A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
A Multi-center, Open-Label Extension Study of the Safety and Efficacy of Recombinant Human a-Galactosidase A (r-haGAL) Replacement in Patients With Fabry Disease
1 other identifier
interventional
58
5 countries
20
Brief Summary
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. Fabrazyme is a drug that helps to breakdown and remove certain types of fatty substances called "glycolipids." These glycolipids are normally present within the body in most cells. In Fabry disease, glycolipids build up in various tissues such as the liver, kidney, skin, and blood vessels because a-galactosidase A is not present, or is present in small quantities. The build up of glycolipid ("globatriaosylceramide" or "GL-3") levels in these tissues in particular is thought to cause the clinical symptoms that are common to Fabry disease. This study will test the safety and efficacy of Fabrazyme in the treatment of patients with Fabry disease.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Oct 1999
Longer than P75 for phase_3
20 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
October 1, 1999
CompletedFirst Submitted
Initial submission to the registry
December 24, 2003
CompletedFirst Posted
Study publicly available on registry
December 25, 2003
CompletedStudy Completion
Last participant's last visit for all outcomes
December 1, 2004
CompletedDecember 4, 2013
December 1, 2013
December 24, 2003
December 2, 2013
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Safety and efficacy
Morphologic assessment of GL-3 inclusions in the capillary endothelium (vasculature) of the kidney
Secondary Outcomes (6)
Changes in McGill Pain Questionnaire
Autonomic status
Glomerular filtration
Functional assessment of urinary protein excretion Ophthalmic changes
SF-36 Health Survey
- +1 more secondary outcomes
Interventions
Eligibility Criteria
You may qualify if:
- Patients must have successfully completed the previous double-blind study (AGAL-1-002-98)
- Patients must provide written informed consent prior to study participation
- Female patients must have a negative pregnancy test prior to each dosing and use a medically accepted method of contraception throughout the study
You may not qualify if:
- Patient has undergone kidney transplant or is currently on dialysis
- Patient is pregnant or lactating
- Patient is unwilling to comply with the requirements of the protocol
- Patient has a clinically significant organic disease (with the exception of symptoms related to Fabry disease), including clinically significant cardiovascular, hepatic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstances that, in the opinion of the investigator, would preclude participation in the study
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (20)
Cedars-Sinai Medical Center
Los Angeles, California, 90048, United States
University of California San Fransisco
San Francisco, California, 94143, United States
Northwest Oncology & Hematology Associates
Coral Springs, Florida, 33065, United States
Children's Memorial Hospital
Chicago, Illinois, 60614, United States
University of Iowa Hospital and Clinics
Iowa City, Iowa, 52242, United States
Greater Baltimore Medical Center
Baltimore, Maryland, 21204, United States
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215, United States
Fetal Diagnostic and Imaging Center
Billings, Montana, 59101, United States
University of New Mexico
Albuquerque, New Mexico, 87131, United States
Mount Sinai School of Medicine
New York, New York, 10029, United States
University of Rochester School of Medicine
Rochester, New York, 14642, United States
Hart Family Practice
Hickory, North Carolina, 28601, United States
Hematology/Oncology Associates of South Texas
San Antonio, Texas, 78229, United States
University of Washington School of Medicine
Seattle, Washington, 98195, United States
Hospital Edouard Herriot
Lyon, Cedex 03, France
Hospital Europeen Georges Pompidou
Paris, Cedex 15, France
Academisch Medisch Centrum
Amsterdam, 1105 AZ, Netherlands
University of Puerto Rico
San Juan, 00935, Puerto Rico
National Hospital for Neurology and Neurosurgery
London, WC1N 3BG, United Kingdom
Hope Hospital
Manchester, M6 8HD, United Kingdom
Related Publications (1)
Germain DP, Charrow J, Desnick RJ, Guffon N, Kempf J, Lachmann RH, Lemay R, Linthorst GE, Packman S, Scott CR, Waldek S, Warnock DG, Weinreb NJ, Wilcox WR. Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease. J Med Genet. 2015 May;52(5):353-8. doi: 10.1136/jmedgenet-2014-102797. Epub 2015 Mar 20.
PMID: 25795794DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Medical Monitor
Genzyme Coorporation
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
Study Record Dates
First Submitted
December 24, 2003
First Posted
December 25, 2003
Study Start
October 1, 1999
Study Completion
December 1, 2004
Last Updated
December 4, 2013
Record last verified: 2013-12