Antiproteinuric Agents and Fabry Disease

Fabry Disease Clinical Trial

Official title:

Observational Study of Antiproteinuric Agents in Patients With Fabry Disease Treated With Enzyme Replacement Therapy

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00343577
• Other study ID #: X050202007
• Status: Completed
• Phase: N/A
• First received: June 21, 2006
• Last updated: November 17, 2013
• Start date: January 2001
• Est. completion date: December 2006

Study information

• Verified date: June 2006
• Source: University of Alabama at Birmingham
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 12
• Est. completion date: December 2006
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 14 Years to 95 Years

Eligibility

Inclusion Criteria:

• genetically confirmed Fabry disease

• institution of commercially available agalsidase-beta

Exclusion Criteria:

• s/p kidney transplant

Study Design

Time Perspective: Prospective

Related Conditions & MeSH terms

• Fabry Disease
• Proteinuria

Locations

Country	Name	City	State
United States	University of Alabama at Birmingham	Birmingham	Alabama

Sponsors (1)

Lead Sponsor	Collaborator
University of Alabama at Birmingham

Country where clinical trial is conducted

United States, 

References & Publications (1)

Tahir H, Jackson LL, Warnock DG. Antiproteinuric therapy and fabry nephropathy: sustained reduction of proteinuria in patients receiving enzyme replacement therapy with agalsidase-beta. J Am Soc Nephrol. 2007 Sep;18(9):2609-17. Epub 2007 Jul 26. — View Citation