NCT07157345

Testing if PDR-001 Can Safely and Effectively Remove Harmful Brain Protein in Parkinson's Disease

A Study on the Safety, Tolerability, and Efficacy of PDR-001 Injection for Bilateral Stereotactic Subthalamic Nucleus (STN) Clearance of α-synuclein

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Ruijin Hospital · Academic / Other
• Sex: All
• Age: 40 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

Parkinson's disease (PD) poses a severe threat to human health, and its incidence is rising year by year. Current therapeutic options are limited by significant shortcomings. Pathological aggregation of α-synuclein and the consequent death of dopaminergic neurons are the primary drivers of PD pathogenesis. While siRNA-mediated knockdown of α-synuclein can offer some protection to dopaminergic neurons, its clinical utility is hampered by low cellular uptake, off-target effects, and transient activity. These drawbacks underscore the urgent need for novel strategies that can efficiently and specifically degrade α-synuclein to delay or even halt PD progression. Our prior work identified tat-βsyn-deg (PDR-001), a three-segment peptide that selectively targets α-synuclein. When packaged into AAV9 capsids and delivered via bilateral stereotaxic injection into the subthalamic nucleus, this peptide effectively reduces α-synuclein within the target region. Pre-clinical studies in both human-α-synuclein-expressing mice and non-human primate models of PD have demonstrated robust α-synuclein clearance and marked improvements in motor deficits (see Research Foundation). The present project will advance PDR-001 into first-in-human studies to evaluate safety and explore preliminary efficacy. Unlike conventional symptomatic therapies, this approach targets the root cause of PD, setting the stage for disease-modifying treatment. Successful translation would establish a new therapeutic paradigm capable of slowing or preventing PD progression.

Conditions

• Parkinson Disease (PD)

Interventions

Type	Name	Description
DRUG	PDR001	This drug was packaged into AAV9 capsids and delivered via bilateral stereotaxic injection into the subthalamic nucleus

Timeline

• Start date: 2025-10-20
• Primary completion: 2029-01-31
• Completion: 2029-12-31
• First posted: 2025-09-05
• Last updated: 2025-09-05

Locations

1 site across 1 country: China