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History of Changes for Study: NCT00140621
A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients with Cardiac Fabry Disease.
Latest version (submitted April 16, 2015) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 August 30, 2005 None (earliest Version on record)
2 September 9, 2005 Study Status and Contacts/Locations
3 September 26, 2005 Contacts/Locations, Study Identification, Eligibility, Conditions and Study Status
4 March 28, 2006 Contacts/Locations, Outcome Measures and Study Status
5 October 19, 2006 Study Status
6 November 14, 2006 Outcome Measures, Conditions, Eligibility, Study Description, Oversight, Study Status and Study Identification
7 June 6, 2007 Contacts/Locations, Study Status, Outcome Measures, Study Design and Study Description
8 July 16, 2007 Outcome Measures, Arms and Interventions and Study Status
9 August 30, 2007 Study Status
10 April 3, 2008 Study Status and Eligibility
11 March 9, 2009 Study Status, Arms and Interventions and Sponsor/Collaborators
12 July 6, 2009 Contacts/Locations and Study Status
13 September 29, 2009 Recruitment Status, Study Status, Contacts/Locations, Study Design and Study Identification
14 April 25, 2011 Study Status, Contacts/Locations, Eligibility, Outcome Measures and Study Description
15 November 29, 2011 Sponsor/Collaborators and Study Status
16 April 13, 2012 Study Status, Contacts/Locations and Oversight
17 August 9, 2012 Recruitment Status, Study Status and Contacts/Locations
18 April 1, 2015 Outcome Measures, Study Status, Arms and Interventions, Study Identification, Contacts/Locations, Results, Eligibility and Study Description
19 April 16, 2015 Outcome Measures and Study Status
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Study NCT00140621
Submitted Date:  August 30, 2005 (v1)

Open or close this module Study Identification
Unique Protocol ID: AGAL03204
Brief Title: A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients with Cardiac Fabry Disease.
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
Secondary IDs:
Open or close this module Study Status
Record Verification: August 2005
Overall Status: Recruiting
Study Start: May 2005
Primary Completion:
Study Completion: March 2010
First Submitted: August 30, 2005
First Submitted that
Met QC Criteria:
August 30, 2005
First Posted: September 1, 2005 [Estimate]
Last Update Submitted that
Met QC Criteria:
August 30, 2005
Last Update Posted: September 1, 2005 [Estimate]
Open or close this module Sponsor/Collaborators
Sponsor: Genzyme, a Sanofi Company
Responsible Party:
Collaborators:
Open or close this module Oversight
U.S. FDA-regulated Drug:
U.S. FDA-regulated Device:
Data Monitoring:
Open or close this module Study Description
Brief Summary:

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.

Primary evaluation variables that will be explored:

  1. Changes in interventricular septum and left ventricular posterior wall thickness from baseline until Week 156 or discontinuation of treatment as assessed by echocardiogram.
  2. Changes in left ventricular mass (LVM) from baseline until Week 156 or discontinuation of treatment as assessed by echocardiogram.

Secondary evaluation variables that will be explored:

  1. Results of overall evaluation of cardiac function assessed by cardiac function tests (echocardiogram, cardiac catheterization, electrocardiogram, BNP), clinical symptoms (subjective symptoms) and the NYHA cardiac functional classification
  2. The mean change and changes in plasma GL-3 levels
  3. Changes in GL-3 accumulation scores in myocardial tissue from baseline until Week 156 or discontinuation of treatment as assessed by light microscopy
  4. Changes in SF-36 Health Survey scores
  5. Safety evaluation Adverse events, vital signs, head MRI, clinical laboratory data, immunogenicity
Detailed Description:
Open or close this module Conditions
Conditions: cardiac fabry disease
Keywords:
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Treatment
Study Phase: Phase 4
Interventional Study Model: Single Group Assignment
Number of Arms:
Masking: None (Open Label)
Allocation: Non-Randomized
Enrollment: 10
Open or close this module Arms and Interventions
Intervention Details:
Drug: Agalsidase beta (recombinant form)
Open or close this module Outcome Measures
Primary Outcome Measures:
1. - To evaluate the efficacy of Fabrazyme in reducing interventricular septum and left ventricular posterior wall thickness assessed by echocardiogram
2. - To evaluate the efficacy of Fabrazyme in reducing left ventricular mass (LVM) assessed by echocardiogram.
Secondary Outcome Measures:
1. - 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.
2. - To evaluate the efficacy of this drug in lowering plasma globotriaosylceramide (hereinafter referred to as GL-3) level.
3. - To evaluate in evaluable subjects the efficacy of this drug in reducing GL-3 accumulation in myocardial tissue.
4. - To evaluate the efficacy of this drug according to SF-36 Health Survey scores.
5. - To evaluate the safety of this drug.
Open or close this module Eligibility
Minimum Age: 20 Years
Maximum Age: 64 Years
Sex: All
Gender Based:
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) diagnosed 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 13mm 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.
  • Age: between 20 years and 64 years
  • Sex: males and females
  • 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.
  • Patients who are unwilling to comply with the requirements of the protocol.
  • Others judged by the investigator or subinvestigator to be ineligible for the study
Open or close this module Contacts/Locations
Central Contact Person: Clinical Affairs
Telephone: 81-3-3230-8285
Study Officials: Kazuyuki Hatakeyama, M.D., Ph.D.
Study Director
Genzyme, a Sanofi Company
Locations: Japan
Umajima City Hospital
[Recruiting]
Ehime, Japan, 798-8510
Contact:Principal Investigator: Mareomi Hamada, M.D., Ph.D.
Kagoshima University Hospital
[Recruiting]
Kagoshima, Japan, 890-8520
Contact:Principal Investigator: Toshihiro Takenaka, M.D., Ph.D.
Contact:Sub-Investigator: Chuwa Tei, Ph.D.
Open or close this module IPDSharing
Plan to Share IPD:
Open or close this module References
Citations:
Links:
Available IPD/Information:

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