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Trial record 1 of 1 for:    agal03204
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A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00140621
Recruitment Status : Completed
First Posted : September 1, 2005
Results First Posted : April 15, 2015
Last Update Posted : May 12, 2015
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Tracking Information
First Submitted Date  ICMJE August 30, 2005
First Posted Date  ICMJE September 1, 2005
Results First Submitted Date  ICMJE April 1, 2015
Results First Posted Date  ICMJE April 15, 2015
Last Update Posted Date May 12, 2015
Study Start Date  ICMJE July 2005
Actual Primary Completion Date August 2012   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 1, 2015)
  • Percent Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156 [ Time Frame: Baseline to Week 156 ]
    Interventricular septum and left ventricular posterior wall thickness was assessed by echocardiogram.
  • Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156 [ Time Frame: Baseline to Week 156 ]
    Interventricular septum and left ventricular posterior wall thickness was assessed by echocardiogram.
  • Percent Change From Baseline in Left Ventricular Mass (LVM) at Week 156 [ Time Frame: Baseline to Week 156 ]
    Left ventricular mass was assessed by echocardiogram.
  • Change From Baseline in LVM at Week 156 [ Time Frame: Baseline to Week 156 ]
    Left ventricular mass was assessed by echocardiogram.
Original Primary Outcome Measures  ICMJE
 (submitted: August 30, 2005)
  • - 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.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 1, 2015)
  • Number of Participants in Overall Cardiac Function Assessment and Clinical Symptoms at Week 156: Change From Baseline in Cardiac Function Test [ Time Frame: Baseline to Week 156 ]
    Overall cardiac function assessment was assessed by tests (echocardiogram,cardiac catheterization (optional),electrocardiogram,B-type natriuretic peptide [BNP]), clinical symptoms (subjective symptoms) and the New York Heart Association (NYHA) cardiac functional classification.Overall assessment of cardiac function was assessed based on the evaluation items including interventricular septum thickness, left ventricular posterior wall thickness, left ventricular mass, clinical function tests and clinical symptoms. A subject was considered to be Improved: if Improved in 2 items or more, Unchanged: Improved in one item and unchanged in 2 items or unchanged in all 3 items, Aggravated: Aggravated in one item or more.
  • Percent Change From Baseline in GL-3 Plasma Levels at Week 156 [ Time Frame: Baseline to Week 156 ]
  • Change From Baseline in Short Form (36) Health Survey (SF-36) Scores at Week 156 [ Time Frame: Baseline to Week 156 ]
    The 36-Item Short-Form Health Survey (SF-36) is a standardized survey evaluating 8 aspects of functional health and well-being: physical and social functioning, physical and emotional role limitations, bodily pain, general health, vitality, mental health. These 8 aspects can also be summarized as physical component score (PCS) and mental component score (MCS). The score for a section is an average of the individual question scores, which are scaled 0-100 (100=highest level of functioning).
Original Secondary Outcome Measures  ICMJE
 (submitted: August 30, 2005)
  • - 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.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease
Official Title  ICMJE A Multicenter Open-label Study of the Safety and Efficacy of α-galactosidase A (R-h α-GAL) Replacement Therapy in Patients With Cardiac Fabry Disease
Brief Summary

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

Participants participated for 4 weeks or less in the baseline period and 156 weeks for the treatment period.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Fabry Disease
Intervention  ICMJE Drug: Agalsidase beta
Other Name: Fabrazyme®
Study Arms  ICMJE Experimental: Agalsidase Beta
Agalsidase beta 1 milligram per kilogram (mg/kg) intravenously once every 2 weeks up to 156 weeks.
Intervention: Drug: Agalsidase beta
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: June 6, 2007)
6
Original Enrollment  ICMJE
 (submitted: August 30, 2005)
10
Actual Study Completion Date  ICMJE August 2012
Actual Primary Completion Date August 2012   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

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

    • In the case of male participants, documented plasma or leukocyte alpha-galactosidase A (α-GAL) activity was no more than 20 percent (%) of normal value (except for heterozygous female participants)
    • Left ventricular hypertrophy was noted.
    • Accumulation of globotriaosylceramide (GL-3) in the myocardium or a genetic deficiency associated with α-GAL was confirmed
    • Or in the case of heterozygous female participants, when the family (father or son) was diagnosed with Fabry disease. (Father or son was 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 were observed, except for proteinuria sign.
  • Participants with interventricular and posterior wall thickness of at least 13 millimeter (mm) on echocardiography within 3 months before signed date to informed consent
  • Participants in whom cardiac function was rated as Class I or II according to the New York Heart Association (NYHA) classification when giving informed consent.
  • Participants classification: inpatients and outpatients
  • Participants who had given written informed consent before the study-related baseline tests.

Exclusion Criteria:

  • Participants with severe hypertension (for example, blood pressure more than or equal to 180 millimeter of mercury [mmHg] and/or blood pressure more than or equal to 110 mmHg in spite of adequate medication)
  • Participants whose serum creatinine level was higher than the upper normal limit within 3 months (12 weeks) prior to giving informed consent.
  • Participants who had undergone kidney transplantation or were currently on dialysis.
  • Participants with any serious hepatic disorder. Participants who had abnormal hepatic function test values within 3 months (12 weeks) prior to giving informed consent (when either alanine aminotransferase [ALT] or aspartate aminotransferase [AST] level exceeded the value five times as high as the upper normal limit).
  • Permanent pacemaker or defibrillator implanted participants
  • Pregnant or lactating women
  • Participants who had taken this drug for 6 months (26 weeks) or more before giving informed consent.
  • Participants who had participated in a clinical study employing any other investigational product within 3 months prior to giving informed consent.
  • Enzyme replacement therapy history, except for agalsidase beta
  • Participants who were unwilling to comply with the requirements of the protocol.
  • Others judged by the investigator or sub-investigator to be ineligible for the study
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 20 Years to 64 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Japan
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00140621
Other Study ID Numbers  ICMJE AGAL03204
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Sanofi ( Genzyme, a Sanofi Company )
Original Responsible Party Not Provided
Current Study Sponsor  ICMJE Genzyme, a Sanofi Company
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Medical Monitor Genzyme, a Sanofi Company
PRS Account Sanofi
Verification Date April 2015

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