Recruiting · registry status

Testing a treatment aimed at a protein linked to Parkinson’s

Researchers investigate PDR001, an experimental treatment intended to help remove a protein associated with Parkinson’s from the brain.

Testing a treatment or activity · Study reference: NCT07157345

Plain-language introduction written with AI from the registry; not independently checked by a clinician. Read the original details below ↓

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Looking for volunteers

Start (planned): 2025-10-20
Main measurements finished (planned): 2029-01-31
Study finished (planned): 2029-12-31

Planned dates can move. A study finishing does not tell us when a paper will be published.

No results summary has been confirmed in the registry records we imported. See connected papers below; we keep checking after recruitment ends.

Changes we have recorded

2026-10-11 — recruiting

These are dates we observed a change, not necessarily the dates it happened.

Papers connected to this study

No connected paper has been found yet. The tracker checks the growing library for study identifiers and registry-linked publications.

Who can join?

Ages 40 years to 65 years · Does not accept healthy volunteers

These are starting points, not the full rules. The research team can tell you whether the study is right for your situation.

Read all the rules for taking part

Sex eligibility reported by registry: all

Inclusion Criteria: To be eligible for inclusion in this clinical study, all of the following criteria must be met: 1. Clinically confirmed diagnosis of primary PD (in accordance with the 2016 Chinese Diagnostic Criteria for Parkinson's Disease or the 2015 MDS Clinical Diagnostic Criteria for primary PD); 2. Age 40-65 years (inclusive) at screening, either sex; 3. Disease duration ≤ 5 years; 4. Hoehn \& Yahr stage ≤ 2 in the "off" state. Exclusion Criteria: Exclusion Criteria 1. Atypical or secondary parkinsonian syndromes (e.g., Parkinson-plus syndromes, hereditary parkinsonism, drug-induced parkinsonism, etc.). 2. Contra-indications to surgery, or any prior intracranial procedure such as deep-brain stimulation, pallidotomy, or other extrapyramidal surgery, or any other neurosurgical intervention deemed by the investigator to interfere with study participation. 3. Previous neuroimaging revealing structural brain abnormalities, cerebral vascular malformations, intracranial tumors, risk of intracranial hemorrhage, traumatic brain injury, or other significant findings. 4. Mini-Mental State Examination (MMSE) score \< 24. 5. Patient Health Questionnaire-9 (PHQ-9) score ≥ 16. 6. Abnormal hepatic or renal function: aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \> 1.5 × upper limit of normal (ULN), or serum creatinine (Cr) \> 1.5 × ULN. 7. Coagulation disorders or current use of anticoagulants. 8. Positive screening for infectious diseases: * Hepatitis B surface antigen (HBsAg) or Hepatitis B virus DNA (HBV-DNA) positive; * Hepatitis C virus RNA (HCV-RNA) positive; * Human immunodeficiency virus (HIV) positive; * Positive syphilis serology. 9. Currently receiving antiviral therapy for hepatitis B or C.

Full study name & original research details

Official study title

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

Short title used by the registry

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

Original 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.

Conditions reported: Parkinson Disease (PD)

Registry records for this study

Records are joined using registration identifiers. Titles alone do not establish that two studies are the same.

ClinicalTrials.gov · NCT07157345 ↗ — RECRUITING; checked 2026-10-13
ClinicalTrials.gov · NCT07157345 ↗ — Recruiting; WHO ICTRP processed 2025-09-08; checked 2026-10-13

Study type: Interventional
Interventions: PDR001
Phases: PHASE1
Sponsor: Ruijin Hospital
Start date reported by registry: 2025-10-20 (estimated)

Registry updated: 2025-09-05 · Status last verified by the registry submitter: 2025-08

Registry records retrieved 2026-10-13 (UTC). Individual records may have older updates. Recruitment and eligibility must be confirmed with the study team.

Contact the research team

Public study contacts supplied to the registry. Ask whether recruitment is still open and what participation involves.

Yi Zhang · 64370045 · zy12893@rjh.com.cn

Study locations

Site status can differ from overall study status. “Status not reported” means local availability needs confirmation. Remote participation and travel arrangements must be checked with the team.

Ruijin hospital

Shanghai, Shanghai Municipality, China

Recruiting

Facility not reported

China

Status not reported

The original descriptions and participation rules come from the registry. Participation is voluntary and does not guarantee benefit.