Overviewing First-Ever Platform Trial for Progressive Supranuclear Palsy: Julio Rojas, MD, PhD

WATCH TIME: 4 minutes

Progressive supranuclear palsy (PSP) is a rare, progressive neurodegenerative disorder characterized by parkinsonism alongside features that can include early falls, oculomotor abnormalities, cognitive impairment, and behavioral changes. Epidemiologic estimates vary, although a recent systematic review and meta-analysis calculated a pooled prevalence of approximately 6.9 cases per 100,000 individuals.¹ PSP is also a primary tauopathy, making abnormal tau biology an important target in ongoing efforts to develop disease-modifying treatments.

Despite growing knowledge of PSP biology, translating potential therapeutic targets into effective treatments remains difficult. Traditional randomized trials generally evaluate individual therapies sequentially, an especially challenging model when several potential treatments need to be studied in a relatively small patient population. Platform trials offer an alternative by allowing multiple investigational therapies to be evaluated under a common infrastructure, an approach already introduced into neurodegenerative disease research through the HEALEY ALS Platform Trial.²

Julio C. Rojas, MD, PhD, is a neurologist at the UCSF Edward and Pearl Fein Memory and Aging Center whose clinical and research work encompasses PSP and other neurodegenerative disorders. Rojas is an investigator in the newly launched Progressive Supranuclear Palsy Clinical Trial Platform, a multicenter, multi-regimen study designed to evaluate investigational therapies simultaneously or sequentially under a master protocol.³

In an interview with NeurologyLive^®, Rojas discussed how the first platform trial dedicated to PSP came together and why the model may be particularly valuable for a rare neurodegenerative disease. He also addressed lessons from oncology and ALS research, the growing number of biologically informed therapeutic targets in PSP, and how a more efficient trial infrastructure could reshape opportunities for patients and investigators.

REFERENCES
1. Coyle-Gilchrist ITS, Dick KM, Patterson K, et al. The prevalence and incidence of progressive supranuclear palsy and corticobasal syndrome: a systematic review and meta-analysis. J Neurol. 2023.
2. Paganoni S, Berry JD, Quintana M, et al. Adaptive platform trials to transform amyotrophic lateral sclerosis therapy development. Ann Neurol. 2022;91(2):165-175. doi:10.1002/ana.26285.
3. University of California, San Francisco. The Progressive Supranuclear Palsy Clinical Trial Platform. ClinicalTrials.gov Identifier: NCT07173803. Study initiated July 2026.