Back to resultsA Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
Primary Purpose
Fabry Disease
Status
Completed
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
agalsidase beta
Sponsored by
About this trial
This is an interventional treatment trial for Fabry Disease focused on measuring alpha-galactosidase A, a-GAL, r-haGAL, Fabry, GL-3, Fabrazyme
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
Sites / Locations
- University of Alabama at Birmingham
- Cedars-Sinai Medical Center
- University of San Francisco
- University of Connecticut Health Partners
- Oncology Hematology Association
- Emory University School of Medicine
- Children's Memorial Hospital
- University of Kansas Medical Center
- Gene Therapy Center - Department of Pediatrics and Institute of Human Genetics
- Children's Hospital
- Mount Sinai School of Medicine
- University of Rochester School of Medicine
- Duke University Medical Center
- Children's Hospital Medical Center
- Children's Hospital of Philadelphia
- University of Pittsburgh
- Baylor College of Medicine
- University of Washington School of Medicine
- Queen Elizabeth II Health Center
- North York General Hospital
- Hopital du Sacre-Coeur de Montreal
- University Hospital
- Sopron Megyei Jogu Varos Erzsebet Korhaz
- Klinika Chorob Metabolicznych Instytut
- Hope Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Fabrazyme 1.0 mg/kg every 2 weeks
Arm Description
This is an open-label extension study to AGAL-008-00 (NCT00074984) and all patients received Fabrazyme treatment.
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
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).
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).
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.
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).
Full Information
NCT ID
NCT00081497
First Posted
April 14, 2004
Last Updated
March 13, 2015
Sponsor
Genzyme, a Sanofi Company
1. Study Identification
Unique Protocol Identification Number
NCT00081497
Brief Title
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
2. Study Status
Record Verification Date
March 2015
Overall Recruitment Status
Completed
Study Start Date
January 2004 (undefined)
Primary Completion Date
September 2005 (Actual)
Study Completion Date
September 2005 (Actual)
3. Sponsor/Collaborators
Name of the Sponsor
Genzyme, a Sanofi Company
4. Oversight
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fabry Disease
Keywords
alpha-galactosidase A, a-GAL, r-haGAL, Fabry, GL-3, Fabrazyme
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
67 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Fabrazyme 1.0 mg/kg every 2 weeks
Arm Type
Experimental
Arm Description
This is an open-label extension study to AGAL-008-00 (NCT00074984) and all patients received Fabrazyme treatment.
Intervention Type
Biological
Intervention Name(s)
agalsidase beta
Other Intervention Name(s)
Fabrazyme, r-hαGAL
Intervention Description
1.0 mg/kg every 2 weeks
Primary Outcome Measure Information:
Title
Difference in Inverse Serum Creatinine Within Patients' Slopes Between the Placebo AGAL-008-00 (NCT00074984) and Fabrazyme AGAL02503 (NCT00081497) Periods
Description
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.
Time Frame
Placebo period AGAL-008-00 (up to 35 months) through Fabrazyme period AGAL02503 (18 months)
Secondary Outcome Measure Information:
Title
Serum Creatinine at Pre-Fabrazyme and 6, 12, and 18 Months
Description
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).
Time Frame
Pre-Fabrazyme, 6, 12, and 18 months
Title
Estimated Glomerular Filtration Rate (eGFR) at Pre-Fabrazyme and 6, 12, and 18 Months
Description
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).
Time Frame
Pre-Fabrazyme, 6, 12, and 18 months
Title
Plasma Globotriaosylceramide (GL-3) (Normal Plasma GL-3 Level is ≤ 7.03 µg/mL) at Pre-Fabrazyme and 6, 12, and 18 Months
Description
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.
Time Frame
Pre-Fabrazyme and 6, 12, and 18 months
Title
Proteinuria at Pre-Fabrazyme and 6, 12, and 18 Months
Description
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).
Time Frame
Pre-Fabrazyme and 6, 12, and 18 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Monitor
Organizational Affiliation
Genzyme, a Sanofi Company
Official's Role
Study Director
Facility Information:
Facility Name
University of Alabama at Birmingham
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35294-0006
Country
United States
Facility Name
Cedars-Sinai Medical Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90048
Country
United States
Facility Name
University of San Francisco
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
University of Connecticut Health Partners
City
West Hartford
State/Province
Connecticut
ZIP/Postal Code
06119
Country
United States
Facility Name
Oncology Hematology Association
City
Coral Springs
State/Province
Florida
ZIP/Postal Code
33065
Country
United States
Facility Name
Emory University School of Medicine
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Children's Memorial Hospital
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60614
Country
United States
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160-7233
Country
United States
Facility Name
Gene Therapy Center - Department of Pediatrics and Institute of Human Genetics
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Facility Name
Children's Hospital
City
Buffalo
State/Province
New York
ZIP/Postal Code
14209
Country
United States
Facility Name
Mount Sinai School of Medicine
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
University of Rochester School of Medicine
City
Rochester
State/Province
New York
ZIP/Postal Code
14642
Country
United States
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
University of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15261
Country
United States
Facility Name
Baylor College of Medicine
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
University of Washington School of Medicine
City
Seattle
State/Province
Washington
ZIP/Postal Code
98195
Country
United States
Facility Name
Queen Elizabeth II Health Center
City
Halifax
State/Province
Nova Scotia
ZIP/Postal Code
B3H 1V8
Country
Canada
Facility Name
North York General Hospital
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M2K 1E1
Country
Canada
Facility Name
Hopital du Sacre-Coeur de Montreal
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H4J 1C5
Country
Canada
Facility Name
University Hospital
City
Prague
Country
Czech Republic
Facility Name
Sopron Megyei Jogu Varos Erzsebet Korhaz
City
Sopron
ZIP/Postal Code
9400
Country
Hungary
Facility Name
Klinika Chorob Metabolicznych Instytut
City
Warsaw
ZIP/Postal Code
04-730
Country
Poland
Facility Name
Hope Hospital
City
Manchester
ZIP/Postal Code
M6 8HD
Country
United Kingdom
12. IPD Sharing Statement
Learn more about this trial
A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
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