Safety Study of AADC Gene Therapy (VY-AADC01) for Parkinson's Disease (NCT01973543) | Clinical Trial Compass
CompletedPhase 1
Safety Study of AADC Gene Therapy (VY-AADC01) for Parkinson's Disease
United States15 participantsStarted 2013-10
Plain-language summary
Safety study of AADC gene transfer (VY-AADC01) in subjects with Parkinson's disease.
Who can participate
Age range40 Years – 70 Years
SexALL
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 Parkinson's disease
* Disease duration of at least 5 years or more
* Adequate duration of levodopa therapy
* Modified Hoehn and Yahr Staging of at least 2.5 in the OFF state
* Candidate for surgical intervention because of disabling motor complications.
* UPDRS Part III (total motor) score ≥ 25 and a maximum of 60 in the OFF state.
* Unequivocal responsiveness to dopaminergic therapy.
* Stable Parkinson's symptoms and medication regimen for at least 4 weeks prior to screening examination.
* Ability to comprehend and sign the informed consent.
* Normal Laboratory values prior to surgery.
* Neutralizing AAV2 antibody titer ≤ 1:1200
* 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).
* Subject agrees to not participate in any other therapeutic intervention study for 12 months after surgery.
* Subject agrees to not have any vaccinations within 30 days of surgery.
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) of less than 130 at screening.
* Presence or hi…