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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Genzyme
ClinicalTrials.gov Identifier:
NCT00233870
First received: October 5, 2005
Last updated: December 21, 2011
Last verified: December 2011
History of Changes
  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.

  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."


Condition Intervention Phase
Fabry Disease
 Drug: Agalsidase beta (recombinant form)
 Phase 4

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

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Genzyme:

Enrollment: 405
Study Start Date: June 2004
Study Completion Date: March 2011
Primary Completion Date: March 2011 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: Agalsidase beta (recombinant form)
    agalsidase beta 1.0 mg/kg body weight infused every 2 weeks as an intravenous infusion
    Other Name: Fabrazyme
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

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 [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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00233870

Locations
Japan
Tohoku University Hospital
Sendai, Japan, 980-8574
Sponsors and Collaborators
Genzyme
Investigators
Study Director: Medical Monitor Genzyme
  More Information

No publications provided

Responsible Party: Genzyme
ClinicalTrials.gov Identifier: NCT00233870     History of Changes
Other Study ID Numbers: AGAL03004
Study First Received: October 5, 2005
Last Updated: December 21, 2011
Health Authority: Japan: Ministry of Health, Labor and Welfare

Additional relevant MeSH terms:
Fabry Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
 Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on June 18, 2013