Ignite Creation Date:
2024-05-05 @ 11:32 AM
Last Modification Date:
2024-10-26 @ 9:09 AM
Study NCT ID:
NCT00075244
Status:
COMPLETED
Last Update Posted:
2008-03-04
First Post:
2004-01-06
Brief Title:
Alternative Dosing and Regimen of Replagal to Treat Fabry Disease
Sponsor:
National Institute of Neurological Disorders and Stroke NINDS
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
A Phase I-II PharmacokineticPharmacodynamic Study of Replagal to Assess the Effects of Alternative Dose and Regimen in Patients With Fabry Disease
Status:
COMPLETED
Status Verified Date:
2005-11
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:
The main goal of this study is to assess the pharmacodynamic effects of different or more frequent doses of Replagal compared to the standard dosing regimen Replagal is a genetically engineered form of alpha-Galactosidase A an enzyme that normally breaks down a fatty substance called globotriaosylceramide Gb3 In patients with Fabry disease GB3 does not function properly and therefore builds up causing problems with the kidneys heart nerves and blood vessels
Male patients 18 years of age or older with Fabry disease who are not on dialysis and have not received a kidney transplant may be eligible for this study
Participants are randomly assigned to receive one of the following five regimens of Replagal infusions given through a vein over 20 to 80 minutes
01 mgkg body weight every week
02 mgkg body weight every week
02 mgkg body weight every other week
04 mgkg body weight every week
04 mgkg body wieght every other week
In the US the infusions are given at the NIH Clinical Center Vital signs are measured before immediately after and 1 hour after each infusion
Baseline evaluations are done on an inpatient or outpatient basis Baseline tests include a check of vital signs temperature respiratory rate pulse rate and blood pressure physical examination laboratory tests and review of treatment side effects Evaluations are also done at every infusion visit and 1 week and 1 month after the last infusion
Safety evaluations are done periodically and include vital sign measurements physical examination blood and urine tests review of drug side effects electrocardiogram ECG Holder monitor 2 hour ECG and QSART NIH only The QSART quantitative sudomotor axon reflex test measures the amount of sweat in a particular area of skin mostly the forearm For this test a cup partly filled with a liquid is strapped on the arm A weak electric current is turned on stimulating the sweat glands and the amount of sweat produced is measured There is a tingling sensation when the current is turned on
Patients who complete the study will be offered the opportunity of receiving Replagal for 6 months in an extension study
Male patients 18 years of age or older with Fabry disease who are not on dialysis and have not received a kidney transplant may be eligible for this study
Participants are randomly assigned to receive one of the following five regimens of Replagal infusions given through a vein over 20 to 80 minutes
01 mgkg body weight every week
02 mgkg body weight every week
02 mgkg body weight every other week
04 mgkg body weight every week
04 mgkg body wieght every other week
In the US the infusions are given at the NIH Clinical Center Vital signs are measured before immediately after and 1 hour after each infusion
Baseline evaluations are done on an inpatient or outpatient basis Baseline tests include a check of vital signs temperature respiratory rate pulse rate and blood pressure physical examination laboratory tests and review of treatment side effects Evaluations are also done at every infusion visit and 1 week and 1 month after the last infusion
Safety evaluations are done periodically and include vital sign measurements physical examination blood and urine tests review of drug side effects electrocardiogram ECG Holder monitor 2 hour ECG and QSART NIH only The QSART quantitative sudomotor axon reflex test measures the amount of sweat in a particular area of skin mostly the forearm For this test a cup partly filled with a liquid is strapped on the arm A weak electric current is turned on stimulating the sweat glands and the amount of sweat produced is measured There is a tingling sensation when the current is turned on
Patients who complete the study will be offered the opportunity of receiving Replagal for 6 months in an extension study
Detailed Description:
OBJECTIVES The goal of this study is to assess the pharmacodynamic effects of alternative weekly and every two week dosing regimens of Replagal agalsidase alfa in comparison to the current standard Replagal treatment regimen of 02 mgkg given intravenously every two weeks
STUDY POPULATION Hemizygous males with Fabry disease who are 18 years of age or older
DESIGN This is a randomized open-label study that will assess the pharmacodynamics and pharmacokinetics of five different dosing regimens of enzyme replacement therapy with Replagal The effects of dose as well as dosing frequency will be evaluated and compared to the standard Replagal regimen
OUTCOME MEASURES The pharmacodynamic parameter to be assessed is plasma globotriaosylceramide Gb3 The hypothesis to be tested is the role that frequency weekly andor dose 01 to 04 mgkg of Replagal will play in pharmacodynamics as measured by reductions in plasma Gb3 compared to the current dose and frequency of 02 mgkg given every two weeks Clinical parameters including sweating heart rate variability proteinuria severity of neuropathic pain pain and anti-diarrheal medication usage frequency and severity of abdominal pain and frequency of diarrhea also will be assessed
STUDY POPULATION Hemizygous males with Fabry disease who are 18 years of age or older
DESIGN This is a randomized open-label study that will assess the pharmacodynamics and pharmacokinetics of five different dosing regimens of enzyme replacement therapy with Replagal The effects of dose as well as dosing frequency will be evaluated and compared to the standard Replagal regimen
OUTCOME MEASURES The pharmacodynamic parameter to be assessed is plasma globotriaosylceramide Gb3 The hypothesis to be tested is the role that frequency weekly andor dose 01 to 04 mgkg of Replagal will play in pharmacodynamics as measured by reductions in plasma Gb3 compared to the current dose and frequency of 02 mgkg given every two weeks Clinical parameters including sweating heart rate variability proteinuria severity of neuropathic pain pain and anti-diarrheal medication usage frequency and severity of abdominal pain and frequency of diarrhea also will be assessed
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-0073 | None | None | None |