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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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12 December 2020 |
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Main ID: |
NCT04002531 |
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Date of registration:
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25/03/2019 |
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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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A One Visit Follow Up of Adults With Fabry Disease Who Started Long-term Enzyme Replacement Therapy As Children
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Scientific title:
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A One Visit Follow Up of Adults With Fabry Disease Who Started Long-term Enzyme Replacement Therapy As Children |
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Date of first enrolment:
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November 10, 2018 |
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Target sample size:
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12 |
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Recruitment status: |
Enrolling by invitation |
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URL:
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https://clinicaltrials.gov/show/NCT04002531 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Other. Masking: None (Open Label).
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Phase:
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N/A
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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:
1. Patients who participated in TKT029 and who are willing and able to come to Dallas for
1 visit for standard of care testing.
2. Sign the protocol informed consent form
3. Have been on continuous commercial ERT since TKT029 has ended
Exclusion Criteria:
1. Patients who are unable to understand the nature, scope, and possible consequences of
the study.
2. Patient does not give his written informed consent to participate in this study
3. Patient is unable to comply with the protocol, e.g., uncooperative with protocol
schedule, refusal to agree to all of the study procedures.
4. Patient has been off ERT for an extended period of time as assessed by the
investigator.
Age minimum:
18 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Cardiac Event
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Renal Insufficiency
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Quality of Life
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Intervention(s)
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Procedure: Urine collection
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Other: Brief Pain Inventory questionnaire
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Other: Quality of Life questionnaire
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Procedure: Echocardiogram
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Other: General and Neurological examination
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Other: Vital signs
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Procedure: 2-hour Holter Monitor
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Procedure: Blood draw
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Procedure: 12 lead electrocardiogram
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Primary Outcome(s)
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estimated Glomerular Filtration Rate (eGFR)
[Time Frame: Study involves one visit only - assessed Baseline Visit]
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Secondary Outcome(s)
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Qualify of Life - Your Health and Well-being
[Time Frame: Study involves one visit only - assessed Baseline Visit]
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Urine albumin/creatinine ratio
[Time Frame: Study involves one visit only - assessed Baseline Visit]
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Urine Lyso-Gb3
[Time Frame: Study involves one visit only - assessed Baseline Visit]
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Left Ventricular Mass Index
[Time Frame: Study involves one visit only - assessed Baseline Visit]
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Heart rate variability assessment
[Time Frame: Study involves one visit only - assessed Baseline Visit]
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Short-form Brief Pain Inventory (BPI)
[Time Frame: Study involves one visit only - assessed Baseline Visit]
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Plasma Lyso-Gb3
[Time Frame: Study involves one visit only - assessed Baseline Visit]
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Plasma Gb3 and compared to plasma Gb3 results obtained during participation in study "Replagal Enzyme Replacement Therapy for Children With Fabry Disease" NCT00084084
[Time Frame: Study involves one visit only - assessed Baseline Visit]
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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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