A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease
NCT ID: NCT00074984
Last Updated: 2013-12-27
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE4
82 participants
INTERVENTIONAL
2001-02-28
2004-01-31
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Placebo
Patients randomized to placebo
Placebo
1 mg/kg placebo intravenously every 2 weeks
Fabrazyme (agalsidase beta)
Patients randomized to Fabrazyme (agalsidase beta).
Fabrazyme (agalsidase beta)
1mg/kg Fabrazyme (agalsidase beta) every 2 weeks
Interventions
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Fabrazyme (agalsidase beta)
1mg/kg Fabrazyme (agalsidase beta) every 2 weeks
Placebo
1 mg/kg placebo intravenously every 2 weeks
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Patients must be at least 16 years old
* Patients must have a current diagnosis of Fabry disease and have a clinical presentation consistent of Fabry disease (decreased sweating, Fabry pain, angiokeratoma, etc.)
* Patients may not have received enzyme replacement therapy as a treatment for Fabry disease
* Patients must have a documented plasma a-galactosidase A (aGAL) activity of \< 1.5 nmol/hr/mL or a documented leukocyte aGAL activity of \< 4 nmol/hr/mg
* Patients must have one or more of the following: a serum creatinine measurement of 1.2 to 3 mg/dL (106.1 to 265 umol/L) OR estimated creatinine clearance \< 80 mL/min only if the patient's serum creatinine measurement is \< 1.2 mg/dL
* Female patients of childbearing potential must have a negative pregnancy test prior to each dosing and all female patients must use a medically accepted form of contraception
Exclusion Criteria
* Patient has acute renal failure
* Patient has participated in a study employing an investigational drug within 30 days of study entry
* Patient has diabetes mellitus or presence of confounding renal disease
* Patient has a history of transient ischemic attack (TIA) or ischemic stroke within 3 months of study entry documented by mild-to-moderate neurological deficit
* Patient has critical coronary disease
* Patient has congestive heart failure
* Patient has severe residual neurological deficit that will confound the detection of new events as determined by an attending neurologist and/or Principal Investigator
* Patient is unwilling to comply with the requirements of the protocol or the patient has a medical condition, serious intercurrent illness, or extenuating circumstances that would significantly decrease study compliance, including prescribed follow-up
16 Years
ALL
No
Sponsors
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Genzyme, a Sanofi Company
INDUSTRY
Responsible Party
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Genzyme Coporation
Principal Investigators
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Medical Monitor
Role: STUDY_DIRECTOR
Genzyme Coorporation
Locations
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University of Alabama at Birmingham
Birmingham, Alabama, United States
Cedars-Sinai Medical Center
Los Angeles, California, United States
University of San Francisco
San Francisco, California, United States
University of Connecticut Health Partners
Farmington, Connecticut, United States
Oncology Hematology Association
Coral Springs, Florida, United States
Emory University School of Medicine
Atlanta, Georgia, United States
Children's Memorial Hospital
Chicago, Illinois, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Gene Therapy Center - Dept. of Pediatrics and Institute of Human Genetics
Minneapolis, Minnesota, United States
Children's Hospital
Buffalo, New York, United States
Mount Sinai School of Medicine
New York, New York, United States
University of Rochester School of Medicine
Rochester, New York, United States
Duke University Medical Center
Durham, North Carolina, United States
Children's Hospital Medical Center
Cincinnati, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
University of Pittsburgh
Pittsburgh, Pennsylvania, United States
Baylor College of Medicine
Houston, Texas, United States
University of Washington School of Medicine
Seattle, Washington, United States
Queen Elizabeth II Health Center
Halifax, Nova Scotia, Canada
North York General Hospital
Toronto, Ontario, Canada
Hopital du Sacre-Coeur de Montreal
Montreal, Quebec, Canada
University Hospital
Prague, , Czechia
Sopron Megyei Jogu Varos Erzsebet Korhaz
Sopron, , Hungary
Klinika Chorob Metabolicznych Instytut
Warsaw, , Poland
Hope Hospital
Manchester, , United Kingdom
Countries
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References
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Banikazemi M, Bultas J, Waldek S, Wilcox WR, Whitley CB, McDonald M, Finkel R, Packman S, Bichet DG, Warnock DG, Desnick RJ; Fabry Disease Clinical Trial Study Group. Agalsidase-beta therapy for advanced Fabry disease: a randomized trial. Ann Intern Med. 2007 Jan 16;146(2):77-86. doi: 10.7326/0003-4819-146-2-200701160-00148. Epub 2006 Dec 18.
Other Identifiers
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AGAL-008-00
Identifier Type: -
Identifier Source: org_study_id