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A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Genzyme
ClinicalTrials.gov Identifier:
NCT00140621
First received: August 30, 2005
Last updated: August 9, 2012
Last verified: August 2012
History of Changes
  Purpose

This is a multi-center, open label, phase IV study conducted to evaluate the efficacy and safety of Fabrazyme [agalsidase beta (recombinant form)] administered by intravenous drip infusion in patients with cardiac Fabry disease.

Patients will participate for 4 weeks or less in the baseline period and 156 weeks for the treatment period.


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

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Multicenter Open-label Study of the Safety and Efficacy of A-galactosidase A (R-h a-GAL) Replacement Therapy in Patients With Cardiac Fabry Disease

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Genzyme:

Primary Outcome Measures:
  • To evaluate the efficacy of Fabrazyme [agalsidase beta (recombinant form)] in reducing interventricular septum and left ventricular posterior wall thickness assessed by echocardiogram [ Time Frame: Up to 156 weeks ] [ Designated as safety issue: No ]
  • To evaluate the efficacy of Fabrazyme [agalsidase beta (recombinant form)] in reducing left ventricular mass (LVM) assessed by echocardiogram [ Time Frame: Up to 156 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Results of overall evaluation of changes in cardiac function assessed by tests (echocardiogram,cardiac catheterization(optional),electrocardiogram,BNP), clinical symptoms(subjective symptoms) and the NYHA cardiac functional classification [ Time Frame: Up to 156 weeks ] [ Designated as safety issue: No ]
  • To evaluate in evaluable subjects the efficacy of this drug in reducing GL-3 accumulation in myocardial tissue [ Time Frame: Up to 156 weeks ] [ Designated as safety issue: No ]
  • To evaluate the efficacy of this drug according to SF-36 Health Survey scores [ Time Frame: Up to 156 weeks ] [ Designated as safety issue: No ]
  • To evaluate the safety of this drug [ Time Frame: Up to 156 weeks ] [ Designated as safety issue: Yes ]

Enrollment: 6
Study Start Date: July 2005
Study Completion Date: August 2012
Primary Completion Date: August 2012 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: Agalsidase beta (Fabrazyme)
    Fabrazyme 1.0 mg/kg body weight infused every 2 weeks as an intravenous infusion.
  Eligibility

Ages Eligible for Study:   20 Years to 64 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients definitively diagnosed with cardiac Fabry disease (who fulfill all of the following criteria):

    • In the case of male patients, documented plasma or leukocyte a- GAL activity is no more than 20% of normal value (except for heterozygous female patients.)
    • Left ventricular hypertrophy is noted.
    • Accumulation of GL-3 in the myocardium or a genetic deficiency associated with a-GAL has been confirmed
    • Or in the case of heterozygous female patients, when the family (father or son) is diagnosed with Fabry disease. (Father or son is related by birth.)
    • Without symptoms or signs of Fabry, such as
    • acroparesthesia
    • angiokeratomas
    • abnormal sweating
    • pain of distal extremities
    • chronic abdominal pain/diarrhea and corneal opacities are observed, except for proteinuria sign.
  • Patient with interventricular and posterior wall thickness of at least 13 mm on echocardiography within 3 months before signed date to informed consent
  • Patients in whom cardiac function is rated as Class I or II according to the NYHA classification when giving informed consent.
  • Patient classification: inpatients and outpatients
  • Patients who have given written informed consent before the study-related baseline tests.

Exclusion Criteria:

  • Patient with severe hypertension (e.g., systolic blood pressure 180 mmHg and/or diastolic blood pressure 110 mmHg in spite of adequate medication)
  • Patients whose serum creatinine level is higher than the upper normal limit within 3 months (12 weeks) prior to giving informed consent.
  • Patients who have undergone kidney transplantation or are currently on dialysis.
  • Patients with any serious hepatic disorder. Patients who have abnormal hepatic function test values within 3 months (12 weeks) prior to giving informed consent (when either ALT or AST level exceeds the value five times as high as the upper normal limit).
  • Permanent pacemaker or defibrillator implanted patients
  • Pregnant or lactating women
  • Patients who have taken this drug for 6 months (26 weeks) or more before giving informed consent.
  • Patients who have participated in a clinical study employing any other investigational product within 3 months prior to giving informed consent.
  • Enzyme replacement therapy history, except for Fabrazyme [agalsidase beta (recombinant form)].
  • Patients who are unwilling to comply with the requirements of the protocol.
  • Others judged by the investigator or sub-investigator to be ineligible for the study
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00140621

Locations
Japan
Fujita Health University Hospital
Aichi, Japan, 470-1192
Uwajima City Hospital
Ehime, Japan, 798-8510
Sapporo Medical University Hospital
Hokkaido, Japan, 060-8543
Kagoshima University Hospital
Kagoshima, Japan, 890-8520
Akune Citizen Hospital
Kagoshima, Japan, 899-1611
Tohoku University Hospital
Miyagi, Japan, 980-8574
Nihon University Nerima Hikarigaoka Hospital
Tokyo, Japan, 179-0072
Nihon University Itabashi Hospital
Tokyo, Japan, 173-8610
Yamanashi Prefectural central Hospital
Yamanashi, Japan, 400-8506
Sponsors and Collaborators
Genzyme
Investigators
Study Director: Medical Monitor Genzyme
  More Information

No publications provided

Responsible Party: Genzyme
ClinicalTrials.gov Identifier: NCT00140621     History of Changes
Other Study ID Numbers: AGAL03204
Study First Received: August 30, 2005
Last Updated: August 9, 2012
Health Authority: Japan: Ministry of Health, Labor and Welfare

Keywords provided by Genzyme:
cardiac fabry disease

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