NCT04408625
Prevail Therapeutics, Inc. / A Phase 1/2 Ascending Dose Study to Evaluate the Safety and Effects on Progranulin Levels of PR006A in Patients with Fronto-Temporal Dementia with Progranulin Mutations
Fronto-temporal dementia or frontotemporal lobar degeneration refers to a group of disorders caused by progressive cell loss in the brain's frontal lobes or its temporal lobes. These areas of the brain play a significant role in decision-making, behavioral control, emotion, and language. FTD can be inherited, with genetic forms making up to 20% of all FTD cases. Mutations in the progranulin gene are a common genetic cause of FTD.
This study is currently enrolling.