A Phase 1 interventional study of Replagal agalsidase alfa and Replagal in Fabry Disease, sponsored by National Institute of Neurological Disorders and Stroke (NINDS). Completed at 1 site in United States. Open to male participants aged 39 Years to 45 Years. Per ClinicalTrials.gov, last updated 2011-08-17.
Sponsored by National Institute of Neurological Disorders and Stroke (NINDS) · Phase 1, Interventional, and Treatment
This study will continue to evaluate the safety of using intravenous doses of Replagal for two patients with Fabry disease. Fabry disease is a genetic disorder inherited as an X-linked recessive trait. It causes a deficiency in the enzyme alpha galactosidase, which normally breaks down a lipid, or fatty substance called ceramidetrihexoside, a building block in all cells of the body. The deficiency in breaking down the lipid eventually causes that lipid to accumulate and injure cells. Vascular, renal, and neurological problems are the results. It is not known exactly how lipid accumulation brings about such problems, studies of another lipid storage disorder.
Two patients 7 to 17 years of age who have Fabry disease and have been receiving intravenous infusions of Replagal at a dose of 0.2 mg/kg of body weight every 2 weeks may be eligible for this study.
Participants will undergo the following tests and procedures:
Participants will go through a baseline evaluation, over a period of about 1 day. They will receive an intravenous infusion of Replagal every other week, at the dose of 0.2 mg/kg of body weight. Vital signs will be measured before the infusion and immediately and after and 1 hour afterward. There will be careful monitoring for allergic reactions and side effects. The infusion time takes approximately 40 minutes.
This study will last at least 1 year, or until the sponsor doing the investigating or the drug manufacturer decides to withdraw support of the study.
Objectives: This goal of this study is to continue treating two patients with Fabry disease using enzyme replacement therapy (ERT) using Replagal (agalsidase alfa) at a dose of 0.2 mg/kg of body weight administered every 2 weeks. Study Population: Two patients with Fabry disease who are currently on clinical research protocols 00-N-0185/TKT011 or 02-N-0220/TKT015 and who are stable on ERT. Design: This is an open label study. Outcome Measures: Mainly safety parameters will be obtained. Study duration is estimated to be 2 years.
242 studies on the registry are indexed under Fabry Disease; 54 are open to participants now.
This study's enrollment of 3 is below the median of 22 across 105 interventional studies indexed under Fabry Disease.
Browse Fabry Disease studies →National Institute of Neurological Disorders and Stroke (NINDS) is the lead sponsor of 592 studies on the registry; 56 are open to participants now.
Of its 18 completed or terminated interventional studies of FDA-regulated products, 8 (44%) have results posted.
Counted across the registry records on this site, refreshed daily.
Enzyme replacement for Fabry's Disease
Drug: Replagal agalsidase alfa · Drug: Replagal
Kidney function
Time frame: 3 years
This study is completed, as verified in Dec 2008. You cannot join it, but the record below documents what was studied.
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National Institute of Neurological Disorders and Stroke (NINDS)