Ignite Creation Date:
2024-05-05 @ 11:39 AM
Last Modification Date:
2024-10-26 @ 9:11 AM
Study NCT ID:
NCT00097890
Status:
COMPLETED
Last Update Posted:
2008-03-04
First Post:
2004-11-30
Brief Title:
Replagal Enzyme Replacement Therapy for Adults With Fabry Disease
Sponsor:
National Institute of Neurological Disorders and Stroke NINDS
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
An Open Label Six-Month Maintenance Clinical Trial of Replagal Enzyme Replacement Therapy in Patients With Fabry Disease Who Have Completed TKT027
Status:
COMPLETED
Status Verified Date:
2005-12
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 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
Intravenous IV infusions of Replagal every other week over 25 weeks for a total of 13 infusions with close monitoring during and after the infusions
Brief safety evaluations at the time of each infusion including a check of vital signs blood pressure pulse breathing rate temperature review of any side effects and review of medications
Comprehensive evaluations at baseline before starting Replagal therapy after 13 and 25 weeks of therapy and 30 days after completing therapy These include a medical history and physical examination symptoms and pain questionnaire blood and urine tests check of vital signs electrocardiogram EKG 2-hour Holter monitor and sweat test QSART
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
Intravenous IV infusions of Replagal every other week over 25 weeks for a total of 13 infusions with close monitoring during and after the infusions
Brief safety evaluations at the time of each infusion including a check of vital signs blood pressure pulse breathing rate temperature review of any side effects and review of medications
Comprehensive evaluations at baseline before starting Replagal therapy after 13 and 25 weeks of therapy and 30 days after completing therapy These include a medical history and physical examination symptoms and pain questionnaire blood and urine tests check of vital signs electrocardiogram EKG 2-hour Holter monitor and sweat test QSART
Detailed Description:
Objectives To evaluate the safety of the current standard Replagal treatment regimen of 02 mgkg 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 02 mgkg every two weeks of enzyme replacement therapy with Replagal
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 02 mgkg every two weeks of enzyme replacement therapy with Replagal
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 |
|---|---|---|---|
| 05-N-0046 | None | None | None |