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LTF-001 is a long-term follow-up study of participants who received an investigational gene-editing therapy developed by the sponsor to evaluate the long-term effects of the investigational therapy. Participants will be followed for a total of 15 years after the first administration of the gene-editing therapy.
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| Measure | Description | Time Frame |
|---|---|---|
| Incidence of treatment-related adverse events (AEs), serious adverse events (SAEs) and other events of interest using CTCAE 5.0 to assess the long-term safety of gene-editing therapies. | To assess the long-term safety of gene-editing therapies, the following will be assessed: Incidence of treatment-related adverse events (AEs), serious adverse events (SAEs) and any CTCAE grade 3 or higher AEs. | up to 15 years |
| Measure | Description | Time Frame |
|---|---|---|
| Percent and absolute change from baseline of lipid concentrations over time after administration of a gene-editing therapy. | Blood samples will be collected to assess percent change from baseline in lipid concentrations (including LDL-C). | up to 15 years |
| Percent and absolute change from baseline of target biomarkers over time after administration of a gene-editing therapy. |
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Inclusion Criteria
Exclusion Criteria: N/A
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Participants who received an investigational gene-editing therapy developed by the sponsor.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Study Center | Auckland | New Zealand | ||||
| Clinical Study Center |
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Blood samples will be collected to assess percent change from baseline in target biomarkers. |
| up to 15 years |
| Christchurch |
| New Zealand |
| Clinical Study Center | London | United Kingdom |
| ID | Term |
|---|---|
| D050197 | Atherosclerosis |
| D006937 | Hypercholesterolemia |
| D002318 | Cardiovascular Diseases |
| D006938 | Hyperlipoproteinemia Type II |
| ID | Term |
|---|---|
| D001161 | Arteriosclerosis |
| D001157 | Arterial Occlusive Diseases |
| D014652 | Vascular Diseases |
| D006949 | Hyperlipidemias |
| D050171 | Dyslipidemias |
| D052439 | Lipid Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D008052 | Lipid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D006951 | Hyperlipoproteinemias |
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