Cerebrovascular AccidentNCT00001774
Vasodilation in Patients With Fabry's Disease
An observational study of Cerebrovascular Accident and Fabry Disease, run by National Institute of Neurological Disorders and Stroke (NINDS) at 1 site in United States.
Trial summary
Fabry's 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… Read more about this study
- Who is it for?
- Any age, men onlyHealthy volunteers can take part
- Conditions the study is for
- Cerebrovascular Accident, Fabry Disease, Healthy
- How many people
- 48 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 1997to Oct 2000
- 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 Any age
- Men only
- Healthy volunteers accepted
- Child, Adult, Older adult
Male patients with the classic form of Fabry disease, aged 18-50.
Normal male volunteers of the same approximate age will be included as a control.
No patients or volunteers with hypertension, hypercholesterolemia, diabetes, peripheral vascular disease, coagulopathy, or any other disease predisposing to vasculitis or Raynaud's phenomenon.
No volunteers who are taking any kind of medication.
Must be able to give informed consent.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institute of Neurological Disorders and Stroke (NINDS)
Bethesda, Maryland, 20892
About this study
Official title: An Investigation of Endothelium-Derived Vasodilation in Patients With Fabry's Disease
Fabry's 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 Fabry's 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 Fabry's disease.
Read the 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.
- Conditions
- Cerebrovascular Accident, Fabry Disease, Healthy
- Keywords
- Acetylcholine, Blood Flow, Blood vessel, Resistance, Sodium Nitroprusside, Fabry Disease, Normal Volunteer
- Medical subject headings
- Stroke, Fabry Disease
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Dec 2002 (estimated)
- Study startOct 1997
- Study completionOct 2000
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Cerebrovascular AccidentStudies of the same condition, open and under way.
- More trials run by National Institute of Neurological Disorders and Stroke (NINDS)The sponsor's other registered studies.
- 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
- Enrolment
- 48 participants
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00001774
- Sponsor's protocol ID
- 980013
- Other IDs
- 98-N-0013
- Status last verified
- Nov 1999
- First submitted
- 3 Nov 1999
- Last updated
- 4 Mar 2008
- Results posted
- No
Publications
Panza JA, Quyyumi AA, Brush JE Jr, Epstein SE. Abnormal endothelium-dependent vascular relaxation in patients with essential hypertension. N Engl J Med. 1990 Jul 5;323(1):22-7. doi: 10.1056/NEJM199007053230105.
PubMed 2355955Casino PR, Kilcoyne CM, Quyyumi AA, Hoeg JM, Panza JA. The role of nitric oxide in endothelium-dependent vasodilation of hypercholesterolemic patients. Circulation. 1993 Dec;88(6):2541-7. doi: 10.1161/01.cir.88.6.2541.
PubMed 8252665
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2008. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Investigation of Endothelium-Derived Vasodilation in Patients With Fabry's Disease
- ClinicalTrials.gov ID
- NCT00001774
- Acronym / sponsor ID
- 980013
- Conditions
- Cerebrovascular Accident, Fabry Disease, Healthy
- Keywords
- Acetylcholine, Blood Flow, Blood vessel, Resistance, Sodium Nitroprusside, Fabry Disease, Normal Volunteer
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 48 participants
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)US National Institutes of Health
- Study start
- Oct 1997
- Study completion
- Oct 2000
- First posted
- 10 Dec 2002 (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.
Fabry's 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 Fabry's 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 Fabry's 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 design
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
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
Not reported in the source record.
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 4 Mar 2008.
- Background: PubMed 2355955Panza JA, Quyyumi AA, Brush JE Jr, Epstein SE. Abnormal endothelium-dependent vascular relaxation in patients with essential hypertension. N Engl J Med. 1990 Jul 5;323(1):22-7. doi: 10.1056/NEJM199007053230105.
- Background: PubMed 8252665Casino PR, Kilcoyne CM, Quyyumi AA, Hoeg JM, Panza JA. The role of nitric oxide in endothelium-dependent vasodilation of hypercholesterolemic patients. Circulation. 1993 Dec;88(6):2541-7. doi: 10.1161/01.cir.88.6.2541.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cerebrovascular Accident and Fabry Disease, run by National Institute of Neurological Disorders and Stroke (NINDS) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Mar 2008. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00001774, which also lists 2 linked publications.
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 4 Mar 2008.