Parkinson's Disease Clinical Trial
Official title:
An Open-label Safety and Efficacy Study of VY-AADC01 Administered by MRI-Guided Convective Infusion Using a Posterior Trajectory Into the Putamen of Participants With Parkinson's Disease With Fluctuating Responses to Levodopa
Verified date | March 2022 |
Source | Neurocrine Biosciences |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
Safety and efficacy of AADC gene transfer in participants with Parkinson's disease.
Status | Completed |
Enrollment | 16 |
Est. completion date | August 10, 2021 |
Est. primary completion date | August 10, 2021 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 40 Years to 75 Years |
Eligibility | 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 believed in the judgment of the Investigator to be related to Parkinson's medications. - Presence of severe depression, as indicated by a BDI-II score greater than 28, or a history of a major affective disorder within 5 years of screening evaluation. - Active suicidal ideation or suicide attempt within 5 years of screening evaluation. - History of substance abuse within 2 years of screening evaluation. - Brain imaging abnormalities in the striatum or other regions that would substantially increase risk of surgery. - Contraindication to MRI and/or gadoteridol. - Coagulopathy or inability to temporarily stop any anticoagulation or antiplatelet therapy prior to surgery. - Prior brain surgery including lesioning procedures, deep brain stimulation, infusion therapies or any other brain surgery. - Prior gene transfer. - History of stroke, poorly controlled or significant cardiovascular disease, diabetes, or any other acute or chronic medical condition. - History of malignancy other than treated carcinoma in situ within 3 years of screening evaluation. - Clinically apparent or laboratory-detected infection. - Prior or current treatment with any investigational agent within 2 months of screening evaluation. - Inability to comply with the procedures of the protocol, including completion of paper Parkinson's disease diaries, frequent and prolonged study visits including off medication visits, and travel. - Chronic immunosuppressive therapy, including chronic steroids, immunotherapy, cytotoxic therapy, and chemotherapy. - Any serious medical condition or abnormal finding on physical examination or laboratory investigation that would substantially increase the risks of the study procedures. - Any medical condition that is likely to lead to disability during the course of the study and interfere with or confound study assessments. - Pregnant and lactating women. - Male or female with reproductive capacity who is unwilling to use barrier contraception for 6 months after surgery. - Plans to receive any vaccination within 30 days of surgery. - Any factors, medical or social, which would likely cause the participant to be unable to follow the study protocol, including geographical inaccessibility. - Ongoing treatments including neuroleptic medications, apomorphine, or levodopa infusion therapy (Duopa®). - Plans to participate in any other therapeutic intervention study within 12 months after surgery. |
Country | Name | City | State |
---|---|---|---|
United States | Emory University | Atlanta | Georgia |
United States | Ohio State University (OSU) | Columbus | Ohio |
United States | University of Pittsburgh Medical Center (UPMC) | Pittsburgh | Pennsylvania |
United States | University of California, San Francisco (UCSF) | San Francisco | California |
Lead Sponsor | Collaborator |
---|---|
Neurocrine Biosciences | Voyager Therapeutics |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Grading of adverse Events/Serious Adverse Events (AE's/SAE's) | Grading will be assessed using NCI CTCAE, version 4.03. | Baseline to 3 Years After Gene Transfer | |
Primary | Magnetic Resonance Imaging (MRI) | Safety of VY-AADC01 will be assessed by any clinically significant abnormalities on MRI scans as compared to Baseline. | Baseline to 3 Years After Gene Transfer | |
Primary | Routine physical examinations | Safety of VY-AADC01 will be assessed by routine physical examinations. | Baseline to 3 Years After Gene Transfer | |
Primary | Routine clinical laboratory analysis | Safety of VY-AADC01 will be assessed by routine clinical laboratory analysis. | Baseline to 3 Years After Gene Transfer | |
Primary | Change in Columbia-Suicide Severity Rating Scale (C-SSRS) results | C-SSRS is a standardized suicidal rating system. | Baseline to 3 Years After Gene Transfer | |
Secondary | Change in Parkinson's medications | Change in Parkinson's medications compared to Baseline. | Baseline to 3 Years After Gene Transfer | |
Secondary | Change in motor function using Parkinson Disease Diaries | Diary used to assess changes in PD motor symptoms. | Baseline to 3 Years After Gene Transfer | |
Secondary | Change in motor function using a Stand-Walk-Sit Test | Standardized test used in PD studies to assess functional mobility. | Baseline to 3 Years After Gene Transfer | |
Secondary | Change in motor function using Modified Hoehn and Yahr Scale | Scale used to measure overall level of disability due to PD. | Baseline to 3 Years After Gene Transfer | |
Secondary | Change in motor function using Unified Parkinson's Disease Rating Scale (UPDRS) | Standard assessment scale used to quantify signs and symptoms of PD. | Baseline to 3 Years After Gene Transfer | |
Secondary | Change in occurrence of dyskinesia using Unified Dyskinesia Rating Scale (UDysRS) | Comprehensive rating tool used to assess essential features of dyskinesia in PD. | Baseline to 3 Years After Gene Transfer | |
Secondary | Change in mood using Beck Depression Inventory II (BDI-II) | Self-administered measure of depression symptoms. | Baseline to 3 Years After Gene Transfer | |
Secondary | Change in cognitive function using Modified Cognitive Assessment (MoCA) | Rapid screening instrument for mild cognitive dysfunction. | Baseline to 3 Years After Gene Transfer | |
Secondary | Change in cognitive function using Mattis Dementia Rating Scale - Second Edition (MDRS-2) | Standardized neuropsychological test battery to measure dementia. | Baseline to 3 Years After Gene Transfer | |
Secondary | Change in compulsive behavior using the Questionnaire Impulsive-Compulsive Disorders in Parkinson's Disease-Rating Scale (QUIP-RS) | Rating scale to quantify the severity of a variety of compulsive disorders and behaviors. | Baseline to 3 Years After Gene Transfer | |
Secondary | Change in sleep quality and disturbance using the Parkinson's Disease Sleep Scale 2 (PDSS-2) | Visual analogue scale addressing 15 commonly reported symptoms associated with sleep disturbance in PD. | Baseline to 3 Years After Gene Transfer | |
Secondary | Change in Non-Motor System Scale (NMSS) | Standard rating scale that assesses the non-motor symptoms that may be associated with PD. | Baseline to 3 Years Gene Transfer | |
Secondary | Change in quality of life using Parkinson's Disease Questionnaire (PDQ39) | Validated questionnaire to measure health related quality of life in PD patients. | Baseline to 3 Years After Gene Transfer | |
Secondary | Change in quality of life using Schwab and England Scale | PD specific disability scale used to express the levels of independence with activities of daily living. | Baseline to 3 Years After Gene Transfer | |
Secondary | Change in quality of life using in Clinical Global Impression Scale (CGI) | Scale used to assess treatment response in psychiatric patients. | Baseline to 3 Years After Gene Transfer | |
Secondary | Change in quality of life using Patient Global Impression Scale (PGI) | Scale used to assess participants improvement in their PD symptoms. | Baseline to 3 Years After Gene Transfer |
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