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NCT07157345 | RECRUITING | Parkinson Disease (PD)

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

Ruisin Hospital

Brief 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.

Condition or disease

Parkinson Disease (PD)

Intervention/treatment

PDR001

Phase

PHASE1

Study Type : INTERVENTIONAL
Estimated Enrollment : 12 participants
Masking : NONE
Primary Purpose : TREATMENT
Official Title : A Study on the Safety, Tolerability, and Efficacy of PDR-001 Injection for Bilateral Stereotactic Subthalamic Nucleus (STN) Clearance of α-synuclein
Actual Study Start Date : 2025-10-20
Estimated Primary Completion Date : 2029-01-31
Estimated Study Completion Date : 2029-12-31

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 40 Years to 65 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers:
Criteria
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.
    Testing if PDR-001 Can Safely and Effectively Remove Harmful Brain Protein in Parkinson's Disease

    Location Details

    NCT07157345

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    How to Participate

    Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

    Locations

    RECRUITING

    China, Shanghai Municipality

    Household

    Shanghai, Shanghai Municipality, China, 200000