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
Data Collection
Fabry Disease+17
+ Brain Diseases
+ Brain Diseases, Metabolic
Treatment Study
Summary
Study start date: October 1, 1999
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 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.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.58 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-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
Study Plan
Find out more about all the medication administered in this study, their detailed description and what they involve.Study 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 20 locations
University of California San Fransisco
San Francisco, United StatesNorthwest Oncology & Hematology Associates
Coral Springs, United StatesChildren's Memorial Hospital
Chicago, United States