Safety and Efficacy Study of VY-AADC01 for Advanced Parkinson's Disease (NCT03065192) | Clinical Trial Compass
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 range40 Years – 75 Years
SexALL
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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…
What they're measuring
1
Grading of adverse Events/Serious Adverse Events (AE's/SAE's)
Timeframe: Baseline to 3 Years After Gene Transfer
2
Magnetic Resonance Imaging (MRI)
Timeframe: Baseline to 3 Years After Gene Transfer
3
Routine physical examinations
Timeframe: Baseline to 3 Years After Gene Transfer
4
Routine clinical laboratory analysis
Timeframe: Baseline to 3 Years After Gene Transfer
5
Change in Columbia-Suicide Severity Rating Scale (C-SSRS) results
Timeframe: Baseline to 3 Years After Gene Transfer