A Phase 1/2 Ascending Dose Study to Evaluate the Safety and Effects on Progranulin Levels of LY3884963 in Patients with Fronto-Temporal Dementia with Progranulin Mutations (FTD-GRN) (PROCLAIM)

The purpose of this open label study is to assess the safety, tolerability, and effects of LY3884963 on plasma (the liquid component of blood) and/or cerebrospinal fluid (CSF; fluid from the area surrounding the brain) PGRN levels, in order to inform further clinical investigation and/or allow marketing registration of LY3884963 in a patient population suffering from FTD-GRN. The Sponsor is testing to determine whether LY3884963 will increase PGRN levels and result in modification of the disease process and will slow or halt progression.
LY3884963 is an investigational gene therapy product, which means Health Authorities have not approved it for the treatment of FTD-GRN or any other indication. LY3884963 has not been tested previously in humans. It has been tested in animal experiments (mice and non-human primates), where no related adverse events were observed at the dose that will be used in this clinical study.