Parkinson Disease (PD)Phase 1NCT07157345
Testing if PDR-001 Can Safely and Effectively Remove Harmful Brain Protein in Parkinson's Disease
A Phase 1 study testing PDR001 in people with Parkinson Disease (PD), run by Ruijin Hospital at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 40 to 65 years
- Conditions the study is for
- Parkinson Disease (PD)
- What is being tested?
- PDR001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteChina
- Study dates
- 20 Oct 2025to 31 Dec 2029
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 40 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- To be eligible for inclusion in this clinical study, all of the following criteria must be met:
- 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);
- Age 40-65 years (inclusive) at screening, either sex;
- Disease duration ≤ 5 years;
- Hoehn \& Yahr stage ≤ 2 in the "off" state.
Who cannot join
- Atypical or secondary parkinsonian syndromes (e.g., Parkinson-plus syndromes, hereditary parkinsonism, drug-induced parkinsonism, etc.).
- 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.
- Previous neuroimaging revealing structural brain abnormalities, cerebral vascular malformations, intracranial tumors, risk of intracranial hemorrhage, traumatic brain injury, or other significant findings.
- Mini-Mental State Examination (MMSE) score < 24.
- Patient Health Questionnaire-9 (PHQ-9) score ≥ 16.
- 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.
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- Coagulation disorders or current use of anticoagulants.
- 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.
- Currently receiving antiviral therapy for hepatitis B or C.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Ruijin hospital
Shanghai, Shanghai Municipality, 200000
About this study
Official title: A Study on the Safety, Tolerability, and Efficacy of PDR-001 Injection for Bilateral Stereotactic Subthalamic Nucleus (STN) Clearance of α-synuclein
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
- Parkinson Disease (PD)
- Being tested
- PDR001
- Keywords
- PDR-001, Parkinson disease
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PDR001Drug
This drug was packaged into AAV9 capsids and delivered via bilateral stereotaxic injection into the subthalamic nucleus
| Study group | Role | What they receive |
|---|---|---|
| primary parkinson's disease | Experimental | PDR-001 injection, inject once, 0.4 milliliters each time Receives: Drug: PDR001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
PDR-001 treatment-related adverse events as assessed by CTCAE v5.0
CTCAE 5.0 records the severity of adverse events, which is divided into grades 1 to 5
Measured: From enrollment to the end of treatment at 52 weeks
Titer levels of capsid neutralizing antibodies and binding antibodies against recombinant adeno-associated virus (rAAV) in serum
Record the titer changes before and after treatment
Measured: From enrollment to the end of treatment at 52 weeks
Titer of rAAV vectors in whole blood
Record the titer changes before and after treatment
Measured: From enrollment to the end of treatment at 52 weeks
Secondary outcomes (8)
Evaluation of the use of antiparkinsonian drugs will be assessed using the Levodopa Equivalent Daily Dose (LEDD)
The total daily dose of antiparkinsonian medication (converted to LEDD, mg/day) will be calculated and compared before and after treatment. A decrease in LEDD indicates reduced drug burden (better outcome), while an increase indicates greater drug requirement (worse outcome).
Measured: From enrollment to the end of treatment at 52 weeks
Treatment efficacy will be evaluated using the Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
This scale has a total score range of 0 to 260, with higher scores indicating more severe motor and non-motor symptoms (worse outcome). Changes in scores before and after treatment will be recorded.
Measured: From enrollment to the end of treatment at 52 weeks
Treatment efficacy will be evaluated using the Patient Global Impression - Improvement scale (PGI-I)
This is a 7-point scale ranging from 1 ("very much improved") to 7 ("very much worse"), with lower scores indicating greater improvement (better outcome). Changes in scores before and after treatment will be recorded.
Measured: From enrollment to the end of treatment at 52 weeks
Treatment efficacy will be evaluated using the Clinical Global Impression - Improvement scale (CGI-I)
This is a 7-point scale ranging from 1 ("very much improved") to 7 ("very much worse"), with lower scores indicating greater clinical improvement (better outcome). Changes in scores before and after treatment will be recorded.
Measured: From enrollment to the end of treatment at 52 weeks
Treatment efficacy will be evaluated using the Mini-Mental State Examination (MMSE)
The total score ranges from 0 to 30, with higher scores indicating better cognitive function (better outcome). Changes in scores before and after treatment will be recorded.
Measured: From enrollment to the end of treatment at 52 weeks
Treatment efficacy will be evaluated using the Hamilton Depression Rating Scale (HAM-D, 17-item version)
The total score ranges from 0 to 52, with higher scores indicating more severe depressive symptoms (worse outcome). Changes in scores before and after treatment will be recorded.
Measured: From enrollment to the end of treatment at 52 weeks
Change in anxiety symptoms as measured by the Hamilton Anxiety Rating Scale (HAM-A)
The Hamilton Anxiety Rating Scale (HAM-A) is a clinician-administered scale with scores ranging from 0 to 56, where higher scores indicate greater severity of anxiety symptoms. Treatment efficacy will be evaluated by the change in HAM-A total score from baseline to 52 weeks
Measured: From enrollment to the end of treatment at 52 weeks
Change in sleep-related problems as measured by the Parkinson's Disease Sleep Scale-2 (PDSS-2)
PDSS-2 is a 15-item questionnaire. The total score ranges from 0 to 60, with higher scores indicating worse sleep-related problems. Treatment efficacy will be evaluated by the changes in PDSS-2 from baseline to 52 weeks
Measured: From enrollment to the end of treatment at 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Sep 2025
- Study start20 Oct 2025 (estimated)
- Primary completion31 Jan 2029 (estimated)
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Parkinson Disease (PD)Studies of the same condition, open and under way.
- More trials run by Ruijin HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ruijin Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07157345
- Sponsor's protocol ID
- V1.0/2025-07-06
- Status last verified
- Aug 2025
- First submitted
- 5 Aug 2025
- Last updated
- 5 Sep 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study on the Safety, Tolerability, and Efficacy of PDR-001 Injection for Bilateral Stereotactic Subthalamic Nucleus (STN) Clearance of α-synuclein
- ClinicalTrials.gov ID
- NCT07157345
- Acronym / sponsor ID
- V1.0/2025-07-06
- Conditions
- Parkinson Disease (PD)
- Keywords
- PDR-001, Parkinson disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Ruijin HospitalUniversity, hospital or other
- Study start
- 20 Oct 2025 (estimated)
- Primary completion
- 31 Jan 2029 (estimated)
- Study completion
- 31 Dec 2029 (estimated)
- First posted
- 5 Sep 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| primary parkinson's disease | Experimental | Drug: PDR001PDR-001 injection, inject once, 0.4 milliliters each time |
Who may qualify
- Ages
- 40 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- To be eligible for inclusion in this clinical study, all of the following criteria must be met:
- 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);
- Age 40-65 years (inclusive) at screening, either sex;
- Disease duration ≤ 5 years;
- Hoehn \& Yahr stage ≤ 2 in the "off" state.
Key exclusion criteria (13 listed)
- Atypical or secondary parkinsonian syndromes (e.g., Parkinson-plus syndromes, hereditary parkinsonism, drug-induced parkinsonism, etc.).
- 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.
- Previous neuroimaging revealing structural brain abnormalities, cerebral vascular malformations, intracranial tumors, risk of intracranial hemorrhage, traumatic brain injury, or other significant findings.
- Mini-Mental State Examination (MMSE) score < 24.
- Patient Health Questionnaire-9 (PHQ-9) score ≥ 16.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PDR001This drug was packaged into AAV9 capsids and delivered via bilateral stereotaxic injection into the subthalamic nucleus | Drug | primary parkinson's disease |
Primary outcome
PDR-001 treatment-related adverse events as assessed by CTCAE v5.0
Time frame: From enrollment to the end of treatment at 52 weeks
CTCAE 5.0 records the severity of adverse events, which is divided into grades 1 to 5
Titer levels of capsid neutralizing antibodies and binding antibodies against recombinant adeno-associated virus (rAAV) in serum
Time frame: From enrollment to the end of treatment at 52 weeks
Record the titer changes before and after treatment
Titer of rAAV vectors in whole blood
Time frame: From enrollment to the end of treatment at 52 weeks
Record the titer changes before and after treatment
8 secondary outcomes
Evaluation of the use of antiparkinsonian drugs will be assessed using the Levodopa Equivalent Daily Dose (LEDD)
Time frame: From enrollment to the end of treatment at 52 weeks
Treatment efficacy will be evaluated using the Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
Time frame: From enrollment to the end of treatment at 52 weeks
Treatment efficacy will be evaluated using the Patient Global Impression - Improvement scale (PGI-I)
Time frame: From enrollment to the end of treatment at 52 weeks
Treatment efficacy will be evaluated using the Clinical Global Impression - Improvement scale (CGI-I)
Time frame: From enrollment to the end of treatment at 52 weeks
Treatment efficacy will be evaluated using the Mini-Mental State Examination (MMSE)
Time frame: From enrollment to the end of treatment at 52 weeks
Treatment efficacy will be evaluated using the Hamilton Depression Rating Scale (HAM-D, 17-item version)
Time frame: From enrollment to the end of treatment at 52 weeks
Change in anxiety symptoms as measured by the Hamilton Anxiety Rating Scale (HAM-A)
Time frame: From enrollment to the end of treatment at 52 weeks
Change in sleep-related problems as measured by the Parkinson's Disease Sleep Scale-2 (PDSS-2)
Time frame: From enrollment to the end of treatment at 52 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Sep 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing PDR001 in people with Parkinson Disease (PD), run by Ruijin Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 5 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Yi Zhang (64370045, zy12893@rjh.com.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
PDR-001 treatment-related adverse events as assessed by CTCAE v5.0 (time frame: From enrollment to the end of treatment at 52 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Sep 2025.