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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00446862
• Date of registration: 11/03/2007
• Prospective Registration: No
• Primary sponsor: University of Alabama at Birmingham
• Public title: The Fabrazyme® and Arbs and ACE Inhibitor Treatment (FAACET) Study FAACET
• Scientific title: Multi-center, Open-label Study of the Safety and Efficacy of Control of Proteinuria With ACE Inhibitors and ARBS in Patients With Fabry Disease Who Are Receiving Fabrazyme®: The FAACET Study
• Date of first enrolment: March 2007
• Target sample size: 36
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00446862
• Study type: Observational
• Study design: Observational Model: Case Control, Time Perspective: Prospective
• Phase: N/A

Countries of recruitment

Germany, Slovenia, United States

Contacts

• Name: David G Warnock, MD
• Affiliation: University of Alabama at Birmingham

Key inclusion & exclusion 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 aGAL activity of < 1.5 nmol/hr/mL, or
leukocyte aGAL 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.



Age minimum: 19 Years
Age maximum: 85 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Fabry Disease

Proteinuria

Intervention(s)

Drug: enalapril and other angiotensin converting enzyme inhibitors; losartan and other angiotensin receptor blockers

Primary Outcome(s)

Determine the relationship of GFR to titrating ACEI/ARB therapy to targeted uring protein ration [Time Frame: from baseline]

Secondary Outcome(s)

Determine if GFR decline can be slowed by titrating ACEI/ARB therapy to target urine protein/creatinine ratio = 0.5, or 50% reduction [Time Frame: baseline to first visit]

Decrease in protein/creatinine ratio after administration of AECI/ARB therapy [Time Frame: from baseline]

Determine if urine protein/creaine reduction results in slower GFR decline [Time Frame: from baseline]

Secondary ID(s)

UAB NEPHROLOGY 001-2006

X070104001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors