VY-AADC02 for Parkinson's Disease With Motor Fluctuations (RESTORE-1)
NCT ID: NCT03562494
Last Updated: 2024-12-10
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE1
14 participants
INTERVENTIONAL
2018-10-17
2024-10-30
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
TRIPLE
Study Groups
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VY-AADC02 (NBIb-1817)
Single administration of up to 3.6 x 10\^12 vector genomes (vg) of VY-AADC02
VY-AADC02
Adeno-associated viral vector serotype 2 encoding human aromatic L-amino acid decarboxylase (AAV2- hAADC) infusion into the brain
Sham (Placebo) Surgery
Sham surgical procedure
Sham (Placebo) Surgery
Bilateral partial burr/twist holes without dura penetration
Interventions
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VY-AADC02
Adeno-associated viral vector serotype 2 encoding human aromatic L-amino acid decarboxylase (AAV2- hAADC) infusion into the brain
Sham (Placebo) Surgery
Bilateral partial burr/twist holes without dura penetration
Eligibility Criteria
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Inclusion Criteria
2. Diagnosis of PD, consistent with United Kingdom Brain Bank Criteria
3. Motor responsiveness to dopaminergic therapy, demonstrated by improvement in Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS III score)
4. Disease duration from diagnosis of ≥4 years
5. An average of ≥3 hours of OFF time (that is, periods of insufficient control of motor PD symptoms) per day over 3 consecutive days as confirmed by the PD Diary
6. A stable, optimal regimen of Parkinson's medications including levodopa for at least weeks prior to screening evaluation. Participants must have a minimum duration of levodopa treatment of ≥1 year
7. In the judgment of the Investigator, stable Parkinson's features and symptoms for at least 4 weeks prior to screening evaluation
8. Agrees to defer any elective neurological surgery, including deep brain stimulation or ablation procedure for PD, levodopa or apomorphine infusion, or the addition of new dopaminergic formulations until after the study is completed, if medically appropriate
9. Ability to travel to study visits
Exclusion Criteria
2. Montreal Cognitive Assessment (MoCA) score \<26
3. New or unstable psychiatric conditions (psychosis, depression) within 1 year of screening
4. Brain imaging abnormalities in the striatum or other regions that would substantially increase risk of surgery
5. Contraindication to magnetic resonance imaging (MRI) and/or gadolinium-based contrast agents
6. Prior brain surgery, infusion therapies or planned treatments that could complicate the study procedure or negatively impact study evaluations as determined from participant interview, screening MRI, or medical records
7. History of malignancy other than treated carcinoma in situ within 3 years of screening evaluation
8. Prior gene transfer, current treatment with any investigational agent (drug or device) within 2 months of screening evaluation, or participation or plans to participate in another research study
9. Severe, biphasic and/or uncontrolled dyskinesia
10. Disabling or uncontrolled impulse control disorders
40 Years
75 Years
ALL
No
Sponsors
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Voyager Therapeutics
INDUSTRY
Neurocrine Biosciences
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Development Lead
Role: STUDY_DIRECTOR
Neurocrine Biosciences
Locations
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UC Irvine
Irvine, California, United States
UC Davis Health System
Sacramento, California, United States
University of California, San Francisco
San Francisco, California, United States
University of Colorado
Aurora, Colorado, United States
Emory University Hospital
Atlanta, Georgia, United States
Northwestern Medical Faculty Foundation
Chicago, Illinois, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
NYU Langone Medical Center
New York, New York, United States
Ohio State University Clinical Trials Management Office
Columbus, Ohio, United States
University of Philadelphia, Dept of Neurology
Philadelphia, Pennsylvania, United States
University of Pittsburgh Medical Center (UPMC)
Pittsburgh, Pennsylvania, United States
Countries
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References
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McFarthing K, Prakash N, Simuni T. CLINICAL TRIAL HIGHLIGHTS: 1. GENE THERAPY FOR PARKINSON'S, 2. PHASE 3 STUDY IN FOCUS - INTEC PHARMA'S ACCORDION PILL, 3. CLINICAL TRIALS RESOURCES. J Parkinsons Dis. 2019;9(2):251-264. doi: 10.3233/JPD-199001. No abstract available.
Other Identifiers
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PD-1105
Identifier Type: -
Identifier Source: org_study_id