
Last Update Posted:
2013-11-05
First Post:
2013-03-07
Brief Title:
CAUSE Trial Patient Specific-Cellular Characterization of Fibromuscular Dysplasia and High-Risk Atherosclerotic Endothelium
Sponsor:
Icahn School of Medicine at Mount Sinai
Organization:
Icahn School of Medicine at Mount Sinai
Study Overview
Official Title:
The CAUSE Trial Genomics of Extreme Trait-Coronary Artery Disease Cells and Fibromuscular Dysplasia Using Induced Pluripotent Stem Cell-Derived Endothelial Cells
Status:
COMPLETED
Status Verified Date:
2013-11
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:
The purpose of this project is to see if heritable alterations in the function biology and vascular repair capacity of vascular cells make a major contribution to the burden of coronary artery disease CAD fibromuscular dysplasia FMD and other vascular diseases
In more detail FMD is a nonatherosclerotic vascular disease that primarily affects women aged 20 to 60 It commonly affects the renal and carotid arteries but may involve almost every artery in the body At the cellular level FMD is characterized by increased fibroblast proliferation and collagen deposition This study aims to define some of these cellular problems by directly studying fibroblast cells from FMD patients and healthy control subjects Similarly CAD is among the leading causes of death worldwide However a large part of the risk for CAD is unexplained It is thought that a major but undefined risk factor may be gene genomic variations causing a change in vascular cell function Here we will study important vascular cell types in patients with severe and early onset CAD in an attempt to define these problems Therefore in summary this study will look to define the various cellular-level problems that occur in patients with both in CAD and FMD These data will be linked to DNA-level analyses to ultimately attempt to define the cause of these conditions
In more detail FMD is a nonatherosclerotic vascular disease that primarily affects women aged 20 to 60 It commonly affects the renal and carotid arteries but may involve almost every artery in the body At the cellular level FMD is characterized by increased fibroblast proliferation and collagen deposition This study aims to define some of these cellular problems by directly studying fibroblast cells from FMD patients and healthy control subjects Similarly CAD is among the leading causes of death worldwide However a large part of the risk for CAD is unexplained It is thought that a major but undefined risk factor may be gene genomic variations causing a change in vascular cell function Here we will study important vascular cell types in patients with severe and early onset CAD in an attempt to define these problems Therefore in summary this study will look to define the various cellular-level problems that occur in patients with both in CAD and FMD These data will be linked to DNA-level analyses to ultimately attempt to define the cause of these conditions
Detailed Description:
The purpose of this project is to see if heritable alterations in the function biology and vascular repair capacity of vascular cells make a major contribution to the burden of coronary artery disease CAD fibromuscular dysplasia FMD and other vascular diseases
Patients will be referred for this study by their physician if heshe feels that the patient qualifies for entry into the study based upon the InclusionExclusion Criteria and is expected by their physician to be a suitable candidate This will include 1 patients with FMD and non-affected control subjects 2 patients with early onset CAD in the absence of significant CAD risk factors or matching healthy controls those with 2 cardiovascular risk factors and no CAD those with angiographically normal coronary arteries Also as an extension of this study patients with rare undiagnosed or unusual forms of CAD eg unexplained dissection fulminant calcification aneurysms etc and appropriate controls will be recruited particularly if there is a strong family pedigree
This study will include collection of whole blood for subsequent DNA isolation and sequencing a plasma sample and a skin biopsy Blood will be handled in a standard fashion to obtain DNA from leukocytes and plasma These will be stored pending later batched analysis Once the skin biopsy tissue is collected the tissue will be sent to the lab for further processing The initial step is that we will derive fibroblasts from the skin biopsies In brief the biopsies are washed cut into small fragments are distributed on a culture dish with growth medium and incubated at 37C Over the next 4 - 6 weeks fibroblasts progressively grow and can be collected We may then induce these fibroblasts to undergo changes so that they become stem cells called induced pluripotent stem cells or iPSCs Once we have made iPSCs we can then make endothelial cells iPSC-ECs or in fact many other cell types The cells can be frozen until analysis or until experiments are done in the future All of the derived cells fibroblasts iPSCs and iPSC-ECs generated under this protocol will be kept indefinitely and may be used for future studies into the causes and other aspects of FMD or CAD
This study is not concerned with any clinical events after patient enrollment Only clinical events that have occurred prior to enrollment eg prior myocardial infarction stroke dissection will be recorded Once we have obtained these cells detailed cellular and molecular analyses will be performed to study the particular cellular defects that are associated with these differing conditions This data will be combined with the DNA- and plasma-derived data in an attempt to define the underlying basis for these disorders
As this is not a treatment no alternative treatment options apply The subject can decide not to participate in the trial No benefit can be promised to any subject in this study The information gained may benefit others with the same condition
Patients will be referred for this study by their physician if heshe feels that the patient qualifies for entry into the study based upon the InclusionExclusion Criteria and is expected by their physician to be a suitable candidate This will include 1 patients with FMD and non-affected control subjects 2 patients with early onset CAD in the absence of significant CAD risk factors or matching healthy controls those with 2 cardiovascular risk factors and no CAD those with angiographically normal coronary arteries Also as an extension of this study patients with rare undiagnosed or unusual forms of CAD eg unexplained dissection fulminant calcification aneurysms etc and appropriate controls will be recruited particularly if there is a strong family pedigree
This study will include collection of whole blood for subsequent DNA isolation and sequencing a plasma sample and a skin biopsy Blood will be handled in a standard fashion to obtain DNA from leukocytes and plasma These will be stored pending later batched analysis Once the skin biopsy tissue is collected the tissue will be sent to the lab for further processing The initial step is that we will derive fibroblasts from the skin biopsies In brief the biopsies are washed cut into small fragments are distributed on a culture dish with growth medium and incubated at 37C Over the next 4 - 6 weeks fibroblasts progressively grow and can be collected We may then induce these fibroblasts to undergo changes so that they become stem cells called induced pluripotent stem cells or iPSCs Once we have made iPSCs we can then make endothelial cells iPSC-ECs or in fact many other cell types The cells can be frozen until analysis or until experiments are done in the future All of the derived cells fibroblasts iPSCs and iPSC-ECs generated under this protocol will be kept indefinitely and may be used for future studies into the causes and other aspects of FMD or CAD
This study is not concerned with any clinical events after patient enrollment Only clinical events that have occurred prior to enrollment eg prior myocardial infarction stroke dissection will be recorded Once we have obtained these cells detailed cellular and molecular analyses will be performed to study the particular cellular defects that are associated with these differing conditions This data will be combined with the DNA- and plasma-derived data in an attempt to define the underlying basis for these disorders
As this is not a treatment no alternative treatment options apply The subject can decide not to participate in the trial No benefit can be promised to any subject in this study The information gained may benefit others with the same condition
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 |
|---|---|---|---|
| HS 11-02041 | None | None | None |