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

April 16, 2015 updated by: Genzyme, a Sanofi Company

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

6

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Japan
      • Aichi, Japan, 470-1192
        • Fujita Health University Hospital
      • Hokkaido, Japan, 060-8543
        • Sapporo Medical University Hospital
      • Kagoshima, Japan, 899-1611
        • Akune Citizen Hospital
      • Miyagi, Japan, 980-8574
        • Tohoku University Hospital
      • Tokyo, Japan, 173-8610
        • Nihon University Itabashi Hospital
      • Tokyo, Japan, 179-0072
        • Nihon University Nerima Hikarigaoka Hospital
      • Yamanashi, Japan, 400-8506
        • Yamanashi Prefectural Central Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

20 years to 64 years (ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NON_RANDOMIZED
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: Agalsidase Beta
Agalsidase beta 1 milligram per kilogram (mg/kg) intravenously once every 2 weeks up to 156 weeks.
Drug: Agalsidase beta
Other Names:
  • Fabrazyme®

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Baseline to Week 156
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.
Baseline to Week 156
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.
Baseline to Week 156
Change From Baseline in LVM at Week 156
Time Frame: Baseline to Week 156
Left ventricular mass was assessed by echocardiogram.
Baseline to Week 156

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Baseline to Week 156
Percent Change From Baseline in GL-3 Plasma Levels at Week 156
Time Frame: Baseline to Week 156
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).
Baseline to Week 156

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Genzyme, a Sanofi Company

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

July 1, 2005

Primary Completion (ACTUAL)

August 1, 2012

Study Completion (ACTUAL)

August 1, 2012

Study Registration Dates

First Submitted

August 30, 2005

First Submitted That Met QC Criteria

August 30, 2005

First Posted (ESTIMATE)

September 1, 2005

Study Record Updates

Last Update Posted (ESTIMATE)

May 12, 2015

Last Update Submitted That Met QC Criteria

April 16, 2015

Last Verified

April 1, 2015

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AGAL03204

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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