The Fabrazyme® and Arbs and ACE Inhibitor Treatment (FAACET) Study

Study Summary

The primary hypothesis is that titration of ACE inhibitor and Angiotensin Receptor Blockers (ARBs)to reduce urine protein excretion to \< 500 mg per day in Fabry Patients receiving agalsidase beta therapy at 1 mg/kg every two weeks will slow the progression rate of decline of glomerular filtration rate (GFR) compared to case controls drawn from the Genzyme-sponsored Phase III extension study (GFR 60 to 125 ml/min/1.73 m², urine protein \> 1 gram/day) or the Phase IV study (GFR 20 to 60 ml/min/1.73 m², urine protein \> 0.5 gram/day). After a 3 month initial Evaluation Phase, the patients will be followed during a 24 month Observation Phase. FAACET is an open label, prospective observational study. The primary objective is reduction of first morning urine protein/creatinine ratio to \< 0.5 gram/gram. The primary outcome measure is the regression slope of MDRD GFR with time in years

Primary Outcome

To determine whether decline of GFR can be slowed by titrating ACEI/ARB anti-proteinuric therapy to target urine protein/creatinine ratio of ≤ 0.5, or 50% reduction from baseline, in Fabry patients with significant kidney involvement (20 ≤ eGFRMDRD ≤ 60 ml/min/1.73 m²), and baseline urine protein/creatinine ratio \> 0.5 (based on the last value obtained before initiating ACEI/ARB therapy or obtained at screening before the first Evaluation Visit of the FAACET Study), who are receiving standard o

Conditions Studied

Interventions

• DRUG enalapril and other angiotensin converting enzyme inhibitors; losartan and other angiotensin receptor blockers

Study Locations (6)