A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease
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.
|Study Design:||Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
|Official Title:||A Multicenter Open-label Study of the Safety and Efficacy of α-galactosidase A (R-h α-GAL) Replacement Therapy in Patients With Cardiac Fabry Disease|
- Percent Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156 [ Time Frame: Baseline to Week 156 ]Interventricular septum and left ventricular posterior wall thickness was assessed by echocardiogram.
- Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156 [ Time Frame: Baseline to Week 156 ]Interventricular septum and left ventricular posterior wall thickness was assessed by echocardiogram.
- Percent Change From Baseline in Left Ventricular Mass (LVM) at Week 156 [ Time Frame: Baseline to Week 156 ]Left ventricular mass was assessed by echocardiogram.
- Change From Baseline in LVM at Week 156 [ Time Frame: Baseline to Week 156 ]Left ventricular mass was assessed by echocardiogram.
- Number of Participants in Overall Cardiac Function Assessment and Clinical Symptoms at Week 156: Change From Baseline in Cardiac Function Test [ Time Frame: Baseline to Week 156 ]Overall cardiac function assessment was assessed by tests (echocardiogram,cardiac catheterization (optional),electrocardiogram,B-type natriuretic peptide [BNP]), clinical symptoms (subjective symptoms) and the New York Heart Association (NYHA) cardiac functional classification.Overall assessment of cardiac function was assessed based on the evaluation items including interventricular septum thickness, left ventricular posterior wall thickness, left ventricular mass, clinical function tests and clinical symptoms. A subject was considered to be Improved: if Improved in 2 items or more, Unchanged: Improved in one item and unchanged in 2 items or unchanged in all 3 items, Aggravated: Aggravated in one item or more.
- Percent Change From Baseline in GL-3 Plasma Levels at Week 156 [ Time Frame: Baseline to Week 156 ]
- Change From Baseline in Short Form (36) Health Survey (SF-36) Scores at Week 156 [ Time Frame: Baseline to Week 156 ]The 36-Item Short-Form Health Survey (SF-36) is a standardized survey evaluating 8 aspects of functional health and well-being: physical and social functioning, physical and emotional role limitations, bodily pain, general health, vitality, mental health. These 8 aspects can also be summarized as physical component score (PCS) and mental component score (MCS). The score for a section is an average of the individual question scores, which are scaled 0-100 (100=highest level of functioning).
|Study Start Date:||July 2005|
|Study Completion Date:||August 2012|
|Primary Completion Date:||August 2012 (Final data collection date for primary outcome measure)|
Experimental: Agalsidase Beta
Agalsidase beta 1 milligram per kilogram (mg/kg) intravenously once every 2 weeks up to 156 weeks.
Drug: Agalsidase beta
Other Name: Fabrazyme®
Please refer to this study by its ClinicalTrials.gov identifier: NCT00140621
|Fujita Health University Hospital|
|Aichi, Japan, 470-1192|
|Sapporo Medical University Hospital|
|Hokkaido, Japan, 060-8543|
|Akune Citizen Hospital|
|Kagoshima, Japan, 899-1611|
|Tohoku University Hospital|
|Miyagi, Japan, 980-8574|
|Nihon University Itabashi Hospital|
|Tokyo, Japan, 173-8610|
|Nihon University Nerima Hikarigaoka Hospital|
|Tokyo, Japan, 179-0072|
|Yamanashi Prefectural Central Hospital|
|Yamanashi, Japan, 400-8506|
|Study Director:||Medical Monitor||Genzyme, a Sanofi Company|