A Long Term Safety and Efficacy Study of Fabrazyme Replacement Therapy in Japanese Patients With Fabry Disease.

• Status: Completed
• Trial end date: 2011-03-01
• Target enrollment: 0
• Participant gender: All

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."

• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Genzyme, a Sanofi Company

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 [agalsidase beta (recombinant form)] 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 [agalsidase beta (recombinant form)] 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.