A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease - Full Text View

Sponsor:	Genzyme
Information provided by:	Genzyme
ClinicalTrials.gov Identifier:	NCT00081497

Purpose

People with Fabry Disease have an alteration in their genetic material (DNA) which causes a deficiency of the alpha-galactosidase A enzyme. Fabrazyme (agalsidase beta) is a drug that helps to break down and removes 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 analyzed the safety and efficacy of Fabrazyme in the treatment of patients with Fabry disease that previously participated in the AGAL-008-00 (NCT0074984) study.

Condition	Intervention	Phase
Fabry Disease	Biological: agalsidase beta	Phase IV

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	Multi-Center, Open-Label Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease That Previously Participated in the AGAL-008-00 Study

Resource links provided by NLM:

Genetics Home Reference related topics: Chanarin-Dorfman syndrome cholesteryl ester storage disease Fabry disease Farber lipogranulomatosis Schindler disease succinic semialdehyde dehydrogenase deficiency

U.S. FDA Resources

Further study details as provided by Genzyme:

Primary Outcome Measures:

Difference in Inverse Serum Creatinine Within Patients' Slopes Between the Placebo AGAL-008-00 (NCT00074984) and Fabrazyme AGAL02503 (NCT00081497) Periods [ Time Frame: Placebo period AGAL-008-00 (up to 35 months) through Fabrazyme period AGAL02503 (18 months) ] [ Designated as safety issue: No ]
The primary efficacy analysis was the summary of change in slope of inverse serum creatinine for Placebo/Fabrazyme patients in the Intent to Treat (ITT) Population. It compared the placebo period slope with the Fabrazyme period slope.

Secondary Outcome Measures:

Serum Creatinine at Pre-Fabrazyme and 6, 12, and 18 Months [ Time Frame: Pre-Fabrazyme, 6, 12, and 18 months ] [ Designated as safety issue: No ]
Pre-Fabrazyme=baseline visit of AGAL-008-00 (NCT00074984) for Fabrazyme patients; assessment prior to open-label for placebo patients who transitioned to Fabrazyme in AGAL-008-00 (NCT00074984); assessment prior to first Fabrazyme infusion in AGAL02503 (NCT00081497) for placebo patients who did not transition to Fabrazyme in AGAL-008-00 (NCT00074984).
Estimated Glomerular Filtration Rate (eGFR) at Pre-Fabrazyme and 6, 12, and 18 Months [ Time Frame: Pre-Fabrazyme, 6, 12, and 18 months ] [ Designated as safety issue: No ]
Pre-Fabrazyme=baseline visit of AGAL-00-800 (NCT00074984) for Fabrazyme patients; assessment prior to open-label for placebo patients who transitioned to Fabrazyme in AGAL-008-00 (NCT00074984); assessment prior to first Fabrazyme infusion in AGAL02503 (NCT00081497) for placebo patients who did not transition to Fabrazyme in AGAL-008-00 (NCT00074984).
Plasma Globotriaosylceramide (GL-3) (Normal Plasma GL-3 Level is ≤ 7.03 µg/mL) at Pre-Fabrazyme and 6, 12, and 18 Months [ Time Frame: Pre-Fabrazyme and 6, 12, and 18 months ] [ Designated as safety issue: No ]
Pre-Fabrazyme=baseline visit of AGAL00800 for Fabrazyme patients; assessment prior to open-label for placebo patients who transitioned to Fabrazyme in AGAL00800; assessment prior to first Fabrazyme infusion in AGAL02503 for placebo patients who did not transition to Fabrazyme in AGAL00800.
Proteinuria at Pre-Fabrazyme and 6, 12, and 18 Months [ Time Frame: Pre-Fabrazyme and 6, 12, and 18 months ] [ Designated as safety issue: No ]
Pre-Fabrazyme=baseline visit of AGAL-008-00 (NCT00074984) for Fabrazyme patients; assessment prior to open-label for placebo patients who transitioned to Fabrazyme in AGAL-008-00 (NCT00074984); assessment prior to first Fabrazyme infusion in AGAL02503 (NCT00081497) for placebo patients who did not transition to Fabrazyme in AGAL-008-00 (NCT00074984).

Enrollment:	67
Study Start Date:	January 2004
Study Completion Date:	September 2005
Primary Completion Date:	September 2005 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: Fabrazyme 1.0 mg/kg every 2 weeks This is an open-label extension study to AGAL-008-00 (NCT00074984) and all patients received Fabrazyme treatment.	Biological: agalsidase beta 1.0 mg/kg every 2 weeks Other Names: Fabrazyme r-hαGAL

Eligibility

Ages Eligible for Study:	16 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Patients must have successfully completed the previous double-blind study AGAL-008-00 (NCT00074984)
Patients must provide written informed consent prior to study participation
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 throughout the study

Exclusion Criteria:

The patient was unable to complete AGAL-008-00 (NCT00074984)
The patient has undergone kidney transplantation or is currently on dialysis
The patient has diabetes mellitus or presence of confounding renal disease
The patient has a clinically significant organic disease or an unstable condition that precludes participation
The patient is unwilling to comply with the protocol requirements

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00081497

Show 25 Study Locations

Sponsors and Collaborators

Genzyme

Investigators

Study Director:

Medical Monitor

Genzyme

More Information

Additional Information:

Link to Results Synopsis for AGAL02503 This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party:	Medical Monitor, Genzyme Corporation
ClinicalTrials.gov Identifier:	NCT00081497 History of Changes
Other Study ID Numbers:	AGAL02503
Study First Received:	April 14, 2004
Results First Received:	December 17, 2008
Last Updated:	August 18, 2010
Health Authority:	United States: Food and Drug Administration; Canada: Health Canada; Hungary: National Institute of Pharmacy; Czech Republic: State Institute for Drug Control; Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products; United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Genzyme:

alpha-galactosidase A
a-GAL
r-haGAL

Fabry
GL-3
Fabrazyme

Additional relevant MeSH terms:

Fabry Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases

Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on February 09, 2012