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Project Number: 1R01AG085029-01  

Contact PI/Project Leader: Adam Boxer, Irene Litvan, Julio Cesar Rojas-Martinez, Anne-Marie Wills

Eligible Participants: Key inclusion criteria will be a diagnosis of mild-moderate PSP, with symptoms < 5 years in duration, preserved ability to ambulate with minimal cognitive impairment.

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Progressive Supranuclear Palsy (PSP) is a severe neurodegenerative disease of aging that usually leads to death within 5-7 years of diagnosis. PSP is believed to be caused by toxic tau protein accumulation in the brain.  There are no effective treatments for PSP, but therapeutic approaches being tested in clinical trials for other neurodegenerative diseases, such as anti-tau therapies for Alzheimer’s (AD) and neuroprotective approaches for amyotrophic lateral sclerosis, may be even more promising for PSP. Despite excellent feasibility of large multicenter PSP clinical trials, there are few new studies, limiting options for patients to access experimental therapies and severely delaying the identification of effective treatments.

New, efficient clinical PSP trial programs and focused efforts to identify PSP biomarkers are urgently needed. The overarching goal of the PSP Trial Platform (PTP) is to conduct a randomized, placebo-controlled, Phase 2 platform trial in mild-moderate PSP that will simultaneously test at least three different tau-related or neuroprotective therapies to determine safety, tolerability and clinical proof of concept based on a multimodal clinical rating scale, the modified PSP Rating Scale-15 (mPSPRS-15). Platform trials create economies of scale through generation of a common clinical trial protocol and the ability to share placebo group information to allow a greater number of therapies to be tested in a shorter amount of time and with less expense than multiple independent clinical trials.  Three therapies will be compared for 12 months, with a 3:1 (drug:placebo) randomization ratio to encourage recruitment, followed by an optional, 12-month open-label extension.

If successful, the PTP will provide key data for decision-making about which therapies to pursue in larger efficacy trials, create a new infrastructure to efficiently evaluate PSP therapies, and a new resource for longitudinal PSP clinical and biomarker data, and biosamples, to be shared with other researchers.