CompletedPhase 1

Safety and Efficacy Study of VY-AADC01 for Advanced Parkinson's Disease

United States16 participantsStarted 2017-05-11

Plain-language summary

Safety and efficacy of AADC gene transfer in participants with Parkinson's disease.

Who can participate

Age range

40 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: * Diagnosed with idiopathic PD. * Adequate duration of levodopa therapy. * Disease duration of at least 5 years or more. * Modified Hoehn \& Yahr Staging with at least 2.5 hours or more in the OFF state. * Candidate for surgical intervention because of disabling motor complications. * UPDRS Part III (total score) of at least 25 in the OFF state. * Unequivocal responsiveness to dopaminergic therapy. * Stable Parkinson's symptoms and medications for at least 4 weeks prior to screening evaluation. * Ability to comprehend and sign the informed consent. * Normal laboratory values prior to surgery. * Medically and mentally capable of undergoing and complying with the surgical procedure and protocol requirements. * Ability to travel to study visits alone or able to designate a caregiver. * Subject agrees to defer any neurological surgery, including deep brain stimulation, until after completing the 12 month study visit (unless recommended by study neurologist). * Approved by the Eligibility Review Committee. Exclusion Criteria: * Atypical or secondary parkinsonism, including but not limited to symptoms believed to be due to trauma, brain tumor, infection, cerebrovascular disease, other neurological disease, or to drugs, chemicals, or toxins. * Presence of dementia as defined by a Mattis Dementia Rating Scale - Second Edition (MDRS-2) score of less than 130 at screening. * Presence or history of psychosis, with the exception of mild, benign hallucinations belie…

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.

What they're measuring

Grading of adverse Events/Serious Adverse Events (AE's/SAE's)

Timeframe: Baseline to 3 Years After Gene Transfer

Magnetic Resonance Imaging (MRI)

Timeframe: Baseline to 3 Years After Gene Transfer

Routine physical examinations

Timeframe: Baseline to 3 Years After Gene Transfer

Routine clinical laboratory analysis

Timeframe: Baseline to 3 Years After Gene Transfer

Change in Columbia-Suicide Severity Rating Scale (C-SSRS) results

Timeframe: Baseline to 3 Years After Gene Transfer

Trial details

NCT IDNCT03065192

SponsorNeurocrine Biosciences

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2021-08-10

View on ClinicalTrials.gov More Idiopathic Parkinson's Disease trials

Who can participate

Age range

40 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.

What they're measuring

Grading of adverse Events/Serious Adverse Events (AE's/SAE's)

Timeframe: Baseline to 3 Years After Gene Transfer

Magnetic Resonance Imaging (MRI)

Timeframe: Baseline to 3 Years After Gene Transfer

Routine physical examinations

Timeframe: Baseline to 3 Years After Gene Transfer

Routine clinical laboratory analysis

Timeframe: Baseline to 3 Years After Gene Transfer

Change in Columbia-Suicide Severity Rating Scale (C-SSRS) results

Timeframe: Baseline to 3 Years After Gene Transfer