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.
Study Type
OBSERVATIONAL
Enrollment
116
Clinical Study Center
Auckland, New Zealand
Clinical Study Center
Christchurch, New Zealand
Clinical Study Center
London, United Kingdom
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.
Time frame: up to 15 years
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).
Time frame: up to 15 years
Percent and absolute change from baseline of target biomarkers over time after administration of a gene-editing therapy.
Blood samples will be collected to assess percent change from baseline in target biomarkers.
Time frame: up to 15 years
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