The Fabrazyme® and Arbs and ACE Inhibitor Treatment (FAACET) Study (FAACET)
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|ClinicalTrials.gov Identifier: NCT00446862|
Recruitment Status : Completed
First Posted : March 13, 2007
Last Update Posted : November 19, 2013
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|Condition or disease||Intervention/treatment|
|Fabry Disease Proteinuria||Drug: enalapril and other angiotensin converting enzyme inhibitors; losartan and other angiotensin receptor blockers|
|Study Type :||Observational|
|Actual Enrollment :||36 participants|
|Official 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|
|Study Start Date :||March 2007|
|Actual Primary Completion Date :||June 2012|
|Actual Study Completion Date :||December 2012|
- Drug: enalapril and other angiotensin converting enzyme inhibitors; losartan and other angiotensin receptor blockers
10 mg by mouth every day; 25 mg by mouth every dayOther Name: agalsidase beta
- Determine the relationship of GFR to titrating ACEI/ARB therapy to targeted uring protein ration [ Time Frame: from baseline ]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 of care treatment for Chronic Kidney Disease (CKD), and Fabry disease.
- Decrease in protein/creatinine ratio after administration of AECI/ARB therapy [ Time Frame: from baseline ]To determine whether urine protein/creatinine ratio ≤ 0.5, or ≥ 50% reduction from baseline, can safely be achieved in Fabry patients with ACEI/ARB therapy.
- Determine if urine protein/creaine reduction results in slower GFR decline [ Time Frame: from baseline ]To determine whether urine protein/creatinine ratio ≤ 0. 5, or ≥ 50% reduction from baseline, result in a slower rate of GFR decline compared to patients who cannot achieve this target.
- 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 ]To determine if GFR decline can be slowed by titrating ACEI/ARB therapy to target urine protein/creatinine ratio ≤ 0.5, or 50% reduction from baseline, in Fabry patients with definite kidney involvement (125 ≥ eGFRMDRD ≥ 60 ml/min/1.73 m²), and baseline urine protein/creatinine ratio > 1.0 (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 of care treatment for Chronic Kidney Disease (CKD), and Fabry disease.
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|Ages Eligible for Study:||19 Years to 85 Years (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
|Sampling Method:||Non-Probability Sample|
- 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.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00446862
|United States, Alabama|
|University of Alabama at Birmingham|
|Birmingham, Alabama, United States, 35294-0006|
|United States, Georgia|
|Atlanta, Georgia, United States, 30322|
|United States, Illinois|
|Feinberg School of Medicine, Northwestern University|
|Chicago, Illinois, United States, 60614|
|United States, Iowa|
|University of Iowa|
|Iowa City, Iowa, United States, 52242|
|United States, Massachusetts|
|Massachusetts General Hospital|
|Boston, Massachusetts, United States, 02114|
|General Hospital Slovenj Gradec|
|Ljubljana, Gradec, Slovenia, 1|
|Principal Investigator:||David G Warnock, MD||University of Alabama at Birmingham|
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
|Responsible Party:||University of Alabama at Birmingham|
|Other Study ID Numbers:||
UAB NEPHROLOGY 001-2006
|First Posted:||March 13, 2007 Key Record Dates|
|Last Update Posted:||November 19, 2013|
|Last Verified:||August 2013|
Definition: Fabry disease
Definition: MDRD GFR
Definition: Chronic Kidney Disease
Definition: Enzyme Replacement Therapy
Definition: Anti-proteinuric therapy
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Lipid Metabolism Disorders
Female Urogenital Diseases
Female Urogenital Diseases and Pregnancy Complications
Male Urogenital Diseases