A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease

NCT ID: NCT00081497

Last Updated: 2015-04-02

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE4

Total Enrollment

67 participants

Study Classification

INTERVENTIONAL

Study Start Date

2004-01-31

Study Completion Date

2005-09-30

Brief Summary

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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.

Detailed Description

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Conditions

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Fabry Disease

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Fabrazyme 1.0 mg/kg every 2 weeks

This is an open-label extension study to AGAL-008-00 (NCT00074984) and all patients received Fabrazyme treatment.

Group Type EXPERIMENTAL

agalsidase beta

Intervention Type BIOLOGICAL

1.0 mg/kg every 2 weeks

Interventions

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agalsidase beta

1.0 mg/kg every 2 weeks

Intervention Type BIOLOGICAL

Other Intervention Names

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Fabrazyme r-hαGAL

Eligibility Criteria

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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
Minimum Eligible Age

16 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Genzyme, a Sanofi Company

INDUSTRY

Sponsor Role lead

Responsible Party

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Genzyme Corporation

Principal Investigators

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Medical Monitor

Role: STUDY_DIRECTOR

Genzyme, a Sanofi Company

Locations

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University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status

Cedars-Sinai Medical Center

Los Angeles, California, United States

Site Status

University of San Francisco

San Francisco, California, United States

Site Status

University of Connecticut Health Partners

West Hartford, Connecticut, United States

Site Status

Oncology Hematology Association

Coral Springs, Florida, United States

Site Status

Emory University School of Medicine

Atlanta, Georgia, United States

Site Status

Children's Memorial Hospital

Chicago, Illinois, United States

Site Status

University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

Gene Therapy Center - Department of Pediatrics and Institute of Human Genetics

Minneapolis, Minnesota, United States

Site Status

Children's Hospital

Buffalo, New York, United States

Site Status

Mount Sinai School of Medicine

New York, New York, United States

Site Status

University of Rochester School of Medicine

Rochester, New York, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

University of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status

Baylor College of Medicine

Houston, Texas, United States

Site Status

University of Washington School of Medicine

Seattle, Washington, United States

Site Status

Queen Elizabeth II Health Center

Halifax, Nova Scotia, Canada

Site Status

North York General Hospital

Toronto, Ontario, Canada

Site Status

Hopital du Sacre-Coeur de Montreal

Montreal, Quebec, Canada

Site Status

University Hospital

Prague, , Czechia

Site Status

Sopron Megyei Jogu Varos Erzsebet Korhaz

Sopron, , Hungary

Site Status

Klinika Chorob Metabolicznych Instytut

Warsaw, , Poland

Site Status

Hope Hospital

Manchester, , United Kingdom

Site Status

Countries

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United States Canada Czechia Hungary Poland United Kingdom

Other Identifiers

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AGAL02503

Identifier Type: -

Identifier Source: org_study_id

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