Clinical Trial: A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Multicenter Open-label Study of the Safety and Efficacy of α-galactosidase A (R-h α-GAL) Replacement Therapy in Patients With Cardiac Fabry Disease

Brief Summary:

This is a multi-center, open label, phase IV study conducted to evaluate the efficacy and safety of agalsidase beta (Fabrazyme [recombinant form]) administered by intravenous drip infusion in participants with cardiac Fabry disease.

Participants participated for 4 weeks or less in the baseline period and 156 weeks for the treatment period.

Detailed Summary:
Sponsor: Genzyme, a Sanofi Company

Current Primary Outcome:

• Percent Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156 [ Time Frame: Baseline to Week 156 ]
  Interventricular septum and left ventricular posterior wall thickness was assessed by echocardiogram.
• Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156 [ Time Frame: Baseline to Week 156 ]
  Interventricular septum and left ventricular posterior wall thickness was assessed by echocardiogram.
• Percent Change From Baseline in Left Ventricular Mass (LVM) at Week 156 [ Time Frame: Baseline to Week 156 ]
  Left ventricular mass was assessed by echocardiogram.
• Change From Baseline in LVM at Week 156 [ Time Frame: Baseline to Week 156 ]
  Left ventricular mass was assessed by echocardiogram.

Original Primary Outcome:

• - To evaluate the efficacy of Fabrazyme in reducing interventricular septum and left ventricular posterior wall thickness assessed by echocardiogram
• - To evaluate the efficacy of Fabrazyme in reducing left ventricular mass (LVM) assessed by echocardiogram.

Current Secondary Outcome:

• Number of Participants in Overall Cardiac Function Assessment and Clinical Symptoms at Week 156: Change From Baseline in Cardiac Function Test [ Time Frame: Baseline to Week 156 ]
  Overall cardiac function assessment was assessed by tests (echocardiogram,cardiac catheterization (optional),electrocardiogram,B-type natriuretic peptide [BNP]), clinical symptoms (subjective symptoms) and the New York Heart Association (NYHA) cardiac functional classification.Overall assessment of cardiac function was assessed based on the evaluation items including interventricular septum thickness, left ventricular posterior wall thickness, left ventricular mass, clinical function tests and clinical symptoms. A subject was considered to be Improved: if Improved in 2 items or more, Unchanged: Improved in one item and unchanged in 2 items or unchanged in all 3 items, Aggravated: Aggravated in one item or more.
• Percent Change From Baseline in GL-3 Plasma Levels at Week 156 [ Time Frame: Baseline to Week 156 ]
• Change From Baseline in Short Form (36) Health Survey (SF-36) Scores at Week 156 [ Time Frame: Baseline to Week 156 ]
  The 36-Item Short-Form Health Survey (SF-36) is a standardized survey evaluating 8 aspects of functional health and well-being: physical and social functioning, physical and emotional role limitations, bodily pain, general health, vitality, mental health. These 8 aspects can also be summarized as physical component score (PCS) and mental component score (MCS). The score for a section is an average of the individual question scores, which are scaled 0-100 (100=highest level of functioning).

Original Secondary Outcome:

• - To make an overall evaluation of changes in cardiac function assessment by cardiac function tests (echocardiogram, cardiac catheterization, electrocardiogram, BNP), clinical symptoms (subjective symptoms) and the NYHA cardiac functional classification.
• - To evaluate the efficacy of this drug in lowering plasma globotriaosylceramide (hereinafter referred to as GL-3) level.
• - To evaluate in evaluable subjects the efficacy of this drug in reducing GL-3 accumulation in myocardial tissue.
• - To evaluate the efficacy of this drug according to SF-36 Health Survey scores.
• - To evaluate the safety of this drug.

Dates:
Date Received: August 30, 2005
Date Started: July 2005
Date Completion:
Last Updated: April 16, 2015
Last Verified: April 2015