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

Genzyme

Status and phase

Completed

Phase 4

Conditions

Fabry Disease

Treatments

Drug: Agalsidase beta

Study type

Interventional

Funder types

Industry

Identifiers

NCT00140621

AGAL03204

Details and patient eligibility

About

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.

Enrollment

6 patients

Sex

All

Ages

20 to 64 years old

Volunteers

No Healthy Volunteers

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