The Progressive Supranuclear Palsy (PSP) Trial Platform (PTP) is soliciting interest from industry partners in collaborating on drug development for PSP. PSP is a severe neurodegenerative disease that usually leads to death within 5-7 years of diagnosis. There are no effective treatments for PSP, but therapeutic approaches being tested in clinical trials for other neurodegenerative diseases, such as Alzheimer's disease (AD) and Frontotemporal Degeneration (FTD) may be even more promising for PSP. Despite excellent feasibility of large multicenter PSP clinical trials, there are few ongoing clinical 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. 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.

The PTP is an NIH-funded Phase 2, multi-center, multi-drug, multi-arm clinical trial evaluating the safety, tolerability, target engagement, and preliminary efficacy in mild-to-moderate PSP. Therapies are being compared for 12 months, with a 3:1 (drug:placebo) randomization ratio to encourage recruitment, followed by an optional, 12-month open-label extension. The platform trial is currently enrolling 440 participants (110 participants per arm) across 50 sites in North America and is powered to detect a 30-35% difference in the rate of change in the modified 15-item PSPRS over one year and is supported by biomarker outcomes such as neurofilament light chain (NfL) and volumetric MRI biomarkers including midbrain volume. The PTP Study is designed to be a perpetual platform trial that will add additional study arms over time. We are currently looking for both industry and academic partners who are looking to work with us to accelerate drug development for this rare and devastating disease.

Collaboration is the key to our success. We believe sharing knowledge and expertise is essential to rapidly bringing innovative therapies to patients. That's why we are partnering with leading industry and academic institutions to launch the first platform trial for PSP and share information with our peers to accelerate therapeutic innovation. The PTP Study 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.

Applications will be reviewed on a bi-annual basis by the Compound Selection Committee (which includes basic science experts, clinicians, and a patient representative) in Q1 and Q3 of each year. The deadline to apply for each Compound Selection Committee is the third Friday of May and November of each year, respectively.

If your team is interested in joining our platform trial, please email psptrialplatform@ucsf.edu to initiate the set-up of a mutual CDA with UCSF.

Current Partners:

Contact Information for Researchers and Industry: To collaborate with us on new treatments or trial sites, please email psptrialplatform@ucsf.edu.