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

Sponsored by:	Genzyme
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.

New adverse drug reactions (ADRs) that cannot be predicted from the Precautions (in particular, clinically significant ADRs)
The incidence of ADRs under the actual conditions of use of the drug
Causal factors that might potentially affect safety
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."

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

Genetics Home Reference related topics: Chanarin-Dorfman syndrome cholesteryl ester storage disease Fabry disease Farber lipogranulomatosis L1 syndrome long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency mitochondrial trifunctional protein deficiency primary carnitine deficiency succinic semialdehyde dehydrogenase deficiency

U.S. FDA Resources

Study Type:	Observational
Study Design:	Cohort, Prospective
Official Title:	Special Survey in Long-Term Use of Fabrazyme

Further study details as provided by Genzyme:

Estimated Enrollment:	350
Study Start Date:	June 2004
Estimated Study Completion Date:	March 2011

Intervention Details:

agalsidase beta 1.0 mg/kg body weight infused every 2 weeks as an intravenous infusion

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, 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

Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No
Sampling Method:	Non-Probability Sample

Study Population

Japanese patients with Fabry Disease

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00233870

Contacts

Contact: Postmarketing Surveillance

81-3-3230-8286

Locations

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

Sponsors and Collaborators

Genzyme

Investigators

Study Director:

Bernard Bénichou, M.D., Ph.D.

Genzyme

More Information

Additional Information:

US FDA Approved Full Prescribing Information for Fabrazyme® This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party:	Genzyme Coporation ( Medical Monitor )
Study ID Numbers:	AGAL03004
Study First Received:	October 5, 2005
Last Updated:	March 18, 2009
ClinicalTrials.gov Identifier:	NCT00233870 History of Changes
Health Authority:	Japan: Ministry of Health, Labor and Welfare

Study placed in the following topic categories:

Lipid Metabolism, Inborn Errors
Sphingolipidoses
Metabolic Diseases
Lysosomal Storage Diseases
Sphingolipidosis
Central Nervous System Diseases
Brain Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn

Fabry Disease
Genetic Diseases, X-Linked
Ceramide Trihexosidosis
Brain Diseases, Metabolic, Inborn
Lipidoses
Metabolic Disorder
Lipid Metabolism Disorders
Brain Diseases, Metabolic

Additional relevant MeSH terms:

Lipid Metabolism, Inborn Errors
Sphingolipidoses
Metabolic Diseases
Lysosomal Storage Diseases, Nervous System
Lysosomal Storage Diseases
Nervous System Diseases
Central Nervous System Diseases
Brain Diseases

Metabolism, Inborn Errors
Genetic Diseases, Inborn
Fabry Disease
Genetic Diseases, X-Linked
Brain Diseases, Metabolic, Inborn
Lipidoses
Lipid Metabolism Disorders
Brain Diseases, Metabolic

ClinicalTrials.gov processed this record on May 07, 2009