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

• Status: Completed
• Trial end date: 2012-12-01
• Target enrollment: 0
• Participant gender: All

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

• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Alabama at Birmingham
• Treatments: Angiotensin Receptor Antagonists; Angiotensin-Converting Enzyme Inhibitors; Enalapril; Enzyme Inhibitors; Losartan

Criteria

Inclusion Criteria:

• The patient must provide written, informed consent, and be ≥ 19 yrs of age.

• The patient is already receiving Fabrazyme® at 1 mg/kg every two weeks at the time of enrollment.

• Patient has confirmed Fabry disease (plasma αGAL activity of < 1.5 nmol/hr/mL, or leukocyte αGAL activity of < 4 nmol/hr/mg), or a known mutation compatible with Fabry disease.

• Patients with either:

1. eGFRMDRD ≥ 20 and ≤ 60 ml/min/1.73 m2, and documented 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; or

2. eGFRMDRD ≤ 125 ml/min/1.73 m2 and > 60 ml/min/1.73 m2 with documented baseline urine protein/creatinine ratio > 1, based on the last value obtained before initiating ACEI/ARB therapy or obtained at screening before the first Evaluation Visit of the FAACET Study.

Exclusion Criteria:

• The patient has undergone kidney transplantation or is currently on dialysis, or is planning on receiving a kidney transplant during the first year of the study.

• The patient has diabetic nephropathy or the presence of another, confounding kidney disease unless there is kidney biopsy confirmation that the patient does not have diabetic nephropathy or another, confounding kidney disease.

• The patient has a clinically significant organic disease, or other condition that in the opinion of the investigator would preclude participation in the full extent of the trial.

• The patient is unwilling to comply with the requirements of the protocol, including continuing on Fabrazyme® at 1 mg/kg body weight every two weeks.

• Patients who have documented allergies to ACE inhibitors and to ARBs are not eligible to participate in the FAACET Study.

• The patient is pregnant or intends to become pregnant during the course of the study.