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

• Status: Completed
• Trial end date: 2012-08-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Genzyme, a Sanofi Company

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