AGAL03204: A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients with Cardiac Fabry Disease

This study is currently recruiting patients.

Sponsored By:	Genzyme Genzyme Corporation (Genzyme Japan K.K.)
Information Provided By:	Genzyme
ClinicalTrials.gov Identifier:	NCT00140621

Purpose

Multi-center, open label, phase IV study conducted to evaluate the efficacy and safety of Fabrazyme [agalsidase beta (recombinant form)] administered by intravenous drip infusion in patients with cardiac Fabry disease.

Patients will participate for 4 weeks or less in the baseline period and 156 weeks for the treatment period.

Primary evaluation variables that will be explored:

Secondary evaluation variables that will be explored:

Condition	Intervention	Phase
Fabry disease	Drug: Agalsidase beta (recombinant form)	Phase IV

Study Type: Interventional

Study Design: Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study

Official Title: A Multicenter Open-Label Study of the Safety and Efficacy of a-Galactosidase A (r-h a-GAL) Replacement Therapy in Patients with Cardiac Fabry Disease

Further Study Details:
Primary Outcomes: To evaluate the efficacy of Fabrazyme in reducing interventricular septum and left ventricular posterior wall thickness assessed by echocardiogram; To evaluate the efficacy of Fabrazyme in reducing left ventricular mass (LVM) assessed by echocardiogram

Secondary Outcomes: To make an overall evaluation of changes in cardiac function assessment by cardiac function tests (echocardiogram, cardiac catheterization, electrocardiogram, BNP), clinical symptoms (subjective symptoms) and the NYHA cardiac functional classification; To evaluate the efficacy of this drug in lowering plasma globotriaosylceramide (hereinafter referred to as GL-3) level; To evaluate in evaluable subjects the efficacy of this drug in reducing GL-3 accumulation in myocardial tissue; To evaluate the efficacy of this drug according to SF-36 Health Survey scores; To evaluate the safety of this drug

Expected Total Enrollment: 6
Study Start: 2005-05; Expected Completion: 2011-03

Eligibility Criteria

Ages Eligible for Study: 20 Years - 64 Years, Genders Eligible for Study: Both

Inclusion Criteria:

Patients definitively diagnosed with cardiac Fabry disease (who fulfill all of the following criteria):

Patient with interventricular and posterior wall thickness of at least 13mm on echocardiography within 3 months before signed date to informed consent

Patients in whom cardiac function is rated as Class I or II according to the NYHA classification when giving informed consent.

Age: between 20 years and 64 years

Sex: males and females

Patient classification: inpatients and outpatients

Patients who have given written informed consent before the study-related baseline tests.

Exclusion Criteria:

Patient with severe hypertension (e. g, systolic blood pressure 180 mmHg and/or diastolic blood pressure 110 mmHg in spite of adequate medication)

Patients whose serum creatinine level is higher than the upper normal limit within 3 months (12 weeks) prior to giving informed consent.

Patients who have undergone kidney transplantation or are currently on dialysis.

Patients with any serious hepatic disorder. Patients who have abnormal hepatic function test values within 3 months (12 weeks) prior to giving informed consent (when either ALT or AST level exceeds the value five times as high as the upper normal limit).

Permanent pacemaker or defibrillator implanted patients

Pregnant or lactating women

Patients who have taken this drug for 6 months (26 weeks) or more before giving informed consent.

Patients who have participated in a clinical study employing any other investigational product within 3 months prior to giving informed consent.

Enzyme replacement therapy history, except for Fabrazyme.

Patients who are unwilling to comply with the requirements of the protocol.

Others judged by the investigator or sub-investigator to be ineligible for the study

Location and Contact Information

Please refer to this study by ClinicalTrials.gov identifier NCT00140621

Japan
Kagoshima University Hospital, Kagoshima, 890-8520, Japan; Recruiting
Uwajima City Hospital, Ehime, 798-8510, Japan; Recruiting
Fujita Health University Hospital, Aichi, 470-1192, Japan; Recruiting
Yamanashi Prefectural central Hospital, Yamanashi, 400-8506, Japan; Recruiting
Tohoku University Hospital, Miyagi, 980-8574, Japan;Recruiting

More Information

Fabrazyme® FDA-approved labeling information

Study ID Numbers: AGAL03204
ClinicalTrials.gov Identifier: NCT00140621
Health Authority: Japan: Ministry of Health, Labor and Welfare