The Progressive Supranuclear Palsy Clinical Trial Platform (PTP) is a multi-center, multi-regimen adaptive platform trial designed to evaluate multiple investigational products for the treatment of PSP simultaneously[1].
| Field | Value |
|---|---|
| NCT ID | NCT07173803 |
| Status | Not Yet Recruiting |
| Phase | Platform Trial |
| Sponsor | University of Pennsylvania |
| Start Date | 2024 |
| Duration | Approximately 5 years |
Unlike traditional single-drug trials, this platform trial allows multiple investigational products to be tested in parallel under a single protocol. This approach offers several advantages:
AADvac1 is a tau vaccine developed by Axon Neuroscience SE. It targets pathological tau proteins by stimulating the immune system to produce antibodies against them. The vaccine has shown promise in earlier studies for removing toxic tau species from the brain[2].
Mechanism: Active immunization with tau peptide conjugated to keyhole limpet hemocyanin (KLH) to generate anti-tau antibodies
Previous studies:
LM11A-31 is a small molecule p75 neurotrophin receptor (p75NTR) modulator developed by Astellas Pharma (formerly known as BMS-902464). It promotes nerve growth factor (NGF) signaling and has demonstrated neuroprotective effects in preclinical models[3].
Mechanism: Binds to p75NTR, preventing pro-NGF-mediated cell death while promoting TrkA-mediated survival signaling
Previous studies:
Both AADvac1 and LM11A-31 target different aspects of tau pathology:
The platform design allows for combinatorial insights, as patients can potentially receive both treatments in future iterations.
Traditional clinical trial designs face significant challenges in PSP:
Platform trials address these challenges through:
AADvac1 is a tau vaccine developed by Axon Neuroscience SE that targets pathological tau proteins:
The vaccine aims to reduce tau pathology by generating antibodies that:
LM11A-31 is a p75 neurotrophin receptor (p75NTR) modulator that promotes neuroprotection:
This approach is novel in PSP as it targets neuronal survival rather than tau pathology directly.
The platform will proceed through multiple phases:
Phase 1 (Current):
Phase 2:
Phase 3:
Primary:
Secondary:
| Trial | Agent | Target | Phase | Outcome |
|---|---|---|---|---|
| NCT07173803 | AADvac1, LM11A-31 | Tau, p75NTR | Platform | Ongoing |
| NCT04564555 | CoQ10 | Mitochondria | Phase 3 | Ongoing |
| E2814 | Tau aggregation | Phase 2 | ||
| NCT04253132 | Tolfenamic acid | Tau | Phase 2 | Completed |
The trial is currently not yet recruiting. Updates will be added as the trial progresses.
| Milestone | Expected Date |
|---|---|
| First patient enrolled | 2025 Q2 |
| Initial safety data | 2026 Q1 |
| Interim efficacy analysis | 2027 Q1 |
| First pivotal arm | 2028 |
NCT07173803 - The Progressive Supranuclear Palsy Clinical Trial Platform. ↩︎
Axon Neuroscience. Tau immunotherapy: AADvac1. Available from:. ↩︎
Longo FM, Massa SM. Small molecule TrkB receptor agonists for treatment of neurodegenerative diseases. Expert Opinion on Therapeutic Targets. 2013. ↩︎