A Phase 4 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. Per ClinicalTrials.gov, last updated 2008-03-04.
Sponsored by National Institute of Neurological Disorders and Stroke (NINDS) · Phase 4, Interventional, and Treatment
This study will determine the safety and effectiveness of the drug Replagal for treating people with Fabry disease, an inherited metabolic disorder. In this disease, an enzyme called alpha-galactosidase A, which normally breaks down a lipid (fatty substance) known as ceramidetrihexoside, is missing or does not function properly. As a result, the lipid accumulates in the body, causing problems with the kidneys, heart, nerves, and blood vessels. This study will examine whether replacing the missing alpha-galactosidase A with a genetically engineered form of the enzyme called Replagal can reverse the illness.
Patients with Fabry disease who are 18 years of age or older and have completed 10 weeks of Replagal therapy as participants in protocol TKT027 may be eligible for this 6-month study extension.
Participants undergo the following tests and procedures:
Objectives: To evaluate the safety of the current standard Replagal treatment regimen of 0.2 mg/kg given intravenously every two weeks over an additional 6 months in patients who have completed 10 weeks of Replagal therapy in the TKT027 study.
Study Population: Hemizygous males with Fabry disease who are 18 years of age or older who have completed 10 weeks of Replagal therapy in TKT027.
Design: This is an open-label, multi-center study that will assess the safety of 0.2 mg/kg every two weeks of enzyme replacement therapy with Replagal.
242 studies on the registry are indexed under Fabry Disease; 54 are open to participants now.
This study's enrollment of 25 is above 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.
Subject must have completed 10 weeks of infusions in Study TKT027 with intent to complete participation in the study.
Subject must have adequate general health (as determined by the investigators) to undergo the specified phlebotomy regimen and protocol related procedures.
Subject must consent to participate in the protocol and must have voluntarily signed an Institutional Review Board/Independent Ethics Committee (IRB/IEC) approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient.
EXCLUSION CRITERIA:
Subject is unable to understand the nature, scope, and possible consequences of the study.
Subject is unable to comply with the protocol, e.g., uncooperative with protocol schedule, refusal to agree to all of the study procedures, inability to return for safety evaluations, or is otherwise unlikely to complete the study, as determined by the investigator or the medical monitor.
This study is completed, as verified in Dec 2005. You cannot join it, but the record below documents what was studied.
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National Institute of Neurological Disorders and Stroke (NINDS)