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Sponsored by: |
National Institute of Neurological Disorders and Stroke (NINDS) |
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Information provided by: | National Institutes of Health Clinical Center (CC) |
ClinicalTrials.gov Identifier: | NCT00097890 |
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:
Condition | Intervention | Phase |
---|---|---|
Fabry Disease |
Drug: Replagal (Agalsidase Alfa) Drug: Replagal |
Phase IV |
Study Type: | Interventional |
Study Design: | Treatment |
Official Title: | An Open Label Six-Month Maintenance Clinical Trial of Replagal Enzyme Replacement Therapy in Patients With Fabry Disease Who Have Completed TKT027 |
Estimated Enrollment: | 25 |
Study Start Date: | November 2004 |
Estimated Study Completion Date: | December 2005 |
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.
Genders Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
INCLUSION CRITERIA:
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.
Study ID Numbers: | 050046, 05-N-0046 |
Study First Received: | November 30, 2004 |
Last Updated: | March 3, 2008 |
ClinicalTrials.gov Identifier: | NCT00097890 |
Health Authority: | United States: Federal Government |
Lysosomes Storage Glycolipid |
Fabry Disease Stroke Fabry Disease |
Lipid Metabolism, Inborn Errors Sphingolipidoses Metabolic Diseases Cerebral Infarction Lysosomal Storage Diseases Stroke Fabry disease Sphingolipidosis Central Nervous System Diseases Brain Diseases |
Metabolism, Inborn Errors Ceramide trihexosidosis Genetic Diseases, Inborn Fabry Disease Genetic Diseases, X-Linked Brain Diseases, Metabolic, Inborn Lipidoses Metabolic disorder Lipid Metabolism Disorders Brain Diseases, Metabolic |
Lysosomal Storage Diseases, Nervous System Nervous System Diseases |