Home
Search
Study Topics
Glossary
|
|
|
|
|
Tracking Information | |
---|---|
First Received Date † | December 24, 2003 |
Last Updated Date | July 16, 2007 |
Start Date † | October 1999 |
Current Primary Outcome Measures † |
|
Original Primary Outcome Measures † |
|
Change History | Complete list of historical versions of study NCT00074971 on ClinicalTrials.gov Archive Site |
Current Secondary Outcome Measures † |
|
Original Secondary Outcome Measures † |
|
Descriptive Information | |
Brief Title † | A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease |
Official Title † | 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 |
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. |
Detailed Description | |
Study Phase | Phase III |
Study Type † | Interventional |
Study Design † | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Condition † | Fabry Disease |
Intervention † | Drug: Fabrazyme (agalsidase beta) |
Study Arms / Comparison Groups | |
Publications * | |
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline. |
|
Recruitment Information | |
Recruitment Status † | Completed |
Enrollment † | 58 |
Completion Date | December 2004 |
Primary Completion Date | |
Eligibility Criteria † | Inclusion Criteria:
Exclusion criteria:
|
Gender | Both |
Ages | 16 Years and older |
Accepts Healthy Volunteers | No |
Contacts †† | |
Location Countries † | United States, France, Netherlands, Puerto Rico, United Kingdom |
Expanded Access Status | |
Administrative Information | |
NCT ID † | NCT00074971 |
Responsible Party | |
Secondary IDs †† | |
Study Sponsor † | Genzyme |
Collaborators †† | |
Investigators † | |
Information Provided By | Genzyme |
Verification Date | July 2005 |
† Required WHO trial registration data element. †† WHO trial registration data element that is required only if it exists. |