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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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19 February 2015 |
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Main ID: |
NCT00075244 |
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Date of registration:
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06/01/2004 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Alternative Dosing and Regimen of Replagal to Treat Fabry Disease
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Scientific title:
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A Phase I-II Pharmacokinetic/Pharmacodynamic Study of Replagal to Assess the Effects of Alternative Dose and Regimen in Patients With Fabry Disease |
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Date of first enrolment:
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January 2004 |
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Target sample size:
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25 |
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Recruitment status: |
Completed |
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URL:
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http://clinicaltrials.gov/show/NCT00075244 |
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Study type:
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Interventional |
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Study design:
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Endpoint Classification: Safety/Efficacy Study, Primary Purpose: Treatment
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Phase:
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Phase 2
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Countries of recruitment
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United States
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Key inclusion & exclusion criteria
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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.
Age minimum:
N/A
Age maximum:
N/A
Gender:
Male
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Health Condition(s) or Problem(s) studied
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Fabry Disease
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Intervention(s)
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Drug: Replagal
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Secondary ID(s)
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04-N-0073
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040073
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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