Ignite Creation Date:
2024-05-05 @ 11:32 AM
Last Modification Date:
2024-10-26 @ 9:09 AM
Study NCT ID:
NCT00071877
Status:
COMPLETED
Last Update Posted:
2008-03-04
First Post:
2003-11-03
Brief Title:
An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease
Sponsor:
National Institute of Neurological Disorders and Stroke NINDS
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
A Clinical Trial of Replagal Enzyme Replacement Therapy in Children Ages 7 - 17 Years With Fabry Disease
Status:
COMPLETED
Status Verified Date:
2005-06
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 evaluate the safety of multiple biweekly intravenous doses of Replagal over 26 weeks in 25 children with Fabry disease and the way in which that agent can improve the health of this patient population Fabry disease is a genetic disorder inherited as an X-linked recessive trait It causes a deficiency in the enzyme alpha galactosidase which normally breaks down a lipid or fatty substance called ceramidetrihexosidase a building block in all cells of the body
The deficiency in breaking down the lipid eventually causes that lipid to accumulate and injure cells Problems in the blood vessels kidneys heart and nerves are the result The disease typically occurs in childhood or adolescence with repeated episodes of severe pain in the extremities and other symptoms There is no definitive treatment but pain management is important in caring for patients with Fabry disease Although it is not known exactly how lipid accumulation brings about such problems studies of another lipid storage disorder Gauchers disease have shown that the illness can be reversed if the lipid is removed when an appropriate enzyme Replagal is given intravenously In this study the gene response of the bodys cells to Fabry disease will be described as will any gene responses that change when the enzyme is used
Patients 7 to 17 years of age who have Fabry disease may be eligible for this study They will undergo the following tests and procedures
Physical examination
Neurological examination
Vital signs
Urinalysis
Blood tests to determine complete blood count and chemistries
Questionnaire on pain
Tests pertaining to sweating
Electrocardiogram
Doppler blood flow study
Diary for recording symptoms and the use of pain medications
Participants will go through the evaluation over a period of about 5 days either as an inpatient or outpatient Participants will receive an intravenous infusion of Replagal every other week at the dose of 02 mgkg of body weight Vital signs will be measured before the infusion and immediately and after and 1 hour afterward There will be careful monitoring for allergic reactions and side effects The infusion time takes approximately 40 minutes
This study will last 6 months with the possibility of being extended another 6 months-a maintenance study in which patients will continue to receive Replagal at the same dose every 2 weeks
The deficiency in breaking down the lipid eventually causes that lipid to accumulate and injure cells Problems in the blood vessels kidneys heart and nerves are the result The disease typically occurs in childhood or adolescence with repeated episodes of severe pain in the extremities and other symptoms There is no definitive treatment but pain management is important in caring for patients with Fabry disease Although it is not known exactly how lipid accumulation brings about such problems studies of another lipid storage disorder Gauchers disease have shown that the illness can be reversed if the lipid is removed when an appropriate enzyme Replagal is given intravenously In this study the gene response of the bodys cells to Fabry disease will be described as will any gene responses that change when the enzyme is used
Patients 7 to 17 years of age who have Fabry disease may be eligible for this study They will undergo the following tests and procedures
Physical examination
Neurological examination
Vital signs
Urinalysis
Blood tests to determine complete blood count and chemistries
Questionnaire on pain
Tests pertaining to sweating
Electrocardiogram
Doppler blood flow study
Diary for recording symptoms and the use of pain medications
Participants will go through the evaluation over a period of about 5 days either as an inpatient or outpatient Participants will receive an intravenous infusion of Replagal every other week at the dose of 02 mgkg of body weight Vital signs will be measured before the infusion and immediately and after and 1 hour afterward There will be careful monitoring for allergic reactions and side effects The infusion time takes approximately 40 minutes
This study will last 6 months with the possibility of being extended another 6 months-a maintenance study in which patients will continue to receive Replagal at the same dose every 2 weeks
Detailed Description:
The objectives of this clinical trial are to evaluate 1 the safety of multiple biweekly ie every other week intravenous IV doses of Replagal over 26 weeks in 25 children ages 7-17 years old with Fabry Disease and 2 the pharmacokinetics of Replagal in this patient population Safety will be determined by standard clinical and laboratory measurements
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 |
|---|---|---|---|
| 04-N-0029 | None | None | None |