NCT07157345

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

Recruiting now Phase 1 Last updated 5 September 2025

What this trial tests

Phase 1 trial testing PDR001 in Parkinson Disease (PD) in 12 participants. Currently enrolling.

Timeline

20 October 2025

Primary endpoint
31 January 2029

31 December 2029

Quick facts

Lead sponsor	Ruijin Hospital
Phase	Phase 1
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	12
Start date	20 October 2025
Primary completion	31 January 2029
Estimated completion	31 December 2029
Sites	1 location across China

Drugs / interventions tested

• PDR001 — full drug profile →

Conditions studied

• Parkinson Disease (PD) — all drugs for Parkinson Disease (PD) →

Sponsor

Ruijin Hospital

Who can join

Adults 40 to 65, any sex, with Parkinson Disease (PD). Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

1. Exploring the Potential of Receptor Silencing in the Tumor Microenvironment by RNA Interference
   · 2026