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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
6
Fujita Health University Hospital
Aichi, Japan
Sapporo Medical University Hospital
Hokkaido, Japan
Akune Citizen Hospital
Kagoshima, Japan
Tohoku University Hospital
Miyagi, Japan
Percent Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156
Interventricular septum and left ventricular posterior wall thickness was assessed by echocardiogram.
Time frame: Baseline to Week 156
Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156
Interventricular septum and left ventricular posterior wall thickness was assessed by echocardiogram.
Time frame: Baseline to Week 156
Percent Change From Baseline in Left Ventricular Mass (LVM) at Week 156
Left ventricular mass was assessed by echocardiogram.
Time frame: Baseline to Week 156
Change From Baseline in LVM at Week 156
Left ventricular mass was assessed by echocardiogram.
Time frame: Baseline to Week 156
Number of Participants in Overall Cardiac Function Assessment and Clinical Symptoms at Week 156: Change From Baseline in Cardiac Function Test
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.
Time frame: Baseline to Week 156
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Nihon University Itabashi Hospital
Tokyo, Japan
Nihon University Nerima Hikarigaoka Hospital
Tokyo, Japan
Yamanashi Prefectural Central Hospital
Yamanashi, Japan
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
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).
Time frame: Baseline to Week 156