AGAL03004: A Long Term Safety and Efficacy Study of Fabrazyme Replacement Therapy in Japanese Patients with Fabry Disease

This study is currently recruiting patients.

Sponsored By:	Genzyme Genzyme Corporation (Genzyme Japan K.K.)
Information Provided By:	Genzyme
ClinicalTrials.gov Identifier:	NCT00233870

Purpose

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:

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."

Condition	Intervention	Phase
Fabry Disease	Drug: Agalsidase beta (recombinant form)	Phase IV

Study Type: Observational

Study Design: Natural History, Longitudinal, Defined Population, Retrospective/Prospective Study

Official Title: Special Survey in Long-Term Use of Fabrazyme

Further Study Details:

Study Start: 2004-06; Expected Completion: 2011-03

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, 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

Ages Eligible for Study: 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.

This will exclude, however, patients registered in the post-marketing trials during the post-marketing clinical trial period.

Exclusion Criteria:

See above

Location and Contact Information

Please refer to this study by ClinicalTrials.gov identifier NCT00233870

Japan
Tohoku University Hospital, Sendai, 980-8574, Japan; Recruiting

More Information

Fabrazyme® FDA-approved labeling information

Study ID Numbers: AGAL03004
ClinicalTrials.gov Identifier: NCT00233870
Health Authority: Japan: Ministry of Health, Labor and Welfare