This is a Phase 2a multi-center, randomized, double-blind, placebo-controlled parallel-group, 4-arm study with an open-label treatment phase in patients with progressive supranuclear palsy (PSP). This study includes a 6-week screening period, a 24-week double-blind treatment period, a 24-week open label treatment period, and a follow-up visit four weeks post treatment.
The main purpose of this study is to investigate the safety and tolerability of TPN-101 compared to a placebo for the treatment of PSP. The study will also evaluate whether TPN-101 affects the symptoms of patients with PSP. Transposon (sponsor) is developing TPN-101 for the treatment of neurodegenerative diseases, including PSP. Nonclinical pharmacology studies demonstrate that TPN-101 inhibits hL1 RT-dependent retrotransposition in cultured cells with a potency directly comparable to its antiviral half maximal effective concentration (EC50) for HIV-1. The attendant effects of hL1 ORF2 inhibition are anticipated to be a reduction in the cDNA burden in affected cells of neuronal and glial origin in which overexpression of hL1 is occurring.
Who Can Participate
Males and females between the ages of 41-86 years old; with presence of PSP symptoms for less than 5 years, with at least a 12 month history of postural instability OR at least a 12-month history of falls during the first 3 years that symptoms are present.
Study participants will have 13 visits within 52 weeks.