A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease
Information source: Sanofi
ClinicalTrials.gov processed this data on August 20, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Fabry Disease
Intervention: Fabrazyme (agalsidase beta) (Biological); Placebo (Biological)
Phase: Phase 4
Status: Completed
Sponsored by: Genzyme, a Sanofi Company Official(s) and/or principal investigator(s): Medical Monitor, Study Director, Affiliation: Genzyme Coorporation
Summary
People with Fabry disease have an alteration in their genetic material (DNA) which causes a
deficiency of the a-galactosidase A enzyme. Fabrazyme (agalsidase beta) 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 ("globotriaosylceramide" 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.
Clinical Details
Official title: Multi-Center, Randomized, Double-Blind, Placebo-Controlled Study of the Safety and Efficacy of Fabrazyme on Progression of Renal Disease and Significant Clinical Events in Patients With Fabry Disease
Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Primary outcome: Number of Participants Experiencing a Clinically Significant Renal, Cardiac or Cerebrovascular Event and/or Death in Fabrazyme (Agalsidase Beta) Patients as Compared to Placebo Patients
Secondary outcome: Number of Participants Experiencing a Renal Event in Fabrazyme (Agalsidase Beta) Patients as Compared to Placebo PatientsSlope of Estimated Glomerular Filtration Rate (eGFR) Comparing Placebo vs Fabrazyme (Agalsidase Beta) Patients Slope of Inverse Serum Creatinine Values Comparing Placebo vs Fabrazyme (Agalsidase Beta)Patients Neuropathic Pain as Assessed by Question 12 of the Brief Pain Inventory (BPI) Questionnaire (Pain at Its Worst)
Eligibility
Minimum age: 16 Years.
Maximum age: N/A.
Gender(s): Both.
Criteria:
Inclusion Criteria:
- Patients must provide written informed consent
- Patients must be at least 16 years old
- Patients must have a current diagnosis of Fabry disease and have a clinical
presentation consistent of Fabry disease (decreased sweating, Fabry pain,
angiokeratoma, etc.)
- Patients may not have received enzyme replacement therapy as a treatment for Fabry
disease
- Patients must have a documented plasma a-galactosidase A (aGAL) activity of < 1. 5
nmol/hr/mL or a documented leukocyte aGAL activity of < 4 nmol/hr/mg
- Patients must have one or more of the following: a serum creatinine measurement of
1. 2 to 3 mg/dL (106. 1 to 265 umol/L) OR estimated creatinine clearance < 80 mL/min
only if the patient's serum creatinine measurement is < 1. 2 mg/dL
- 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
Exclusion Criteria:
- Patient has undergone or is currently scheduled for kidney transplantation or is
currently on dialysis
- Patient has acute renal failure
- Patient has participated in a study employing an investigational drug within 30 days
of study entry
- Patient has diabetes mellitus or presence of confounding renal disease
- Patient has a history of transient ischemic attack (TIA) or ischemic stroke within 3
months of study entry documented by mild-to-moderate neurological deficit
- Patient has critical coronary disease
- Patient has congestive heart failure
- Patient has severe residual neurological deficit that will confound the detection of
new events as determined by an attending neurologist and/or Principal Investigator
- Patient is unwilling to comply with the requirements of the protocol or the patient
has a medical condition, serious intercurrent illness, or extenuating circumstances
that would significantly decrease study compliance, including prescribed follow-up
Locations and Contacts
University Hospital, Prague 128 08, Czech Republic
Sopron Megyei Jogu Varos Erzsebet Korhaz, Sopron 9400, Hungary
Klinika Chorob Metabolicznych Instytut, Warsaw 04-730, Poland
Hope Hospital, Manchester M6 8HD, United Kingdom
University of Alabama at Birmingham, Birmingham, Alabama 35294, United States
Cedars-Sinai Medical Center, Los Angeles, California 90048, United States
University of San Francisco, San Francisco, California 94143, United States
University of Connecticut Health Partners, Farmington, Connecticut 06119, United States
Oncology Hematology Association, Coral Springs, Florida 33065, United States
Emory University School of Medicine, Atlanta, Georgia 30322, United States
Children's Memorial Hospital, Chicago, Illinois 60614, United States
University of Kansas Medical Center, Kansas City, Kansas 66160, United States
Massachusetts General Hospital, Boston, Massachusetts 02114, United States
Gene Therapy Center - Dept. of Pediatrics and Institute of Human Genetics, Minneapolis, Minnesota 55455, United States
Children's Hospital, Buffalo, New York 14209, United States
Mount Sinai School of Medicine, New York, New York 10029, United States
University of Rochester School of Medicine, Rochester, New York 14642, United States
Duke University Medical Center, Durham, North Carolina 27710, United States
Queen Elizabeth II Health Center, Halifax, Nova Scotia B3H 1V8, Canada
Children's Hospital Medical Center, Cincinnati, Ohio 45229, United States
North York General Hospital, Toronto, Ontario M2K 1E1, Canada
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States
University of Pittsburgh, Pittsburgh, Pennsylvania 15261, United States
Hopital du Sacre-Coeur de Montreal, Montreal, Quebec H4J 1C5, Canada
Baylor College of Medicine, Houston, Texas 77030, United States
University of Washington School of Medicine, Seattle, Washington 98195, United States
Additional Information
Link to Result synopsis for AGAL00800
Starting date: February 2001
Last updated: December 2, 2013
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