AAV2-hAADC for Parkinson's Disease (PDCS-01) (NCT07267065) | Clinical Trial Compass
Not Yet RecruitingPhase 1
AAV2-hAADC for Parkinson's Disease (PDCS-01)
United States9 participantsStarted 2026-02-01
Plain-language summary
This study is looking at whether a gene therapy called AAV2-hAADC is safe and may help people with Parkinson's Disease.
AAV2-hAADC is intended to increase the levels of dopamine in your brain. It contains a virus called adeno-associated virus 2 (AAV2) that has been modified to carry the genetic code for an enzyme called Aromatic L-Amino Acid Decarboxylase, or AADC for short.
In this study, AAV2-hAADC is delivered to two parts of the brain called the putamen and the caudate. Increasing the amount of AADC gene in these parts of the brain converts more levodopa into dopamine, the chemical that is lacking in PD.
Who can participate
Age range
18 Years – 75 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Male and female adults 18-75 years of age (inclusive), at the time of signing the informed consent.
. Diagnosed with idiopathic Parkinson's Disease as defined by the following:
. Disease duration since diagnosis of \>3 years.
. Patient reported symptom onset prior to the age of 50 (inclusive).
. Modified Hoehn \& Yahr Staging ≥2.5 in the practically defined OFF medication state (≥ 12 hours from last dose of anti-parkinsonian medications)
. Disabling motor complications with an average of ≥3 hours of OFF time per day during waking hours within 7 days of the screening visit as confirmed by the Parkinson's Disease (PD) diary.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
. International Parkinson and Movement Disorder Society (MDS) Unified Parkinson's Disease Rating Scale (UPDRS) Part III (total motor) score ≥25 in the clinically defined OFF state.
. Unequivocal responsiveness to dopaminergic therapy for a minimum of 1 year, including a 30% or greater improvement in the UPDRS III (motor score) between ON and OFF states, as determined by the Investigator after overnight withdrawal of Parkinson's medications.
Exclusion criteria
. Atypical or secondary parkinsonism, including but not limited to symptoms resulting from trauma, brain tumor, infection, cerebrovascular disease, other neurological disease, or to drugs, chemicals, or toxins, as determined by the Investigator.
. Presence of clinically significant cognitive impairment as defined by a Montreal Cognitive Assessment (MoCA) score of less than 241 at screening and/or clinical diagnosis of dementia by Investigator based on MoCA diagnostic criteria.
. Presence or history of psychosis, except for minor psychosis.
. Presence of severe depression, as indicated by a BDI-II score \>28 within 5 years of screening evaluation.
. Active suicidal ideation as indicated by positive response to items 4 or 5 on the screening C-SSRS, or any history of a suicide attempt
. Presence of impulse control disorder, defined as a total score ≥10 on the Questionnaire for Impulsive-Compulsive Disorders in Parkinson's Disease-Rating Scale (QUIP-RS).
. History of substance use disorder within 2 years of screening evaluation, as determined from interview with the participant and/or review of medical records.
. Brain imaging abnormalities in the striatum or other regions that would substantially increase risk of surgery in the opinion of the investigator.