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Home > "A" Clinical Trials Conditions > A Study of Fabrazyme in Pediatric Patients with Fabry Disease  A Study of Fabrazyme in Pediatric Patients with Fabry Disease
A Study of Fabrazyme in Pediatric Patients with Fabry Disease
For Condition: Fabry Disease
Status: No longer recruiting
Sponsor(s): Genzyme ,
Synopsis: 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 "globotriasylceramide" 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 will explore the safety and pharmacokinetics of Fabrazyme in pediatric patients aged between 7 and 15 years.
Details:
Eligibility:
Study Type: Interventional, Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety Study
Minimum Age/Maximum Age: 7 Years/15 Years
Genders: Both
Protocol Entry Criteria: 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 between age 7 and 15 - 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
Total Enrollment: 20
Location and Contact Information:
Istituto per l'Infanzia Burlo Garofolo
Trieste, , 34137
Italy
University of Arizona
Tucson, Arizona, 85724
United States
Hopital de la Timone Enfants
Marseille, , Cedex 05
France
Hopital Europeen Georges Pompidou
Paris, , Cedex 15
France
Hopital Edouard Herriot
Lyon, , Cedex 03
France
Great Ormond Street Hospital for Sick Children
London, , WC1N 3JH
United Kingdom
Instytut Pomnik Centrum Zdrowia Dziecka
Warsaw, , 04-730
Poland
Additional Information:
Study ID Numbers: AGAL-016-01;
Study Start Date:
Record last reviewed: December 2003
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00074958
Other Fabry Disease Studies:
1. Dosing Study of Replagal in Patients with Fabry Disease
2. A Study of the Safety and Efficacy of Fabrazyme in Patients with Fabry Disease
3. Data Collection in Women with Fabry Disease
4. Vasodilation in Patients with Fabry's Disease
5. A Study of Fabrazyme in Pediatric Patients with Fabry Disease
Related Studies:
Other Fabry Disease Clinical Trials
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A Study of Fabrazyme in Pediatric Patients with Fabry Disease
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