Antiproteinuric Agents and Fabry Disease

Completed

• Conditions: Proteinuria; Fabry Disease

• Registration Number: NCT00343577
• Lead Sponsor: University of Alabama at Birmingham

Brief Summary

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.

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.

Study Timeline

• Study Start: 2001-01
• Status Verified: 2006-06
• Study First Submitted: 2006-06-21
• Study First Submitted QC: 2006-06-21
• Study First Posted: 2006-06-23
• Study Completion: 2006-12
• Last Update Submitted: 2013-11-17
• Last Update Posted: 2013-11-19

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

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

Exclusion Criteria

• s/p kidney transplant

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Trial Locations

Locations (1)

University of Alabama at Birmingham; 🇺🇸 Birmingham, Alabama, United States