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

PROTOCOL NUMBER

PD-1105

PHASE

2

PATIENT POPULATION

Males and females,
40 to 75 years of age (inclusive)

CONDITION

Diagnosis of Parkinson’s disease, consistent with United Kingdom Brain Bank Criteria

Diagnosis of Parkinson’s disease ≥ 4 years

Unequivocal responsiveness to dopaminergic therapy

Stable Parkinson’s features and symptoms for at least 4 weeks prior to screening evaluation

STUDY DRUGS

Experimental gene therapy (VY-AADC02)

—compared to—

Placebo surgical procedure

OBJECTIVE

To assess:

  • The delivery and enzyme activity of VY‑AADC02 administered to the putamen, bilaterally, using stereotactic neurosurgery with MRI guidance
  • The efficacy and safety of VY‑AADC02

METHOD OF ADMINISTRATION

Infused bilaterally into the putamen during a one-time surgical procedure

LENGTH OF STUDY PARTICIPATION

Approximately 21 study visits over 14 months, including 2 months of screening visits and 12 months of post‑surgical visits

EXTENSION STUDY

Those who complete study participation have the option to take part in a long‑term extension for up to eight years

Study Treatment, Follow-up, and Long-term Extension

Potential participants will be screened for study eligibility within the 60 days before Day 0 (the day of surgery). After treatment on Day 0, participants will be followed for 12 months. Patients will then be invited to participate in a long‑term extension study for up to an additional 8 years.