Lead Investigator: Adam Boxer, UCSF
Title of Proposal Research: Bayesian Disease Progression Modeling and Clinical Trial Design for Progressive Supranuclear Palsy – Richardson’s Syndrome
Vivli Data Request: 7621
Funding Source: Rainwater Charitable Foundation; Association for Frontotemporal Degeneration
Potential Conflicts of Interest: Barbara Wendelberger and Melanie Quintana are statisticians at Berry Statistical Consultants. They will maintain confidentiality around this project and these data will only be used for the analyses proposed here.
Dr. Boxer was a consultant for Abbvie in the development of the current clinical trial dataset. He currently consults for several other companies involved in PSP research including Roche and Transposon, and receives research support from Biogen and Regeneron.
Summary of the Proposed Research:
Progressive Supranuclear Palsy (PSP) is a rare primary tauopathy, e.g. a disorder caused by the deposit of abnormal tau protein in the brain (tau is a protein that stabilises neuron cells in the nervous system), neurodegenerative disease that usually affects people in their 50s, 60s and 70s, and is generally fatal within 5-7 years of diagnosis. It is a progressively worsening condition of the nervous system that causes problems with balance, vision, speech, movement and swallowing. Approximately 200,000 US individuals are estimated to currently have PSP or a related disorder. There are no effective treatments for PSP. We have previously led the design and conduct of large, multicenter clinical trials for PSP. Although none of the therapies that we tested showed any benefit, we have improved our methods of designing clinical trials. To find an effective therapy we need to test more therapies. Clinical trials are expensive and difficult to complete. Multi-arm platform trials are clinical trials that allow the testing of multiple therapies in the same clinical trial, which offers many economies of scale, and reduces costs and time to test new therapies. The goal of this project is to use recent clinical trial data to model disease progression in PSP as a basis for the design of a multi-arm platform trial. This design will be used to apply for funding from the National Institutes of Health (NIH) and hopefully actually initiate the trial.
A Randomized, Double-Blind, Placebo-Controlled Multiple Dose Study to Assess Efficacy, Safety, Tolerability, and Pharmacokinetics of ABBV-8E12 in Progressive Supranuclear Palsy
Data Contributor: AbbVie
Study ID: NCT02985879
Sponsor ID: M15-562
An Extension Study of ABBV-8E12 in Progressive Supranuclear Palsy (PSP)
Data Contributor: AbbVie
Study ID: NCT03391765
Sponsor ID: M15-563