a safety and efficacy study of fabrazyme® replacement therapy in patients with cardiac fabry disease

Conditions: Fabry Disease

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.


Eligibility criteria:
inclusion criteria: * participants definitively diagnosed with cardiac fabry disease (who fulfill all of the following criteria) * in the case of male participants, documented plasma or leukocyte alpha-galactosidase a (α-gal) activity was no more than 20 percent (%) of normal value (except for heterozygous female participants) * left ventricular hypertrophy was noted. * accumulation of globotriaosylceramide (gl-3) in the myocardium or a genetic deficiency associated with α-gal was confirmed * or in the case of heterozygous female participants, when the family (father or son) was diagnosed with fabry disease. (father or son was related by birth.) * without symptoms or signs of fabry, such as acroparesthesia, angiokeratomas, abnormal sweating, pain of distal extremities, chronic abdominal pain/diarrhea and corneal opacities were observed, except for proteinuria sign. * participants with interventricular and posterior wall thickness of at least 13 millimeter (mm) on echocardiography within 3 months before signed date to informed consent * participants in whom cardiac function was rated as class i or ii according to the new york heart association (nyha) classification when giving informed consent. * participants classification: inpatients and outpatients * participants who had given written informed consent before the study-related baseline tests. exclusion criteria: * participants with severe hypertension (for example, blood pressure more than or equal to 180 millimeter of mercury \[mmhg\] and/or blood pressure more than or equal to 110 mmhg in spite of adequate medication) * participants whose serum creatinine level was higher than the upper normal limit within 3 months (12 weeks) prior to giving informed consent. * participants who had undergone kidney transplantation or were currently on dialysis. * participants with any serious hepatic disorder. participants who had abnormal hepatic function test values within 3 months (12 weeks) prior to giving informed consent (when either alanine aminotransferase \[alt\] or aspartate aminotransferase \[ast\] level exceeded the value five times as high as the upper normal limit). * permanent pacemaker or defibrillator implanted participants * pregnant or lactating women * participants who had taken this drug for 6 months (26 weeks) or more before giving informed consent. * participants who had participated in a clinical study employing any other investigational product within 3 months prior to giving informed consent. * enzyme replacement therapy history, except for agalsidase beta * participants who were unwilling to comply with the requirements of the protocol. * others judged by the investigator or sub-investigator to be ineligible for the study

Keywords: cardiac fabry disease