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VY-AADC02 for Parkinson's Disease With Motor Fluctuations (RESTORE-1)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03562494
Recruitment Status : Active, not recruiting
First Posted : June 19, 2018
Last Update Posted : March 22, 2023
Sponsor:
Collaborator:
Voyager Therapeutics
Information provided by (Responsible Party):
Neurocrine Biosciences

Study Description
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Brief Summary:
The objectives of this study are to assess the efficacy, safety and tolerability of VY-AADC02 in Patients with Parkinson's Disease with Motor Fluctuations.

Condition or disease Intervention/treatment Phase
Parkinson's Disease Biological: VY-AADC02 Other: Sham (Placebo) Surgery Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 85 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Randomized, Sham Surgery Controlled, Double-blind, Multi-center
Masking: Triple (Participant, Care Provider, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Sham Surgery Controlled, Double-blind, Multi-center, Phase 2 Clinical Trial, Evaluating the Efficacy and Safety of VY-AADC02 in Moderate to Advanced Parkinson's Disease Patients With Motor Fluctuations
Actual Study Start Date : October 17, 2018
Estimated Primary Completion Date : December 2023
Estimated Study Completion Date : December 2023

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Arms and Interventions
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Arm Intervention/treatment
Experimental: VY-AADC02 (NBIb-1817)
Single administration of up to 3.6 x 10^12 vector genomes (vg) of VY-AADC02
 Biological: VY-AADC02
Adeno-associated viral vector serotype 2 encoding human aromatic L-amino acid decarboxylase (AAV2- hAADC) infusion into the brain
Placebo Comparator: Sham (Placebo) Surgery
Sham surgical procedure
 Other: Sham (Placebo) Surgery
Bilateral partial burr/twist holes without dura penetration


Outcome Measures
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Primary Outcome Measures :
  1. Change in ON time without troublesome dyskinesia as recorded by participant in Parkinson's Disease (PD) diary [ Time Frame: Baseline to Day 360 ]

Secondary Outcome Measures :
  1. Change in Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS) III score in the OFF state [ Time Frame: Baseline to Day 360 ]
  2. Change in MDS-UPDRS II score [ Time Frame: Baseline to Day 360 ]

Other Outcome Measures:
  1. Safety outcome measured by treatment emergent adverse events (TEAEs), serious adverse events (SAEs), changes in vital signs and clinical laboratory analysis, and immunogenicity [ Time Frame: Baseline to Day 360 ]

Eligibility Criteria
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Ages Eligible for Study:   40 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  1. Males and females, 40 to 75 years of age (inclusive)
  2. Diagnosis of PD, consistent with United Kingdom Brain Bank Criteria
  3. Motor responsiveness to dopaminergic therapy, demonstrated by improvement in MDS-UPDRS III score
  4. Disease duration from diagnosis of ≥4 years
  5. An average of ≥3 hours of OFF time (ie, 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

Key Exclusion Criteria:

  1. 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
  2. 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 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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03562494


Locations
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Sponsors and Collaborators
Neurocrine Biosciences
Voyager Therapeutics
Investigators
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Study Director: Clinical Development Lead Neurocrocrine Biosciences
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Neurocrine Biosciences
ClinicalTrials.gov Identifier: NCT03562494    
Other Study ID Numbers: PD-1105
First Posted: June 19, 2018    Key Record Dates
Last Update Posted: March 22, 2023
Last Verified: March 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Neurocrine Biosciences:
Parkinson's Disease (PD)
Aromatic L-Amino Acid Decarboxylase
AADC
AAV
VY-AADC02
AAV2-hAADC
DDC
 Gene Therapy
Levodopa
Dopamine
Neurosurgery
NBIb-1817
Motor Fluctuations
Additional relevant MeSH terms:
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Parkinson Disease
Parkinsonian Disorders
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
 Nervous System Diseases
Movement Disorders
Synucleinopathies
Neurodegenerative Diseases