Antiproteinuric Agents and Fabry Disease

This study has been completed.
Sponsor:
Information provided by:
University of Alabama at Birmingham
ClinicalTrials.gov Identifier:
NCT00343577
First received: June 21, 2006
Last updated: November 17, 2013
Last verified: June 2006
  Purpose

Fabry disease is a rare disorder that often has kidney involvement with increased urine protein excretion. Proteinuria is recognized as an important risk factor for progression of chronic kidney disease. Our hypothesis is that using drugs that reduce urine protein excretion (ACE inhibitors and ARBs) will have a beneficial effect on patients with Fabry disease who already are receiving enzyme replacement therapy. A longitudinal, observational study is being undertaken to determine the utility of these agents in Fabry disease, realizing that these agents are primarily indicated for reducing systemic blood pressure, and most patients with Fabry disease have relatively low blood pressures at baseline.


Condition
Fabry Disease
Proteinuria

Study Type: Observational
Study Design: Time Perspective: Prospective
Official Title: Observational Study of Antiproteinuric Agents in Patients With Fabry Disease Treated With Enzyme Replacement Therapy

Resource links provided by NLM:


Further study details as provided by University of Alabama at Birmingham:

Estimated Enrollment: 12
Study Start Date: January 2001
Estimated Study Completion Date: December 2006
Detailed Description:

Fabry disease is a rare disorder that often has kidney involvement with increased urine protein excretion. Proteinuria is recognized as an important risk factor for progression of chronic kidney disease. Our hypothesis is that using drugs that reduce urine protein excretion (ACE inhibitors and ARBs) will have a beneficial effect on patients with Fabry disease who already are receiving enzyme replacement therapy. A longitudinal, observational study is being undertaken to determine the utility of these agents in Fabry disease, realizing that these agents are primarily indicated for reducing systemic blood pressure, and most patients with Fabry disease have relatively low blood pressures at baseline.

  Eligibility

Ages Eligible for Study:   14 Years to 95 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • genetically confirmed Fabry disease
  • institution of commercially available agalsidase-beta

Exclusion Criteria:

  • s/p kidney transplant
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00343577

Locations
United States, Alabama
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35294-0006
Sponsors and Collaborators
University of Alabama at Birmingham
Investigators
Principal Investigator: David G Warnock, MD University of Alabama at Birmingham
  More Information

Publications:
ClinicalTrials.gov Identifier: NCT00343577     History of Changes
Other Study ID Numbers: X050202007
Study First Received: June 21, 2006
Last Updated: November 17, 2013
Health Authority: United States: Institutional Review Board

Keywords provided by University of Alabama at Birmingham:
proteinuria
ARBs
agalsidase-beta
MDRD estimated GFR
Chronic kidney disease

Additional relevant MeSH terms:
Proteinuria
Fabry Disease
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on September 18, 2014