PSP Trial Platform Enrolls First Participant in Landmark Multi-Drug Trial for Progressive Supranuclear Palsy
核心洞察
The PSP Trial Platform (PTP), a landmark platform trial testing multiple drugs simultaneously for progressive supranuclear palsy (搜索) (PSP), has enrolled its first participant.
Led by UCSF and funded by a five-year grant of up to $75.4 million from the National Institute on Aging, the trial will be conducted at 50 sites nationwide.
The trial aims to enroll 440 participants over two years, with 75% of participants receiving an active drug during the first year to minimize placebo assignment.
The PSP Trial Platform (PTP), a landmark clinical trial designed to test multiple drugs simultaneously in the search for effective treatments for progressive supranuclear palsy (搜索) (PSP), has enrolled its first participant. The milestone was announced by CurePSP (搜索), the leading nonprofit organization dedicated to PSP, corticobasal degeneration (CBD) and multiple system atrophy (MSA).
PSP is a rare brain disorder that can mimic Parkinson's disease in loss of balance, movement disorders and stiffness. There is no cure and no approved treatment, in part due to the limited number of clinical trials for potential treatments.
A Platform Designed to Accelerate Discovery
Led by the University of California, San Francisco (UCSF) and funded by a five-year grant of up to $75.4 million from the National Institute on Aging (NIA), the PTP is being conducted at 50 sites nationwide. Modeled on the HEALY ALS Platform Trial, it tests multiple drugs concurrently — and new ones can be added as they become available — accelerating discovery without starting from scratch each time.
The trial aims to enroll 440 participants over two years. Unlike traditional clinical trials, the PTP is designed so that 75% of participants receive an active drug during the first year, reducing the number of patients assigned to placebo. After that, all participants receive an active drug.
"We expect this trial to rapidly accelerate efforts to identify effective PSP therapies by increasing the number of promising drugs tested, while expanding access to potential treatments to more patients. It will also create a wealth of longitudinal data to enable researchers to better understand the causes of PSP and develop new diagnostic tests and therapies," said Adam Boxer, MD, PhD, Endowed Professor in Memory and Aging at UCSF, and lead study Principal Investigator.
Initial Investigational Therapies
Two of the drugs being tested are LM11A-31, from PharmatrophiX (搜索), and AADvac1, from Axon Neuroscience (搜索).
"Rather than targeting a single downstream consequence of disease, LM11A-31 is designed to protect neurons and their synaptic connections while addressing multiple mechanisms that drive neurodegeneration, including toxic tau biology," said Dr. Frank M. Longo, Co-Founder of PharmatrophiX (搜索). "The first patient enrolled represents more than the start of a clinical trial, it is the beginning of a potential paradigm shift for these patients and families."
"AADvac1's inclusion is an important step forward, built on more than two decades of our dedicated research into tau protein (搜索), a key driver of the disease pathology in PSP," said Michal Fresser, CEO of Axon Neuroscience (搜索). "We remain committed to advancing science that may one day lead to meaningful therapy options."
A New Chapter for the PSP Community
"The enrollment of the first participant marks the beginning of an important new chapter for the PSP community," said Kristophe Diaz, PhD, Chief Executive Officer of CurePSP (搜索). "The PTP is one of the most ambitious collaborative efforts ever undertaken in PSP research, bringing together leading researchers, trial sites, industry, government and nonprofit organizations to accelerate the development of better therapies."
"This is good news for patients and their families who are struggling with this disease. We are hopeful that this enrollment is the first step towards finding an effective treatment for this illness," said Anne-Marie Wills, MD, MPH, Director of the CurePSP (搜索) Center of Care at Massachusetts General Hospital, and study co-Principal Investigator.
Eligibility and Participation
The PTP is open to patients with Richardson's syndrome who have experienced progressive symptoms for fewer than five years and are accompanied by a care partner. Additional information is available through the trial's ClinicalTrials.gov listing (NCT07173803).