Summary

for people ages 40-75 (full criteria)
at San Francisco, California and other locations
study started
estimated completion:
Paul Larsen

Description

Summary

The objectives of this study are to assess the distribution, efficacy, and safety of VY-AADC02 in Patients with Parkinson's Disease with Motor Fluctuations.

Official Title

A Randomized, Placebo Surgery Controlled, Double-blinded, Multi-center, Phase 2 Clinical Trial, Evaluating the Efficacy and Safety of VY-AADC02 in Advanced Parkinson's Disease With Motor Fluctuations

Keywords

Parkinson Disease PD, Parkinson's disease Aromatic L-Amino Acid Decarboxylase AADC AAV2-AADC AAV2 VY-AADC AAV2-hAADC Dopa Decarboxylase VY-AADC02

Eligibility

For people ages 40-75

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. Unequivocal responsiveness to dopaminergic therapy, as judged by the Investigator.
  4. Disease duration from diagnosis of ≥4 years.
  5. In the judgment of the Investigator, a stable, optimal regimen of Parkinson's medications for at least 4 weeks prior to screening evaluation.
  6. Stable cognitive and psychological function based on screening evaluations.
  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 neurological surgery, including deep brain stimulation, other invasive treatments for PD including duodopa, or the addition of new dopaminergic formulations until after completing the 12-month study visit.
  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. Use of tetrahydrocannabinol within 6 months of screening evaluation.
  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 or infusion therapies 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. Ongoing treatments or planned treatments that might interfere with interpretation of the study outcome including deep brain stimulation.

Locations

  • San Francisco Veterans Affairs Medical Center accepting new patients
    San Francisco California 94143 United States
  • UC Irvine accepting new patients
    Irvine California 92697 United States

Lead Scientist

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Voyager Therapeutics
ID
NCT03562494
Phase
Phase 2
Study Type
Interventional
Last Updated