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Replagal Enzyme Replacement Therapy for Children with Fabry Disease
This study is currently recruiting patients.
Sponsored by: | National Institute of Neurological Disorders and Stroke (NINDS) |
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Information provided by: | Warren G Magnuson Clinical Center (CC) |
Purpose
This study will determine the safety and effectiveness of the drug Replagal(registered trademark) for treating people with Fabry disease, an inherited metabolic disorder. In this disease, an enzyme called alpha-galactosidase A, which normally breaks down a fatty substance called globotriaosylceramide (Gb3), is missing or does not function properly. The resulting accumulation of Gb3 causes problems with the kidneys, heart, nerves, and blood vessels. Replagal is a genetically engineered form of alpha-galactosidase A.
Patients with Fabry disease who have completed NIH protocol TKT023 may be eligible for this 7-month study extension.
Participants will receive 40-minute intravenous (IV) infusions of Replagal every other week over 29 weeks, with close monitoring during and after the infusions. The effects of the medication will be measured at various time points during the study with the following evaluations: general physical and neurological examinations; quality of life questionnaires; ultrasound blood flow tests; tests of heart function, including electrocardiogram, echocardiogram, and 24-hour Holter monitoring; blood and urine tests; tests for antibodies against Replagal; and measurements of height, weight, and vital signs (blood pressure, pulse, breathing rate, temperature).
Patients who have developed antibodies to Replagal will have blood samples drawn during the final drug infusion to determine how rapidly the drug is removed from the blood stream. For these pharmacokinetic studies, less than a teaspoon of blood will be collected at each of the following time points: immediately before the infusion; 20 minutes into the infusion; at the end of the infusion; after the infusion at 50, 60, and 90 minutes, and 2, 3, 4, and 8 hours.
Thirty days after the last Replagal infusion, patients (or their parents) provide additional information about their health since the last visit.
Condition | Treatment or Intervention | Phase |
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Fabry Disease |
Drug: Replagal (Galactosidase Alfa) |
Phase II |
MedlinePlus related topics: Genetic Brain Disorders; Genetic Disorders; Metabolic Disorders
Genetics Home Reference related topics: Fabry disease
Study Type: Interventional
Study Design: Treatment, Safety/Efficacy
Official Title: An Open Label Clinical Trial of Replagal Enzyme Replacement Therapy In Children With Fabry Disease Who Have Completed Study TKT023
Expected Total Enrollment: 19
Study start: June 3, 2004
The objectives of this clinical trial are to evaluate: 1) the safety of multiple biweekly (i.e. every other week) intravenous (IV) doses of Replagal over an additional 29 weeks, for patients who have completed 6 months of Replagal therapy in TKT023 in the US and Canada, 2) the pharmacokinetics of Replagal in the subset of this patient population which develops IgG antibodies to agalsidase alfa, and 3) the exploration of efficacy of Replagal in terms of sweating, proteinuria, heart rate variability, clinical outcomes, renal function, and left ventricular (LV) mass. Safety will be determined by standard clinical and laboratory measurements.
Eligibility
Genders Eligible for Study: Both
Criteria
Location and Contact Information
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