Status:
COMPLETED
A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease
Lead Sponsor:
Genzyme, a Sanofi Company
Conditions:
Fabry Disease
Eligibility:
All Genders
20-64 years
Phase:
PHASE4
Brief 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 partic...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
July 1 2005
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 1 2012
Estimated Enrollment :
6 Patients enrolled
Trial Details
Trial ID
NCT00140621
Start Date
July 1 2005
End Date
August 1 2012
Last Update
May 12 2015
Active Locations (7)
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1
Fujita Health University Hospital
Aichi, Japan, 470-1192
2
Sapporo Medical University Hospital
Hokkaido, Japan, 060-8543
3
Akune Citizen Hospital
Kagoshima, Japan, 899-1611
4
Tohoku University Hospital
Miyagi, Japan, 980-8574