PDR-001 delivered into the brain to remove harmful alpha-synuclein in early Parkinson's

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 1 interventional study delivers tat-βsyn-deg (PDR-001) packaged in AAV9 by bilateral stereotaxic injection into the subthalamic nucleus to reduce pathological α-synuclein. The primary focus is safety and target engagement in a small cohort of patients with early-stage primary Parkinson's disease. Eligible participants are ages 40–65 with disease duration ≤5 years and Hoehn & Yahr stage ≤2, while those with atypical parkinsonism, prior intracranial surgery, or structural brain abnormalities are excluded. The trial is conducted at a single center (Ruijin Hospital, Shanghai) with surgical delivery and scheduled follow-up visits.

Why it matters

Who can join: the full rules

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.

Where it's running

• Ruijin hospital
  Shanghai, Shanghai Municipality, China Recruiting

Study contacts

• Study coordinator: Yi Zhang
• Email: zy12893@rjh.com.cn
• Phone: 64370045

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.