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

Fabry Disease Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00081497
• Other study ID #: AGAL02503
• Status: Completed
• Phase: Phase 4
• First received: April 14, 2004
• Last updated: March 13, 2015
• Start date: January 2004
• Est. completion date: September 2005

Study information

• Verified date: March 2015
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationCanada: Health CanadaHungary: National Institute of PharmacyCzech Republic: State Institute for Drug ControlPoland: Office for Registration of Medicinal Products, Medical Devices and Biocidal ProductsUnited Kingdom: Medicines and Healthcare Products Regulatory Agency
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 67
• Est. completion date: September 2005
• Est. primary completion date: September 2005
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 16 Years and older

Eligibility

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

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Fabry Disease

Intervention

Biological:
• agalsidase beta
1.0 mg/kg every 2 weeks

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Genzyme, a Sanofi Company

Countries where clinical trial is conducted

United States,  Canada,  Czech Republic,  Hungary,  Poland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Difference in Inverse Serum Creatinine Within Patients' Slopes Between the Placebo AGAL-008-00 (NCT00074984) and Fabrazyme AGAL02503 (NCT00081497) Periods	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.	Placebo period AGAL-008-00 (up to 35 months) through Fabrazyme period AGAL02503 (18 months)	No
Secondary	Serum Creatinine at 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).	Pre-Fabrazyme, 6, 12, and 18 months	No
Secondary	Estimated Glomerular Filtration Rate (eGFR) at Pre-Fabrazyme and 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).	Pre-Fabrazyme, 6, 12, and 18 months	No
Secondary	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=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.	Pre-Fabrazyme and 6, 12, and 18 months	No
Secondary	Proteinuria at 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).	Pre-Fabrazyme and 6, 12, and 18 months	No