The University of California, San Francisco (UCSF) is set to launch a pioneering clinical trial that will simultaneously evaluate multiple drug candidates for progressive supranuclear palsy (PSP), supported by a substantial five-year grant of up to $75.4 million from the National Institute on Aging.
Novel Platform Trial Design
The study employs an innovative platform trial design, allowing researchers to test multiple treatments concurrently against a single control group. "Unlike typical clinical trials, platform trials can remain open with multiple new therapies tested in successive cycles if the first ones don't work," explains Dr. Adam Boxer, MD, PhD, a principal investigator of the trial. "This means there are more opportunities to identify effective treatments in a faster timeframe, with lower cost and less burden to participants."
Participants will have a 75% chance of receiving an active treatment, with all participants gaining access to experimental drugs after the initial 12-month period through an open-label extension. The trial will focus on patients with Richardson's syndrome (PSP-RS), which accounts for 70% of PSP diagnoses, with enrollment scheduled to begin in fall 2025.
Treatment Evaluation and Biomarker Analysis
The trial's effectiveness will be measured using clinical metrics, including the PSP Quality of Life scale. Success will be determined by evidence of slowed or halted disease progression in treated patients compared to the placebo group. Additionally, researchers will collect cerebrospinal fluid samples to monitor changes in PSP-associated molecules and identify novel biomarkers.
Emphasis on Diverse Representation
A distinguishing feature of this trial is its committed focus on including participants from traditionally underrepresented communities. Dr. Julio Rojas, MD, PhD, another principal investigator, emphasizes their approach: "We will build on experience from other community-engaged research programs and develop relationships with Spanish-speaking communities. These participants would be served by Spanish-speaking clinicians, including a neuropsychologist conducting assessments in Spanish."
To facilitate participation from underserved populations, the trial will cover transportation and accommodation costs for participants. UCSF is collaborating with CurePSP to assist in participant recruitment, particularly focusing on African American and other medically underserved communities.
Scientific Rationale
PSP, one of two FTD disorders primarily affecting the motor cortex, currently has no approved treatments. The trial's approach leverages the connection between PSP and other neurodegenerative conditions like ALS and Alzheimer's disease, which share common pathological features involving tau protein dysfunction. This biological overlap suggests that drugs developed for these related conditions might prove effective against PSP.
