Status:
COMPLETED
Alternative Dosing and Regimen of Replagal to Treat Fabry Disease
Lead Sponsor:
National Institute of Neurological Disorders and Stroke (NINDS)
Conditions:
Fabry Disease
Eligibility:
MALE
Phase:
PHASE2
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 ...
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...
Eligibility Criteria
Inclusion
- INCLUSION CRITERIA:
- Subject is a male hemizygote, age 18 years or older, with confirmed diagnosis of Fabry Disease. Diagnosis of Fabry disease may be confirmed by proof of a mutation of the alpha-Galactosidase A gene compatible with Fabry Disease and/or a deficiency of alpha-Galactosidase A (less than 4.0 nmol/mL/hour in plasma or serum or less than 8% of average mean normal in leukocytes).
- Subject must have one or more clinical manifestations of Fabry disease including neuropathic pain, angiokeratoma, corneal verticillata, cardiomyopathy, hypo- or anhydrosis, abdominal pain and/or diarrhea, serum creatinine greater than 1.0 mg/dl or proteinuria greater than 300 mg/24 hours.
- Subject must have voluntarily signed an Institutional Review Board (IRB) approved informed consent form after all relevant aspects of the study have been explained and discussed with the subject.
- EXCLUSION CRITERIA:
- Subject has been previously treated with Replagal or any other enzyme replacement therapy for Fabry Disease. If the patient has previously been treated with Replagal or another enzyme replacement therapy then they must have been off the therapy for at least 30 days and must have a Day-14 antibody blood sample drawn and that test must be negative for anti-agalsidase alfa IgG and IgE antibodies and not experienced a prior severe infusion reactions with prior enzyme replacement therapy.
- Subject has been enrolled in another clinical investigative study in the past 30 days.
- Subject is unable to give informed consent or is deemed unable to comply with all aspects of the clinical trial.
- Subject has plasma Gb(3) drawn on Day -14 less than 4.0 nmol/mL.
- Subject is undergoing dialysis or who has received a renal transplant.
- Subjects who cannot tolerate the study procedures or who are unable or unwilling to travel to the study center as required by this protocol.
- Subjects with an inter-current medical condition that would render them unsuitable for the study (e.g. HIV, diabetes) by confounding an assessment of the effects of the experimental therapy and its adverse events.
- Subjects who in the opinion of the investigator (for whatever reason) are thought to be unsuitable for the study.
Exclusion
Key Trial Info
Start Date :
January 1 2004
Trial Type :
INTERVENTIONAL
End Date :
November 1 2005
Estimated Enrollment :
25 Patients enrolled
Trial Details
Trial ID
NCT00075244
Start Date
January 1 2004
End Date
November 1 2005
Last Update
March 4 2008
Active Locations (1)
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1
National Institute of Neurological Disorders and Stroke (NINDS)
Bethesda, Maryland, United States, 20892