A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease
Condition: Fabry Disease
MeSH terms: Fabry Disease
Intervention: Agalsidase beta
Phase: Phase 4
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 6.0
Sex: All
Minimum age: 20 Years
Maximum age: 64 Years
Healthy volunteers: No
Keywords: cardiac 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