A Long Term Safety and Efficacy Study of Fabrazyme Replacement Therapy in Japanese Patients With Fabry Disease.
Information source: Genzyme
Information obtained from ClinicalTrials.gov on October 04, 2010
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Fabry Disease
Intervention: Agalsidase beta (recombinant form) (Drug)
Phase: Phase 4
Status: Recruiting
Sponsored by: Genzyme Official(s) and/or principal investigator(s):
Medical Monitor, Study Director, Affiliation: Genzyme
Overall contact:
Medical Information, Phone: 800-745-4447, Email: medinfo@genzyme.com
Summary
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.
1. New adverse drug reactions (ADRs) that cannot be predicted from the Precautions (in
particular, clinically significant ADRs)
2. The incidence of ADRs under the actual conditions of use of the drug
3. Causal factors that might potentially affect safety
4. 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."
Clinical Details
Official title: Special Survey in Long-Term Use of Fabrazyme
Study design: Observational Model: Cohort, Time Perspective: Prospective
Detailed description:
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.
Eligibility
Minimum age: N/A.
Maximum age: N/A.
Gender(s): Both.
Criteria:
Inclusion Criteria:
- Patients in Japan with the indication of "Fabry Disease" and for whom the usual
dosage and administration is 1mg of agalsidase beta (recombinant) per 1 kg body
weight each time, administered by intravenous infusion every 2 weeks
- Because the efficacy evaluation of enzyme replacement therapy with Fabrazyme will
require the comparison of findings before and after the start of enzyme replacement
therapy, the efficacy evaluation set will be defined as including patients using
Fabrazyme for the first time in the post-marketing setting and those for whom it is
possible to obtain retrospective data for before the start of enzyme replacement
therapy.
Exclusion Criteria:
- patients registered in the post-marketing trials during the post-marketing clinical
trial period.
Locations and Contacts
Medical Information, Phone: 800-745-4447, Email: medinfo@genzyme.com
Tohoku University Hospital, Sendai 980-8574, Japan; Recruiting
Additional Information
Starting date: June 2004
Last updated: June 25, 2010
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