Table 3.
Summary of GF GT trials to date
| NCT number, sponsor and site | Virus and dosing | Study details and outcomes |
|---|---|---|
|
Sponsor: Ceregene Site: Univ. California, San Francisco, and Rush, Chicago Duration: 12 months Status: completed Refs: [85] |
AAV2-NRTN (CERE-120) Putamen, bilateral Low dose: 1·3 × 1011 vg/patient High dose: 5.4 × 1011 vg/patient Infusion: 8 × 5μl deposits/side, split between 4 needle tracks/side iMRI—no |
Category: phase 1, safety and dosing Patients: 12 subjects Disease stage: advanced PD; avg. 11 years Primary: safe and well tolerated Secondary: no significant benefits in OFF UPDRS3 at 12 months and 18F-L-DOPA PET at 6 and 12 months |
|
Sponsor: Ceregene Site: multicenter Duration: 12 months Status: completed Refs: (86) |
AAV2-NRTN (CERE-120) Putamen, bilateral Dose: 5.4 × 1011 vg/patient Infusion: 8 × 5μl deposits/side (40 μl) 4 needle tracks/side, 2 deposits/track iMRI—no |
Category: phase 2, randomized, double-blind, sham-surgery–controlled Patients: 58 subjects–38/20 in surgery/sham arms Disease stage: advanced PD; avg. 9.5/10.0 years, in active/sham arms Primary outcome: nonsignificant improvements over sham surgery in OFF UPDRS3 Secondary outcome: nonsignificant trends to improvement UPDRS; timed motor tests; dyskinesia rating scale; diaries; PDQ-39, SF-36, and clinical global impression |
|
Sponsor: Ceregene Site: multicenter Duration: 15–24 months Status: completed Refs: (87) |
AAV2-neurturin (CERE-120) Putamen and SN, bilateral Dose: 2.4 × 1011 vg/SNc; 1.0 × 1012 vg/putamen Infusion: two deposits of 15 μl/SNc; one deposit of 50 μl in each of 3 putaminal tracts iMRI—no |
Trial category: phase 2, randomized, double-blind, sham-surgery–controlled Patients: 48 patients, 23/25 treated/sham arms Disease stage: advanced PD; avg. duration 7.8/8.6 years in treatment/sham arms Primary: no significant change in OFF UPDRS part 3 at 15 months Secondary: nonsignificant changes in other UPDRS, diaries, PDQ-39 |
|
Sponsor: NIHCC Site: Bethesda Status: active, NR Duration: 18 months Refs: (88) |
AAV2-GDNF Putamen, bilateral Low dose: 4.5 × 1010 vg per putamen Medium dose: 1.5 × 1011 vg/putamen High dose: 4.5 × 1011 vg/putamen 450 μl/putamen iMRI—yes |
Trial category: phase 1, safety and dose finding, 4 dose levels Patients: 25 subjects; three dose levels Disease stage: > 5 years disease Primary: safety and tolerability. Well tolerated. Six SAEs, none attributable to study drug; all resolved Secondary: Nonsignificant improvements in 18F-F-DOPA PET at 18 months. No significant change in UPDRS, LED at 6/18 months; PET scan at 18 months |
|
Sponsor: Brain Neurotherapy Bio, Inc. Site: University of California NYR Ohio, Duration: TBA Status: recruiting REFS: clinicaltrials.gov |
AAV2-GDNF Putamen, bilateral iMRI—yes |
Trial category: phase 1, safety Patients: 12 subjects, 2 cohorts Disease stage: earlier (< 5 years) and later (> 5 years, moderate–severe disease) Primary: safety tolerability over 5 years Secondary: UPDRS, nonmotor scores, and DAT scan changes over 18 months |