A Long Term Safety and Efficacy Study of Fabrazyme Replacement Therapy in Japanese Patients With Fabry Disease.
The purpose of this survey is to identify any concerns regarding the following efficacy and safety-related issues in clinical practice with the new drugs "Fabrazyme for intravenous infusion 5mg" and "Fabrazyme for intravenous infusion 35mg" and to confirm the safety of these products in long-term use in the clinical setting.
- New adverse drug reactions (ADRs) that cannot be predicted from the Precautions (in particular, clinically significant ADRs)
- The incidence of ADRs under the actual conditions of use of the drug
- Causal factors that might potentially affect safety
- Efficacy evaluation in long-term use
This survey will be conducted in accordance with the approval condition established for Fabrazyme:
"To conduct a special surveillance of Efficacy and Safety in long term treatment and Pediatric with the drug."
|Study Design:||Observational Model: Cohort
Time Perspective: Prospective
|Official Title:||Special Survey in Long-Term Use of Fabrazyme|
- Change in blood GL-3 level [ Time Frame: 6 months ] [ Designated as safety issue: No ]
|Study Start Date:||June 2004|
|Study Completion Date:||March 2011|
|Primary Completion Date:||March 2011 (Final data collection date for primary outcome measure)|
Drug: Agalsidase beta (recombinant form)
Medical institutions or physicians will be asked to periodically complete the survey forms for all patients registered. Survey forms include baseline information available, and then data collected every 6 months, as available including: demographic information, concomitant medications/therapy, treatment record, ECG, Echocardiogram, computed tomography scan / magnetic resonance imaging (CT/MRI), Fabry symptoms, labs, functional disorder, blood concentration of GL-3, and anti-agalsidase beta antibody test (IgE testing) to survey whether the productions of antibodies to agalsidase beta is a causal factor of treatment-related reactions.
The survey period shall be approximately 7 years from June 1, 2004 during which survey shall be undertaken as follows:
- The observation period for each patient shall range from 1 to about 7 years after starting treatment
- Registration period: June 1, 2004 to March 31, 2010
- Survey period: June 1, 2004 to March 31, 2011
In institutions for which retrospective surveys are feasible, the survey period will trace back to the date of approval (January 29, 2004), as far as possible.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00233870
|Tohoku University Hospital|
|Sendai, Japan, 980-8574|
|Study Director:||Medical Monitor||Genzyme, a Sanofi Company|