Antiproteinuric Agents and Fabry Disease

NCT ID: NCT00343577

Last Updated: 2013-11-19

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 12 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2001-01-31
• Study Completion Date: 2006-12-31

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.

Conditions

Fabry Disease; Proteinuria

Study Design

• Study Time Perspective: PROSPECTIVE

Eligibility Criteria

Inclusion Criteria

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

Exclusion Criteria

• s/p kidney transplant

• Minimum Eligible Age: 14 Years
• Maximum Eligible Age: 95 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of Alabama at Birmingham

OTHER

Sponsor Role: lead

Principal Investigators

David G Warnock, MD

Role: PRINCIPAL_INVESTIGATOR

University of Alabama at Birmingham

Locations

University of Alabama at Birmingham

Birmingham, Alabama, United States

Countries

United States

References

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. doi: 10.1681/ASN.2006121400. Epub 2007 Jul 26.

Reference Type: RESULT

PMID: 17656478 (View on PubMed)

Other Identifiers

X050202007

Identifier Type: -

Identifier Source: org_study_id