Testing if PDR-001 Can Safely and Effectively Remove Harmful Brain Protein in Parkinson's
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-
| Condition(s) | Parkinson Disease (PD) |
|---|---|
| Status | Recruiting |
| Phase | Phase 1 |
| Study type | Interventional |
| 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 AAV |
| Who can participate | 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 inte |
| Ages | 40 Years to 65 Years |
| Sex | All |
| Lead sponsor | Ruijin Hospital |
| Locations | Shanghai, Shanghai Municipality, China |
| Start date | 2025-10-20 |
| NCT ID | NCT07157345 |
| Official listing | https://clinicaltrials.gov/study/NCT07157345 |