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, a Sanofi Company
ClinicalTrials.gov Identifier:
NCT00140621
First received: August 30, 2005
Last updated: August 9, 2012
Last verified: August 2012
  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:


Further study details as provided by Genzyme, a Sanofi Company:

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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, a Sanofi Company
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

No publications provided

Responsible Party: Genzyme, a Sanofi Company
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, a Sanofi Company:
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
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular 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 August 18, 2014