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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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20 March 2023 |
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Main ID: |
NCT04080050 |
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Date of registration:
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03/09/2019 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501
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Scientific title:
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A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501 |
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Date of first enrolment:
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September 30, 2019 |
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Target sample size:
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8 |
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Recruitment status: |
Active, not recruiting |
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URL:
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https://clinicaltrials.gov/show/NCT04080050 |
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Study type:
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Observational |
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Study design:
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Phase:
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Countries of recruitment
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Canada
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Italy
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Netherlands
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United States
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- To be eligible to participate in this study, a participant must have previously
received RGX-501 in a separate parent trial, and the participant or participant's
legal guardian(s) is/(are) willing and able to provide written, signed informed
consent after the nature of the study has been explained, prior to any
research-related procedures.
Exclusion Criteria:
- None
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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Homozygous Familial Hypercholesterolemia (HoFH)
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Intervention(s)
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Drug: Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
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Primary Outcome(s)
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Number of incidents of new and unexpected adverse events and serious adverse events.
[Time Frame: Up to 5 years after receiving treatment with RGX-501]
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Secondary Outcome(s)
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Absolute total cholesterol, LDL-C, very low density lipoprotein cholesterol (VLDL-C), high density lipoprotein cholesterol (HDL-C), calculated non-HDL-C, triglycerides (TG), and lipoprotein a (Lp(a)) over the study duration
[Time Frame: Up to 5 years after receiving treatment with RGX-501]
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Usage of lipid-lowering therapies over time
[Time Frame: Up to 5 years after receiving treatment with RGX-501]
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The absolute LDL-C level in mg/dL by beta quantification
[Time Frame: Year 3 after receiving treatment with RGX-501]
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Secondary ID(s)
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RGX-501-102
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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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