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

Phase 4

Completed

Conditions: Fabry Disease

Interventions: Biological: agalsidase beta

Registration Number: NCT00081497

Lead Sponsor: Genzyme, a Sanofi Company

Brief Summary: 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.

Detailed Description: Not available

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 67

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

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Fabrazyme 1.0 mg/kg every 2 weeks	agalsidase beta	This is an open-label extension study to AGAL-008-00 (NCT00074984) and all patients received Fabrazyme treatment.

Primary Outcome Measures

Name	Time	Method
Difference in Inverse Serum Creatinine Within Patients' Slopes Between the Placebo AGAL-008-00 (NCT00074984) and Fabrazyme AGAL02503 (NCT00081497) Periods	Placebo period AGAL-008-00 (up to 35 months) through Fabrazyme period AGAL02503 (18 months)	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

Name	Time	Method
Serum Creatinine at Pre-Fabrazyme and 6, 12, and 18 Months	Pre-Fabrazyme, 6, 12, and 18 months	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	Pre-Fabrazyme, 6, 12, and 18 months	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	Pre-Fabrazyme and 6, 12, and 18 months	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	Pre-Fabrazyme and 6, 12, and 18 months	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).

Trial Locations

Locations (25): Oncology Hematology Association
🇺🇸
Coral Springs, Florida, United States
University of San Francisco
🇺🇸
San Francisco, California, United States
University of Connecticut Health Partners
🇺🇸
West Hartford, Connecticut, United States
Gene Therapy Center - Department of Pediatrics and Institute of Human Genetics
🇺🇸
Minneapolis, Minnesota, United States
Duke University Medical Center
🇺🇸
Durham, North Carolina, United States
Children's Hospital
🇺🇸
Buffalo, New York, United States
North York General Hospital
🇨🇦
Toronto, Ontario, Canada
University Hospital
🇨🇿
Prague, Czech Republic
Klinika Chorob Metabolicznych Instytut
🇵🇱
Warsaw, Poland
University of Pittsburgh
🇺🇸
Pittsburgh, Pennsylvania, United States
University of Alabama at Birmingham
🇺🇸
Birmingham, Alabama, United States
Cedars-Sinai Medical Center
🇺🇸
Los Angeles, California, United States
Children's Memorial Hospital
🇺🇸
Chicago, Illinois, United States
Hopital du Sacre-Coeur de Montreal
🇨🇦
Montreal, Quebec, Canada
Mount Sinai School of Medicine
🇺🇸
New York, New York, United States
Children's Hospital of Philadelphia
🇺🇸
Philadelphia, Pennsylvania, United States
Emory University School of Medicine
🇺🇸
Atlanta, Georgia, United States
Children's Hospital Medical Center
🇺🇸
Cincinnati, Ohio, United States
Sopron Megyei Jogu Varos Erzsebet Korhaz
🇭🇺
Sopron, Hungary
Queen Elizabeth II Health Center
🇨🇦
Halifax, Nova Scotia, Canada
Hope Hospital
🇬🇧
Manchester, United Kingdom
University of Washington School of Medicine
🇺🇸
Seattle, Washington, United States
University of Rochester School of Medicine
🇺🇸
Rochester, New York, United States
Baylor College of Medicine
🇺🇸
Houston, Texas, United States
University of Kansas Medical Center
🇺🇸
Kansas City, Kansas, United States