Home
Search
Study Topics
Glossary
|
|
|
|
|
Sponsored by: |
Genzyme |
---|---|
Information provided by: | Genzyme |
ClinicalTrials.gov Identifier: | NCT00074958 |
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. This enzyme helps to break down and remove certain types of fatty substances called "glycolipids". These glycolipids are normally present within the body in most cells. In people with 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 levels (also referred to as "globotriaosylceramide" or "GL-3") in these tissues is thought to cause the clinical symptoms that are common to Fabry disease. Symptoms commonly appear during childhood with pain in the hands and feet. This study explored the safety, efficacy and pharmacokinetics of Fabrazyme in pediatric patients aged between 7 and 15 years.
Condition | Intervention | Phase |
---|---|---|
Fabry Disease |
Biological: Fabrazyme (agalsidase beta) |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety Study |
Official Title: | A Multi-center, Phase 2, Open-Label Study of Fabrazyme (Recombinant Human a-Galactosidase A) Replacement Therapy in Pediatric Patients With Fabry Disease |
Enrollment: | 16 |
Study Start Date: | October 2002 |
Study Completion Date: | July 2005 |
Primary Completion Date: | May 2005 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
---|---|
Fabrazyme: Experimental
1.0 mg/kg of Fabrazyme given to the patients every 2 weeks
|
Biological: Fabrazyme (agalsidase beta)
1 mg/kg every 2 weeks
|
Ages Eligible for Study: | 7 Years to 15 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion criteria:
Exclusion Criteria:
United States, Arizona | |
University of Arizona | |
Tucson, Arizona, United States, 85724 | |
France | |
Hopital Europeen Georges Pompidou | |
Paris, France, Cedex 15 | |
Hopital Edouard Herriot | |
Lyon, France, Cedex 03 | |
Hopital de la Timone Enfants | |
Marseille, France, Cedex 05 | |
Poland | |
Instytut Pomnik Centrum Zdrowia Dziecka | |
Warsaw, Poland, 04-730 | |
United Kingdom | |
Great Ormond Street Hospital for Sick Children | |
London, United Kingdom, WC1N 3JH | |
United Kingdom, Manchester | |
Royal Manchester Children's Hospital | |
Pendlebury, Manchester, United Kingdom, M27 4HA |
Study Director: | Medical Monitor | Genzyme |
Responsible Party: | Genzyme Corporation ( Medical Monitor ) |
Study ID Numbers: | AGAL-016-01 |
Study First Received: | December 24, 2003 |
Results First Received: | March 3, 2009 |
Last Updated: | August 11, 2009 |
ClinicalTrials.gov Identifier: | NCT00074958 History of Changes |
Health Authority: | United States: Food and Drug Administration |
a-Galactosidase A aGal r-haGAL |
Fabry GL-3 Fabrazyme |
Lipid Metabolism, Inborn Errors Sphingolipidoses Metabolic Diseases Lysosomal Storage Diseases Sphingolipidosis Central Nervous System Diseases Brain Diseases Metabolism, Inborn Errors Genetic Diseases, Inborn |
Fabry Disease Genetic Diseases, X-Linked Ceramide Trihexosidosis Brain Diseases, Metabolic, Inborn Lipidoses Metabolic Disorder Lipid Metabolism Disorders Brain Diseases, Metabolic |
Lipid Metabolism, Inborn Errors Sphingolipidoses Metabolic Diseases Lysosomal Storage Diseases, Nervous System Lysosomal Storage Diseases Nervous System Diseases Central Nervous System Diseases Brain Diseases |
Metabolism, Inborn Errors Genetic Diseases, Inborn Fabry Disease Genetic Diseases, X-Linked Brain Diseases, Metabolic, Inborn Lipidoses Lipid Metabolism Disorders Brain Diseases, Metabolic |