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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 October 2017 |
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Main ID: |
NCT00068107 |
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Date of registration:
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06/09/2003 |
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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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Dosing Study of Replagal in Patients With Fabry Disease
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Scientific title:
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Study of Weekly Dosing Regimens of Replagal in Patients With Fabry Disease With Incomplete Clinical Response to Long-Term Therapy |
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Date of first enrolment:
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September 2003 |
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Target sample size:
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13 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT00068107 |
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Study type:
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Interventional |
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Study design:
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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:
Patients with Fabry disease participating in 00-N-0185/TKT011 or 02-N-0220/TKT015 may be
eligible. No other Fabry patients will be eligible.
Patients losing GFR at a rate greater than 5 ml/min/year despite ERT with agalsidase alfa
for greater than or equal to 2.5 years in 00-N-0185/TKT/003/006/011 Study or ERT over
greater than or equal to 1.0 years in 02-N-0220/TKT/010/015 Study.
Patients who at least twice demonstrated significant improvement or normalization of sweat
function (by QSART or thermoregulatory sweat test) or reduction in serum creatinine by at
least 10% but return to the pre-infusion state before the subsequent biweekly enzyme
infusion.
Patients who freely agree to participate in this study and understand the nature, risks and
benefits of this study and give their written informed consent.
EXCLUSION CRITERIA:
Patients with Fabry disease, who are not already part of 00-N-0185/TKT011 or
02-N-0220/TKT015.
Patients on these protocols who have stable serum creatinine (or a lesser rise in serum
creatinine than stipulated in the inclusion criteria), and do not show other objective
evidence of incomplete clinical response between biweekly infusions (e.g. sweat function).
Patients who have begun dialysis or who have received a renal transplant.
Patients who cannot tolerate the study procedures or who are unable or unwilling to travel
to the study center as required by this protocol.
Patients with an intercurrent medical condition that would render them unsuitable for mthe
study e.g. HIV, diabetes. The reason is that the pathologies of these conditions will be
significant confounders in assessing the effect of the experimental therapy and its adverse
events.
Patients 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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Primary Outcome(s)
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Estimated Glomerular Filtration Rate (eGFR)
[Time Frame: Relagal was administered every 2 weeks for 2-4 years pre-study, Relagal was administred weekly during the study (approx. 4.5-10 years)]
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Secondary Outcome(s)
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Globotriaosylceramide (Gb(3)) in Plasma
[Time Frame: Baseline and last observation (up to 10 years)]
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Quantitative Sensory Testing
[Time Frame: pre-study was 2-4 years, during study sensory testing measured for approx. 4.5-5 years]
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Number of Participants With a Change in Quantitative Sudomotor Axon Reflex Test
[Time Frame: Baseline and last observation (up to 10 years)]
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Globotriaosylceramide (Gb(3)) in Urine Sediment
[Time Frame: Baseline and last observation (up to 10 years)]
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Doppler Skin Blood Flow
[Time Frame: 10 years]
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Secondary ID(s)
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030286
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03-N-0286
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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