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A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease

This study has been completed.
Sponsor:
Information provided by:
Genzyme
ClinicalTrials.gov Identifier:
NCT00074971
First received: December 24, 2003
Last updated: August 11, 2009
Last verified: July 2005
History of Changes

December 24, 2003
August 11, 2009
October 1999
Not Provided
  • Safety and efficacy
  • Morphologic assessment of GL-3 inclusions in the capillary endothelium (vasculature) of the kidney
Same as current
Complete list of historical versions of study NCT00074971 on ClinicalTrials.gov Archive Site
  • Changes in McGill Pain Questionnaire
  • Autonomic status
  • Glomerular filtration
  • Functional assessment of urinary protein excretion Ophthalmic changes
  • SF-36 Health Survey
  • Physician's assessment of Fabry Symptoms and pain medication
Same as current
Not Provided
Not Provided
 
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

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.
Not Provided
Interventional
Phase 3
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Fabry Disease
Drug: Fabrazyme (agalsidase beta)
Not Provided
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
58
December 2004
Not Provided

Inclusion Criteria:

  • 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

Exclusion criteria:

  • 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
Both
16 Years and older
No
Contact information is only displayed when the study is recruiting subjects
United States,   France,   Netherlands,   Puerto Rico,   United Kingdom
 
NCT00074971
AGAL-005-99
Not Provided
Medical Monitor, Genzyme Coporation
Genzyme
Not Provided
Study Director: Medical Monitor Genzyme Coorporation
Genzyme
July 2005

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP