VY-AADC02 for Parkinson's Disease With Motor Fluctuations (RESTORE-1)
A Randomized, Sham Surgery Controlled, Double-blind, Multi-center, Phase 1 Clinical Trial, Evaluating the Safety of VY-AADC02 in Moderate to Advanced Parkinson's Disease Patients With Motor Fluctuations
1 other identifier
interventional
14
1 country
11
Brief Summary
The objective of this study is to assess the safety of VY-AADC02 in participants with Parkinson's disease (PD) with motor fluctuations.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_1
Started Oct 2018
Longer than P75 for phase_1
11 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 16, 2018
CompletedFirst Posted
Study publicly available on registry
June 19, 2018
CompletedStudy Start
First participant enrolled
October 17, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 30, 2024
CompletedStudy Completion
Last participant's last visit for all outcomes
October 30, 2024
CompletedDecember 10, 2024
December 1, 2024
6 years
May 16, 2018
December 5, 2024
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Number of participants with treatment-emergent adverse events (TEAEs)
Up to 5 years after surgery
Study Arms (2)
VY-AADC02 (NBIb-1817)
EXPERIMENTALSingle administration of up to 3.6 x 10\^12 vector genomes (vg) of VY-AADC02
Sham (Placebo) Surgery
PLACEBO COMPARATORSham surgical procedure
Interventions
Adeno-associated viral vector serotype 2 encoding human aromatic L-amino acid decarboxylase (AAV2- hAADC) infusion into the brain
Bilateral partial burr/twist holes without dura penetration
Eligibility Criteria
You may qualify if:
- Males and females, 40 to 75 years of age (inclusive)
- Diagnosis of PD, consistent with United Kingdom Brain Bank Criteria
- Motor responsiveness to dopaminergic therapy, demonstrated by improvement in Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS III score)
- Disease duration from diagnosis of ≥4 years
- 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
- 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
- In the judgment of the Investigator, stable Parkinson's features and symptoms for at least 4 weeks prior to screening evaluation
- 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
- Ability to travel to study visits
You may not qualify if:
- 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, as determined by the Investigator
- Montreal Cognitive Assessment (MoCA) score \<26
- New or unstable psychiatric conditions (psychosis, depression) within 1 year of screening
- Brain imaging abnormalities in the striatum or other regions that would substantially increase risk of surgery
- Contraindication to magnetic resonance imaging (MRI) and/or gadolinium-based contrast agents
- 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
- History of malignancy other than treated carcinoma in situ within 3 years of screening evaluation
- 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
- Severe, biphasic and/or uncontrolled dyskinesia
- Disabling or uncontrolled impulse control disorders
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Neurocrine Bioscienceslead
- Voyager Therapeuticscollaborator
Study Sites (11)
UC Irvine
Irvine, California, 92697, United States
UC Davis Health System
Sacramento, California, 95817, United States
University of California, San Francisco
San Francisco, California, 94143, United States
University of Colorado
Aurora, Colorado, 80045, United States
Emory University Hospital
Atlanta, Georgia, 30322, United States
Northwestern Medical Faculty Foundation
Chicago, Illinois, 60611, United States
University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
NYU Langone Medical Center
New York, New York, 10017, United States
Ohio State University Clinical Trials Management Office
Columbus, Ohio, 43210, United States
University of Philadelphia, Dept of Neurology
Philadelphia, Pennsylvania, 19107, United States
University of Pittsburgh Medical Center (UPMC)
Pittsburgh, Pennsylvania, 15213, United States
Related Publications (1)
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.
PMID: 31127735DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Clinical Development Lead
Neurocrine Biosciences
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 16, 2018
First Posted
June 19, 2018
Study Start
October 17, 2018
Primary Completion
October 30, 2024
Study Completion
October 30, 2024
Last Updated
December 10, 2024
Record last verified: 2024-12
Data Sharing
- IPD Sharing
- Will not share