Status:

COMPLETED

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

Lead Sponsor:

University of Alabama at Birmingham

Conditions:

Fabry Disease

Proteinuria

Eligibility:

All Genders

19-85 years

Brief 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 the...

Detailed Description

TITLE: FABRAZYME® + ARBS + ACE INHIBITOR TREATMENT (FAACET) STUDY Multi-center, Open-label Study of the Safety and Efficacy of Control of Proteinuria with Angiotensin Receptors Blockers (ARBs) and Ang...

Eligibility Criteria

Inclusion

  • 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:
  • 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
  • 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

  • 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.

Key Trial Info

Start Date :

March 1 2007

Trial Type :

OBSERVATIONAL

Allocation :

ACTUAL

End Date :

December 1 2012

Estimated Enrollment :

36 Patients enrolled

Trial Details

Trial ID

NCT00446862

Start Date

March 1 2007

End Date

December 1 2012

Last Update

November 19 2013

Active Locations (6)

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Page 1 of 2 (6 locations)

1

University of Alabama at Birmingham

Birmingham, Alabama, United States, 35294-0006

2

Emory University

Atlanta, Georgia, United States, 30322

3

Feinberg School of Medicine, Northwestern University

Chicago, Illinois, United States, 60614

4

University of Iowa

Iowa City, Iowa, United States, 52242