Ignite Creation Date:
2024-05-05 @ 11:26 AM
Last Modification Date:
2024-10-26 @ 9:08 AM
Study NCT ID:
NCT00048906
Status:
COMPLETED
Last Update Posted:
2008-03-04
First Post:
2002-11-08
Brief Title:
Alpha-Galactosidase A Replacement Therapy for Fabry Disease
Sponsor:
National Institute of Neurological Disorders and Stroke NINDS
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
A Safety and Pharmacokinetic Study of Replagal Enzyme Replacement Therapy in Patients With Fabry Disease
Status:
COMPLETED
Status Verified Date:
2003-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This study will determine the safety of the drug Replagal or treating patients 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 Previous studies have shown that patients with Fabry disease who had not progressed to end-stage kidney failure tolerated Replagal replacement therapy well This study will examine the effects of the drug in patients with kidney problems associated with Fabry disease
Patients with Fabry disease who are on kidney dialysis or have had a kidney transplant may be eligible for this study
During this 6 to 12-month study participants will receive a 40-minute intravenous IV infusion of Replagal every other week with close monitoring during and after the infusions Before the first infusion patients will be evaluated with a medical history physical and neurological examinations electrocardiogram ECG routine blood and urine tests kidney test and measurements of height weight and vital signs blood pressure pulse breathing rate temperature In addition they will have pharmacokinetic studies immediately before and following the first infusion of Replagal For these studies blood samples of less than a teaspoon each will be drawn to measure the level of Replagal enzyme activity The samples will be collected at 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
Safety evaluations will be done once a week for the first month and then once a month for the rest of the study period These evaluations include a physical examination measurement of vital signs electrocardiogram routine blood and urine tests and kidney testing
Patients with Fabry disease who are on kidney dialysis or have had a kidney transplant may be eligible for this study
During this 6 to 12-month study participants will receive a 40-minute intravenous IV infusion of Replagal every other week with close monitoring during and after the infusions Before the first infusion patients will be evaluated with a medical history physical and neurological examinations electrocardiogram ECG routine blood and urine tests kidney test and measurements of height weight and vital signs blood pressure pulse breathing rate temperature In addition they will have pharmacokinetic studies immediately before and following the first infusion of Replagal For these studies blood samples of less than a teaspoon each will be drawn to measure the level of Replagal enzyme activity The samples will be collected at 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
Safety evaluations will be done once a week for the first month and then once a month for the rest of the study period These evaluations include a physical examination measurement of vital signs electrocardiogram routine blood and urine tests and kidney testing
Detailed Description:
The objectives of this clinical trial are to study the effects of a range of renal function on the safety and pharmacokinetics of Replagal enzyme replacement therapy in patients with Fabry Disease Patients with clinical and genetic or biochemical evidence of Fabry Disease will be selected for this clinical trial Sixty 60 patients will be enrolled in this clinical trial Enrollment will be stratified based on baseline renal function as per FDA guidelines This is an open label clinical study Baseline evaluations will be conducted over an approximately 3 day period Patients will receive intravenous IV infusions of Replagal at a dose of 02 mgkgdose every 2 weeks Pharmacokinetic studies will be performed with the first administered dose The safety evaluations for patients in this study will include vital signs physical examinations adverse event AE assessments electrocardiograms ECG and a battery of laboratory tests including measurement of anti-Replagal antibodies An overview of the study appears in Appendix A
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 03-N-0043 | None | None | None |