Back to resultsA Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
Primary Purpose
Fabry Disease
Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Fabrazyme (agalsidase beta)
Sponsored by
About this trial
This is an interventional treatment trial for Fabry Disease focused on measuring a-Galactosidase A, aGAL, r-haGAL, Fabry, GL-3, Fabrazyme
Eligibility Criteria
Inclusion Criteria: Patients must have successfully completed the previous double-blind study (AGAL-1-002-98) Patients must provide written informed consent prior to study participation Female patients must have a negative pregnancy test prior to each dosing and use a medically accepted method of contraception throughout the study Exclusion criteria: Patient has undergone kidney transplant or is currently on dialysis Patient is pregnant or lactating Patient is unwilling to comply with the requirements of the protocol Patient has a clinically significant organic disease (with the exception of symptoms related to Fabry disease), including clinically significant cardiovascular, hepatic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstances that, in the opinion of the investigator, would preclude participation in the study
Sites / Locations
- Cedars-Sinai Medical Center
- University of California San Fransisco
- Northwest Oncology & Hematology Associates
- Children's Memorial Hospital
- University of Iowa Hospital and Clinics
- Greater Baltimore Medical Center
- Beth Israel Deaconess Medical Center
- Fetal Diagnostic and Imaging Center
- University of New Mexico
- Mount Sinai School of Medicine
- University of Rochester School of Medicine
- Hart Family Practice
- Hematology/Oncology Associates of South Texas
- University of Washington School of Medicine
- Hospital Edouard Herriot
- Hospital Europeen Georges Pompidou
- Academisch Medisch Centrum
- University of Puerto Rico
- National Hospital for Neurology and Neurosurgery
- Hope Hospital
Outcomes
Primary Outcome Measures
Safety and efficacy
Morphologic assessment of GL-3 inclusions in the capillary endothelium (vasculature) of the kidney
Secondary Outcome Measures
Changes in McGill Pain Questionnaire
Autonomic status
Glomerular filtration
Functional assessment of urinary protein excretion Ophthalmic changes
SF-36 Health Survey
Physician's assessment of Fabry Symptoms and pain medication
Full Information
NCT ID
NCT00074971
First Posted
December 24, 2003
Last Updated
December 2, 2013
Sponsor
Genzyme, a Sanofi Company
1. Study Identification
Unique Protocol Identification Number
NCT00074971
Brief Title
A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
Official Title
A Multi-center, Open-Label Extension Study of the Safety and Efficacy of Recombinant Human a-Galactosidase A (r-haGAL) Replacement in Patients With Fabry Disease
Study Type
Interventional
2. Study Status
Record Verification Date
December 2013
Overall Recruitment Status
Completed
Study Start Date
October 1999 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
December 2004 (undefined)
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 a-galactosidase A enzyme. Fabrazyme is a drug that helps to breakdown and remove 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 will test the safety and efficacy of Fabrazyme in the treatment of patients with Fabry disease.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fabry Disease
Keywords
a-Galactosidase A, aGAL, r-haGAL, Fabry, GL-3, Fabrazyme
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
58 (false)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Fabrazyme (agalsidase beta)
Primary Outcome Measure Information:
Title
Safety and efficacy
Title
Morphologic assessment of GL-3 inclusions in the capillary endothelium (vasculature) of the kidney
Secondary Outcome Measure Information:
Title
Changes in McGill Pain Questionnaire
Title
Autonomic status
Title
Glomerular filtration
Title
Functional assessment of urinary protein excretion Ophthalmic changes
Title
SF-36 Health Survey
Title
Physician's assessment of Fabry Symptoms and pain medication
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-1-002-98)
Patients must provide written informed consent prior to study participation
Female patients must have a negative pregnancy test prior to each dosing and use a medically accepted method of contraception throughout the study
Exclusion criteria:
Patient has undergone kidney transplant or is currently on dialysis
Patient is pregnant or lactating
Patient is unwilling to comply with the requirements of the protocol
Patient has a clinically significant organic disease (with the exception of symptoms related to Fabry disease), including clinically significant cardiovascular, hepatic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstances that, in the opinion of the investigator, would preclude participation in the study
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Monitor
Organizational Affiliation
Genzyme Coorporation
Official's Role
Study Director
Facility Information:
Facility Name
Cedars-Sinai Medical Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90048
Country
United States
Facility Name
University of California San Fransisco
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
Northwest Oncology & Hematology Associates
City
Coral Springs
State/Province
Florida
ZIP/Postal Code
33065
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 Iowa Hospital and Clinics
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
Greater Baltimore Medical Center
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21204
Country
United States
Facility Name
Beth Israel Deaconess Medical Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
Facility Name
Fetal Diagnostic and Imaging Center
City
Billings
State/Province
Montana
ZIP/Postal Code
59101
Country
United States
Facility Name
University of New Mexico
City
Albuquerque
State/Province
New Mexico
ZIP/Postal Code
87131
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
Hart Family Practice
City
Hickory
State/Province
North Carolina
ZIP/Postal Code
28601
Country
United States
Facility Name
Hematology/Oncology Associates of South Texas
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78229
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
Hospital Edouard Herriot
City
Lyon
ZIP/Postal Code
Cedex 03
Country
France
Facility Name
Hospital Europeen Georges Pompidou
City
Paris
ZIP/Postal Code
Cedex 15
Country
France
Facility Name
Academisch Medisch Centrum
City
Amsterdam
ZIP/Postal Code
1105 AZ
Country
Netherlands
Facility Name
University of Puerto Rico
City
San Juan
ZIP/Postal Code
00935
Country
Puerto Rico
Facility Name
National Hospital for Neurology and Neurosurgery
City
London
ZIP/Postal Code
WC1N 3BG
Country
United Kingdom
Facility Name
Hope Hospital
City
Manchester
ZIP/Postal Code
M6 8HD
Country
United Kingdom
12. IPD Sharing Statement
Citations:
PubMed Identifier
25795794
Citation
Germain DP, Charrow J, Desnick RJ, Guffon N, Kempf J, Lachmann RH, Lemay R, Linthorst GE, Packman S, Scott CR, Waldek S, Warnock DG, Weinreb NJ, Wilcox WR. Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease. J Med Genet. 2015 May;52(5):353-8. doi: 10.1136/jmedgenet-2014-102797. Epub 2015 Mar 20.
Results Reference
derived
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A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
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