Summary

Eligibility
for people ages 40-75 (full criteria)
Location
at San Francisco, California and other locations
Dates
study started
estimated completion
Principal Investigator
by Chadwick Christine

Description

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.

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

Keywords

Parkinson's Disease 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 Parkinson Disease VY-AADC02 (NBIb-1817)

Eligibility

You can join if…

Open to people ages 40-75

  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

You CAN'T join if...

  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

Locations

  • San Francisco VA Medical Center accepting new patients
    San Francisco California 94143 United States
  • University of California, San Francisco accepting new patients
    San Francisco California 94143 United States

Lead Scientist at UCSF

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Neurocrine Biosciences
Links
RESTORE-1 Study Website
ID
NCT03562494
Phase
Phase 2
Study Type
Interventional
Last Updated