The Progressive Supranuclear Palsy Clinical Trial Platform (PTP) is a multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of PSP.
The Progressive Supranuclear Palsy Clinical Trial Platform (PTP) is designed as a perpetual platform trial. This means that there is a single Master Protocol dictating the conduct of the trial. In this trial, multiple investigational products for PSP will be tested simultaneously or sequentially. Each investigational product will be tested in a regimen. Each regimen consists of a placebo-controlled trial, meaning that the active investigational product and matching placebo will be tested in each regimen. The additional details that govern the testing of each investigational product will be summarized in separate regimen-specific appendices (RSAs). Each regimen will have a separate ClinicalTrials.gov posting, which will include specific information about the regimen. All regimen-specific outcome measures will be detailed in each regimen posting. Participants will have an equal chance to be randomized to all regimens that are active at the time of screening. Once randomized to a regimen, participants will be randomized as outlined for each individual regimen to either study drug or placebo. New regimens will be continuously added as new investigational products become available. PTP will enroll additional participants as each new regimen becomes available. PTP is expected to launch with the following regimen: * Regimen A: AADvac1 * Regimen B: LM11A-31
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
440
Disease progression
Change in disease severity as measured by the 15-item modified Progressive Supranuclear Palsy Rating Scale (mPSPRS-15) in which the minimum score is 0 and the maximum score is 52, with higher scores indicating a worse outcome.
Time frame: 52 weeks
Disease progression
Change in disease severity as measured by the 10-item modified Progressive Supranuclear Palsy Rating Scale (mPSPRS-10) in which the minimum score is 0 and the maximum score is 30, with higher scores indicating a worse outcome.
Time frame: 52 weeks
Disease progression
Change in disease severity as measured by the 28-item Progressive Supranuclear Palsy Rating Scale (28-item PSPRS) in which the minimum score is 0 and the maximum score is 100, with higher scores indicating a worse outcome.
Time frame: 52 weeks
Experiences of daily living
Change in experiences of daily living over time as measured by the Cortical Basal ganglia Functional Scale (CBFS) in which the minimum score is 0 and the maximum score is 124, with higher scores indicating a worse outcome.
Time frame: 52 weeks
Activities of daily living
Change in activities of daily living over time as measured by the Schwab and England Activities of Daily Living Scale (SE-ADL) in which the minimum score is 0% and the maximum score is 100%, with lower scores indicating a worse outcome.
Time frame: 52 weeks
Disease severity
Change in disease severity over time as measured by the Clinical Global Impression of disease severity (CGIds) using a 7-point scale, ranging from 1 (normal, not ill) to 7 (extremely ill), with a higher score indicating a worse outcome.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Time frame: 52 weeks
Disease progression
Change in disease severity over time as measured by the Clinical Global Impression of Change (CGIC) using a 7-point scale, ranging from 1 (very much improved) to 7 (very much worse), with a score of 4 indicating no change.
Time frame: 52 weeks
Health-related quality of life
Change in quality of life over time as measured by the EuroQoL 5 Dimension - 5 Level (EQ-5D-5L) questionnaire in which the minimum score is 0 and the maximum score is 1, with lower scores indicating a worse outcome.
Time frame: 52 weeks
Brain volume
Change in volume in midbrain, pontine and other regions over time as measured by volumetric MRI.
Time frame: 52 weeks
Neurodegeneration
Change in plasma neurofilament light chain (NfL) concentration.
Time frame: 52 weeks