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
agalsidase beta
Fabry Disease+17
+ Brain Diseases
+ Brain Diseases, Metabolic
Treatment Study
Summary
Study start date: January 1, 2004
Actual date on which the first participant was enrolled.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.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.67 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.Over 16 Years
Range of ages for which participants are eligible to join.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
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
Study Plan
Find out more about all the medication administered in this study, their detailed description and what they involve.One single intervention group is designated in this study
This study does not include a placebo group
Treatment Groups
Group I
ExperimentalStudy Objectives
Primary Objectives
Secondary Objectives
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 25 locations
Cedars-Sinai Medical Center
Los Angeles, United StatesUniversity of San Francisco
San Francisco, United StatesUniversity of Connecticut Health Partners
West Hartford, United States