Ignite Creation Date:
2024-05-05 @ 10:00 AM
Last Modification Date:
2024-10-26 @ 9:02 AM
Study NCT ID:
NCT00001774
Status:
COMPLETED
Last Update Posted:
2008-03-04
First Post:
1999-11-03
Brief Title:
Vasodilation in Patients With Fabrys Disease
Sponsor:
National Institute of Neurological Disorders and Stroke NINDS
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
An Investigation of Endothelium-Derived Vasodilation in Patients With Fabrys Disease
Status:
COMPLETED
Status Verified Date:
1999-11
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:
Fabrys disease a genetic disorder X-linked recessive due to the absence of the enzyme alpha-galactosidase A The disease is characterized by abnormal collections of glycolipids in cells histiocytes within blood vessel walls tumors on the thighs buttocks and genitalia decreased sweating tingling sensations in the extremities and cataracts Patients with Fabry s disease die from complications of the kidney heart or brain
The objective of this study is to test the belief that patients with Fabrys disease have a problem with blood vessels becoming larger The walls of blood vessels contain muscles that when they relax the vessel becomes larger This process is referred to as vasodilation It is controlled by a substance released by cells in blood vessels called EDRF endothelium-derived relaxing factor
Several drugs can affect vasodilation Researchers believe some drugs may work by blocking the affect of EDRF Researchers would like to test the effects of these drugs on the blood vessels of normal volunteers and patients with Fabrys disease
The objective of this study is to test the belief that patients with Fabrys disease have a problem with blood vessels becoming larger The walls of blood vessels contain muscles that when they relax the vessel becomes larger This process is referred to as vasodilation It is controlled by a substance released by cells in blood vessels called EDRF endothelium-derived relaxing factor
Several drugs can affect vasodilation Researchers believe some drugs may work by blocking the affect of EDRF Researchers would like to test the effects of these drugs on the blood vessels of normal volunteers and patients with Fabrys disease
Detailed Description:
Fabry disease is a systematic genetic disease in which patients have abnormal blood vessels and leads to numerous complications including cerebrovascular strokes The objective of this study is to test the hypothesis that patients with Fabry disease have abnormal endothelial-derived vasodilation If found to be abnormal endothelial-derived vasodilation will serve as a useful clinical outcome measure in the evaluation of the efficacy of specific treatment of Fabry disease and possibly of other causes of cerebrovascular stroke The endothelium modulates vascular tone by the release of contracting and relaxing substances that act on the underlying smooth muscle It has been previously demonstrated that patients with essential hypertension have a blunted vascular response to acetylcholine an endothelium-dependent vasodilator In the present study we shall analyze the regional vascular responses to acetylcholine and sodium nitroprusside alone and in the presence of L-NMMA an inhibitor of the synthesis of EDRF by endothelial cells in 12 patients with Fabry disease and 12 normal age matched control subjects We will infuse drugs into the brachial artery and will measure the responses of the forearm vasculature by means of strain gauge plethysmography Forearm blood flow and vascular resistance at baseline and after infusion of vasoactive drugs in Fabry patients will be compared to the responses obtained in the healthy control population This study will be performed with collaboration of Dr Julio A Panza Senior Clinical Investigator from the Cardiology Branch NHLBI
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 |
|---|---|---|---|
| 98-N-0013 | None | None | None |