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

Information source: Sanofi
ClinicalTrials.gov processed this data on August 20, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Fabry Disease

Intervention: Agalsidase beta (recombinant form) (Drug)

Phase: Phase 4

Status: Completed

Sponsored by: Genzyme, a Sanofi Company

Official(s) and/or principal investigator(s):
Medical Monitor, Study Director, Affiliation: Genzyme, a Sanofi Company

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

Primary outcome: Change in blood GL-3 level

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

[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.

Locations and Contacts

Tohoku University Hospital, Sendai 980-8574, Japan
Additional Information

Starting date: June 2004
Last updated: May 7, 2015

Page last updated: August 20, 2015

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