Table 1.

Summary of circuit modifying GT trials to date

NCT number, sponsor, and site	Virus and dosing	Study details and outcomes
NCT00195143 Sponsor: Neurologix Site: Cornell, NY Duration: 6 months Status: completed Refs: [54]	AAV2-GAD65/GAD67 STN, unilateral Low dose: 4.5 × 109 vg/STN Medium dose: 1.4 × 1010 vg/STN High dose: 4.5 × 1010 vg/STN Infusion: 45 μl/STN iMRI—no	Category: phase 1, safety and dosing Patients: 12 subjects Disease stage: advanced PD; avg. duration 9 years Primary: safety. Well tolerated, no AEs Secondary: improvement in UPDRS, mainly on treated side; reduction in thalamic metabolism on PET on treated side
NCT00643890 Sponsor: Neurologix Site: Multicenter Status: Terminated (financial) Duration: 6 months Refs: [55–57] Long-term f/u: NCT01301573	AAV2-GAD65/GAD67 STN, bilateral Dose: 4.5 × 1010 vg/STN Infusion: 45 μl/STN iMRI—no	Category: phase 2, randomized, sham surgery Patients: 45 patients—split 22/23 (active/sham) Disease stage: advanced PD; avg. duration 10.6/12.0 years Primary: UPDRS scores Secondary: multiple including 18F-FDG