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

Fabry Disease Clinical Trial

Official title:

Special Survey in Long-Term Use of Fabrazyme

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00233870
• Other study ID #: AGAL03004
• Status: Completed
• Phase: Phase 4
• First received: October 5, 2005
• Last updated: May 7, 2015
• Start date: June 2004
• Est. completion date: March 2011

Study information

• Verified date: May 2015
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Health authority: Japan: Ministry of Health, Labor and Welfare
• Study type: Observational

Clinical Trial 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."

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 405
• Est. completion date: March 2011
• Est. primary completion date: March 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

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.

Study Design

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

• Fabry Disease

Intervention

Drug:
• Agalsidase beta (recombinant form)
agalsidase beta 1.0 mg/kg body weight infused every 2 weeks as an intravenous infusion

Locations

Country	Name	City	State
Japan	Tohoku University Hospital	Sendai

Sponsors (1)

Lead Sponsor	Collaborator
Genzyme, a Sanofi Company

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in blood GL-3 level		6 months	No