At a glance
ClinicalIndex Comparison Record- ✓Clinical diagnosis of Fabry disease with active clinical signs and symptoms
- ✓Age 7-15 years at enrollment
- ✓Tanner Stage ≤ III
- ✕Clinically significant organic disease (except symptoms related to Fabry disease)
- ✕Prior enzyme replacement therapy (ERT)
- ✕Participation in investigational drug study within 30 days
- ✕Unable to comply with clinical protocol
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A Multi-center, Phase 2, Open-Label Study of Fabrazyme (Recombinant Human a-Galactosidase A) Replacement Therapy in Pediatric Patients With Fabry Disease
In Brief
A Phase 2 clinical trial evaluating Fabrazyme (agalsidase beta) for Fabry Disease. Completed, enrolled 16 participants across 7 sites in 4 countries.
Detailed Summary
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.
Study Details
Timeline
Interventions
1 mg/kg every 2 weeks