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

NCT ID: NCT00140621

Last Updated: 2015-05-12

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 6 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2005-07-31
• Study Completion Date: 2012-08-31

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 participants with cardiac Fabry disease.

Participants participated for 4 weeks or less in the baseline period and 156 weeks for the treatment period.

Conditions

Fabry Disease

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Agalsidase Beta

Agalsidase beta 1 milligram per kilogram (mg/kg) intravenously once every 2 weeks up to 156 weeks.

Group Type: EXPERIMENTAL

Agalsidase beta

Intervention Type: DRUG

Interventions

Agalsidase beta

Intervention Type: DRUG

Other Intervention Names

Fabrazyme®

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

• Minimum Eligible Age: 20 Years
• Maximum Eligible Age: 64 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Genzyme, a Sanofi Company

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Monitor

Role: STUDY_DIRECTOR

Genzyme, a Sanofi Company

Locations

Fujita Health University Hospital

Aichi, , Japan

Sapporo Medical University Hospital

Hokkaido, , Japan

Akune Citizen Hospital

Kagoshima, , Japan

Tohoku University Hospital

Miyagi, , Japan

Nihon University Itabashi Hospital

Tokyo, , Japan

Nihon University Nerima Hikarigaoka Hospital

Tokyo, , Japan

Yamanashi Prefectural Central Hospital

Yamanashi, , Japan

Countries

Japan

Other Identifiers

AGAL03204

Identifier Type: -

Identifier Source: org_study_id