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

• ClinicalTrials.gov Identifier: NCT00074971
• Recruitment Status: Completed
• First Posted: December 25, 2003
• Last Update Posted: December 4, 2013
• Sponsor: Genzyme, a Sanofi Company
• Information provided by: Sanofi

Tracking Information

• First Submitted Date: December 24, 2003
• First Posted Date: December 25, 2003
• Last Update Posted Date: December 4, 2013
• Study Start Date: October 1999
• Primary Completion Date: Not Provided

Current Primary Outcome Measures (submitted: June 23, 2005)

• Safety and efficacy
• Morphologic assessment of GL-3 inclusions in the capillary endothelium (vasculature) of the kidney

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: June 23, 2005)

• 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

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

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: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Non-Randomized; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Fabry Disease
• Intervention: Drug: Fabrazyme (agalsidase beta)
• Study Arms: Not Provided
• Publications *: 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.

Recruitment Information

• Recruitment Status: Completed
• Enrollment (submitted: June 23, 2005): 58
• Original Enrollment: Same as current
• Study Completion Date: December 2004
• Primary Completion Date: Not Provided

Eligibility Criteria

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 16 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: France, Netherlands, Puerto Rico, United Kingdom, United States

Administrative Information

• NCT Number: NCT00074971
• Other Study ID Numbers: AGAL-005-99
• Has Data Monitoring Committee: Not Provided
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: Medical Monitor, Genzyme Coporation
• Study Sponsor: Genzyme, a Sanofi Company
• Collaborators: Not Provided

Investigators

• Study Director: Medical Monitor; Genzyme Coorporation

• PRS Account: Sanofi
• Verification Date: December 2013