A Study of Fabrazyme in Pediatric Patients With Fabry Disease

Completed · Phase 2

Conditions studied: Fabry Disease

In brief

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.

Key facts

Study ID

NCT00074958

Run by

Genzyme, a Sanofi Company

People needed

16

Starts

2002-10-01

Expected to finish

2005-07-01

Last updated by the study team

2015-04-02

Who can join

Age: 7 and older, up to 15. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• University of Arizona — Tucson, Arizona, United States
• Hopital Edouard Herriot — Lyon, France
• Hopital de la Timone Enfants — Marseille, France
• Hopital Europeen Georges Pompidou — Paris, France
• Instytut Pomnik Centrum Zdrowia Dziecka — Warsaw, Poland
• Royal Manchester Children's Hospital — Pendlebury, Manchester, United Kingdom
• Great Ormond Street Hospital for Sick Children — London, United Kingdom

Full record on ClinicalTrials.gov

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