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A Study of the Safety and Efficacy of Fabrazyme as Compared to Placebo in Patients with Advanced Fabry Disease Clinical Trials Resources presented on Clinical Trials Search isn't meant to be a substitute for qualified health advice, visits or professional assistance with a real medical. We aren't doctors. Always consult your mD about A Study of the Safety and Efficacy of Fabrazyme as Compared to Placebo in Patients with Advanced Fabry Disease conditions. Clinical Trials Search.org is a website dedicated to listing clinical research studies in human subjects. A Study of the Safety and Efficacy of Fabrazyme as Compared to Placebo in Patients with Advanced Fabry Disease Clinical research trials and A Study of the Safety and Efficacy of Fabrazyme as Compared to Placebo in Patients with Advanced Fabry Disease health trials occur in a lot of of places throughout the United States of America. A clinical trial or clinical study is a research project with human volunteer subjects. Clinical drug trials and pharmaceutical clinical trials typically assess the effectivity of new does drugs. The role of the studies / projects is to resolve certain human healthcare questions. Clinical trials are a popular way for doctors, government agencies, and private sector corporations to detect remedies for all varieties of circumstances, such as A Study of the Safety and Efficacy of Fabrazyme as Compared to Placebo in Patients with Advanced Fabry Disease. A Study of the Safety and Efficacy of Fabrazyme as Compared to Placebo in Patients with Advanced Fabry Disease Clinical Trials and other clinical trials allow volunteers to obtain health treatment choices before they are available to the general public. Most times the human subjects recieve professional assistance for free of charge, and every now and again they are paid for their time. Sometimes there is a cost for a A Study of the Safety and Efficacy of Fabrazyme as Compared to Placebo in Patients with Advanced Fabry Disease clinical trial. Human subjects frequently get the finest healthcare available for their A Study of the Safety and Efficacy of Fabrazyme as Compared to Placebo in Patients with Advanced Fabry Disease condition. Risks are a reality, however, and may include extra or frequent physician visits, medical dangers (possibly life-threatening), and/or the treatment being uneffective. Trials are federally governed with strict guidelines to protect clinical trials patients.
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Home > "A" Clinical Trials Conditions > A Study of the Safety and Efficacy of Fabrazyme as Compared to Placebo in Patients with Advanced Fabry Disease  A Study of the Safety and Efficacy of Fabrazyme as Compared to Placebo in Patients with Advanced Fabry Disease
A Study of the Safety and Efficacy of Fabrazyme as Compared to Placebo in Patients with Advanced Fabry Disease
For Condition: Fabry Disease
Status: Completed
Sponsor(s): Genzyme ,
Synopsis: People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. Fabrayzme 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 ("globtriaosylceramide" 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.
Details:
Eligibility:
Study Type: Interventional, Treatment, Randomized, Double-Blind, Placebo Control, Parallel Assignment, Safety/Efficacy Study
Minimum Age/Maximum Age: 16 Years/
Genders: Both
Protocol Entry Criteria: Inclusion Criteria: - Patients must provide written informed consent - Patients must be at least 16 years old - Patients must have a current diagnosis of Fabry disease - 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 undergone or is currently scheduled for kidney transplanatation or is currently on dialysis - 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 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
Total Enrollment: 80
Location and Contact Information:
Duke University Medical Center
Durham, North Carolina, 27710
United States
University of Pittsburgh
Pittsburgh, Pennsylvania, 15261
United States
Sopron Megyei Jogu Varos Erzsebet Korhaz
Sopron, , 9400
Hungary
Children's Memorial Hospital
Chicago, Illinois, 60614
United States
Cedars-Sinai Medical Center
Los Angeles, California, 90048
United States
Queen Elizabeth II Health Center
Halifax, Nova Scotia, B3H 1V8
Canada
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
United States
University of Connecticut Health Partners
West Hartford, Connecticut, 06119
United States
Hopital du Sacre-Coeur de Montreal
Montreal, Quebec, H4J 1C5
Canada
Mount Sinai School of Medicine
New York City, New York, 10029
United States
Children's Hospital
Buffalo, New York, 14209
United States
University of Washington School of Medicine
Seattle, Washington, 98195
United States
Children's Hospital Medical Center
Cincinnati, Ohio, 45229
United States
University of Rochester School of Medicine
Rochester, New York, 14642
United States
University Hospital
Prague, , 128 08
Czech Republic
Gene Therapy Center - Dept. of Pediatrics and Institute of Human Genetics
Minneapolis, Minnesota, 55455
United States
Emory University School of Medicine
Atlanta, Georgia, 30322
United States
University of Alabama at Birmingham
Birmingham, Alabama, 35294
United States
Hope Hospital
Manchester, , M6 8HD
United Kingdom
University of San Francisco
San Francisco, California, 94143
United States
North York General Hospital
Toronto, Ontario, M2K 1E1
Canada
University of Kansas Medical Center
Kansas City, Kansas, 66160
United States
Baylor College of Medicine
Houston, Texas, 77030
United States
Oncology Hematology Association
Coral Springs, Florida, 33065
United States
Klinika Chorob Metabolicznych Instytut
Warsaw, , 04-730
Poland
Additional Information:
Study ID Numbers: AGAL-008-00;
Study Start Date: February 2001
Record last reviewed: April 2004
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00074984
Other Fabry Disease Studies:
1. An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years with Fabry Disease
2. Registry of Fabry Disease - A Multicenter Observational Study
3. A Study of the Safety and Efficacy of Fabrazyme in Patients with Fabry Disease
4. Alpha-Galactosidase A Replacement Therapy for Fabry Disease
5. PET Scans in Normal Volunteers and Patients with Fabry Disease
Related Studies:
Other Fabry Disease Clinical Trials
Other Clinical Trials
Other Prague Clinical Trials
A Study of the Safety and Efficacy of Fabrazyme as Compared to Placebo in Patients with Advanced Fabry Disease
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