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

August 30, 2005
August 9, 2012
July 2005
August 2012   (final data collection date for primary outcome measure)
  • 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 ]
  • - To evaluate the efficacy of Fabrazyme in reducing interventricular septum and left ventricular posterior wall thickness assessed by echocardiogram
  • - To evaluate the efficacy of Fabrazyme in reducing left ventricular mass (LVM) assessed by echocardiogram.
Complete list of historical versions of study NCT00140621 on ClinicalTrials.gov Archive Site
  • 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 ]
  • - To make an overall evaluation of changes in cardiac function assessment by cardiac function tests (echocardiogram, cardiac catheterization, electrocardiogram, BNP), clinical symptoms (subjective symptoms) and the NYHA cardiac functional classification.
  • - To evaluate the efficacy of this drug in lowering plasma globotriaosylceramide (hereinafter referred to as GL-3) level.
  • - To evaluate in evaluable subjects the efficacy of this drug in reducing GL-3 accumulation in myocardial tissue.
  • - To evaluate the efficacy of this drug according to SF-36 Health Survey scores.
  • - To evaluate the safety of this drug.
Not Provided
Not Provided
 
A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease
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

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.

Not Provided
Interventional
Phase 4
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Fabry Disease
Drug: Agalsidase beta (Fabrazyme)
Fabrazyme 1.0 mg/kg body weight infused every 2 weeks as an intravenous infusion.
Not Provided
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
6
August 2012
August 2012   (final data collection date for primary outcome measure)

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
Both
20 Years to 64 Years
No
Contact information is only displayed when the study is recruiting subjects
Japan
 
NCT00140621
AGAL03204
No
Genzyme, a Sanofi Company
Genzyme, a Sanofi Company
Not Provided
Study Director: Medical Monitor Genzyme, a Sanofi Company
Genzyme, a Sanofi Company
August 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP