Brain gene therapy targets Parkinson's at its source
NCT ID NCT07157345
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This early-stage trial tests a new treatment called PDR-001 for Parkinson's disease. It uses a harmless virus to deliver a peptide into a specific brain area, aiming to clear toxic protein clumps that cause the disease. The study will enroll 12 people with early Parkinson's to check safety and see if it might slow the disease.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- PDR-001 (a peptide delivered via gene therapy to clear α-synuclein protein in the brain)
- What this could lead to
- If successful, this could lead to a treatment that slows or stops Parkinson's disease progression by targeting its root cause, rather than just easing symptoms.
- What could go wrong
- This is a very early Phase 1 trial with only 12 people, so safety and effectiveness are not yet proven. The gene therapy approach also carries risks like immune reactions or off-target effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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About 12 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Oct 2025
An estimate. Start dates often move.
- Expected to finish
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Dec 2029
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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40 to 65 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Ruijin hospital
RECRUITINGShanghai, Shanghai Municipality, 200000, China
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Other studies related to the condition(s) this trial covers.
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- Vibrating wristband put to the test for Parkinson's relief
- Can a breathing and Nerve-Zapping combo help clear the Parkinson's brain?
- Can group brain training slow Parkinson's cognitive decline?
- Old drug showdown: can benzhexol match procyclidine for Parkinson's tremor?