Expired Study
This study is not currently recruiting Study Participants on ClinicalConnection.com. If you would like to find active studies please search for clinical trials.

Tucson, Arizona 85724


People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. This enzyme helps to break down and remove certain types of fatty substances called "glycolipids". These glycolipids are normally present within the body in most cells. In people with 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 levels (also referred to as "globotriaosylceramide" or "GL-3") in these tissues is thought to cause the clinical symptoms that are common to Fabry disease. Symptoms commonly appear during childhood with pain in the hands and feet. This study explored the safety, efficacy and pharmacokinetics of Fabrazyme in pediatric patients aged between 7 and 15 years.


Inclusion criteria: - Patient or legal guardian must provide written informed consent - Patients must have a clinical diagnosis of Fabry disease and active Fabry disease (clinical signs and symptoms) - Patients must be at least 7 years of age but no older than 15 years of age at time of enrollment - Patients must be Tanner Stage ≤ III - Female patients must have a negative pregnancy test prior to each infusion and use a medically accepted form of contraception throughout the study Exclusion Criteria: - Patient has a clinically significant organic disease (with the exception of symptoms relating to Fabry disease) that in the opinion of the investigator would preclude participation in the trial - Patient has participated in a study employing investigational drug within 30 days of the start of this study - Patient has received prior treatment with enzyme replacement therapy - Patient is unable to comply with the clinical protocol



Primary Contact:

Study Director
Medical Monitor

Backup Contact:


Location Contact:

Tucson, Arizona 85724
United States

There is no listed contact information for this specific location.

Site Status: N/A

Data Source: ClinicalTrials.gov

Date Processed: November 18, 2019

Modifications to this listing: Only selected fields are shown, please use the link below to view all information about this clinical trial.

Click to view Full Listing

This study is not currently recruiting Study Participants on ClinicalConnection.com. The form below is not enabled.