Clinical trial NCT00081497: A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Fabry Disease
Keywords: alpha-galactosidase A
a-GAL
r-haGAL
Fabry
GL-3
Fabrazyme
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2004-04-16
Enrollment: 67
Sex: ALL
Minimum age (years): 16
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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

Primary outcomes:
measure: Difference in Inverse Serum Creatinine Within Patients' Slopes Between the Placebo AGAL-008-00 (NCT00074984) and Fabrazyme AGAL02503 (NCT00081497) Periods
timeFrame: Placebo period AGAL-008-00 (up to 35 months) through Fabrazyme period AGAL02503 (18 months)
Secondary outcomes:
measure: Serum Creatinine at Pre-Fabrazyme and 6, 12, and 18 Months
timeFrame: Pre-Fabrazyme, 6, 12, and 18 months
measure: Estimated Glomerular Filtration Rate (eGFR) at Pre-Fabrazyme and 6, 12, and 18 Months
timeFrame: Pre-Fabrazyme, 6, 12, and 18 months
measure: Plasma Globotriaosylceramide (GL-3) (Normal Plasma GL-3 Level is ≤ 7.03 µg/mL) at Pre-Fabrazyme and 6, 12, and 18 Months
timeFrame: Pre-Fabrazyme and 6, 12, and 18 months
measure: Proteinuria at Pre-Fabrazyme and 6, 12, and 18 Months
timeFrame: Pre-Fabrazyme and 6, 12, and 18 months
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Los Angeles
country: United States
facility: Cedars-Sinai Medical Center
state: California
city: San Francisco
country: United States
facility: University of San Francisco
state: California
city: West Hartford
country: United States
facility: University of Connecticut Health Partners
state: Connecticut
city: Coral Springs
country: United States
facility: Oncology Hematology Association
state: Florida
city: Atlanta
country: United States
facility: Emory University School of Medicine
state: Georgia
city: Chicago
country: United States
facility: Children's Memorial Hospital
state: Illinois
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Minneapolis
country: United States
facility: Gene Therapy Center - Department of Pediatrics and Institute of Human Genetics
state: Minnesota
city: Buffalo
country: United States
facility: Children's Hospital
state: New York
city: New York
country: United States
facility: Mount Sinai School of Medicine
state: New York
city: Rochester
country: United States
facility: University of Rochester School of Medicine
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Cincinnati
country: United States
facility: Children's Hospital Medical Center
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh
state: Pennsylvania
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Seattle
country: United States
facility: University of Washington School of Medicine
state: Washington
city: Halifax
country: Canada
facility: Queen Elizabeth II Health Center
state: Nova Scotia
city: Toronto
country: Canada
facility: North York General Hospital
state: Ontario
city: Montreal
country: Canada
facility: Hopital du Sacre-Coeur de Montreal
state: Quebec
city: Prague
country: Czech Republic
facility: University Hospital
city: Sopron
country: Hungary
facility: Sopron Megyei Jogu Varos Erzsebet Korhaz
city: Warsaw
country: Poland
facility: Klinika Chorob Metabolicznych Instytut
city: Manchester
country: United Kingdom
facility: Hope Hospital
Study officials:
affiliation: Genzyme, a Sanofi Company
name: Medical Monitor
role: STUDY_DIRECTOR
References: