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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 Patients

Slope 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

Page last updated: August 20, 2015

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