drugset / Trial / NCT01973543
Safety Study of AADC Gene Therapy (VY-AADC01) for Parkinson's Disease
Phase 1
Completed
15 enrolled
Neurocrine Biosciences
Feinstein Institute for Medical Research · collabOregon Health and Science University · collabUniversity of California, San Francisco · collabVeristat, Inc. · collabVoyager Therapeutics · collab
Non-randomizedSingle-groupSingle-blindTreatment
Summary
Safety study of AADC gene transfer (VY-AADC01) in subjects with Parkinson's disease.
Timeline
- Start
- 2013-10
- Primary completion
- 2020-01-24
- Completion
- 2020-01-24
Publications
- Christine CW, Richardson RM, Van Laar AD, Thompson ME, Fine EM, Khwaja OS, Li C, Liang GS, Meier A, Roberts EW, Pfau ML, Rodman JR, Bankiewicz KS, Larson PS. Safety of AADC Gene Therapy for Moderately Advanced Parkinson Disease: Three-Year Outcomes From the PD-1101 Trial. Neurology. 2022 Jan 4;98(1):e40-e50. doi: 10.1212/WNL.0000000000012952. Epub 2021 Oct 14.
- Christine CW, Bankiewicz KS, Van Laar AD, Richardson RM, Ravina B, Kells AP, Boot B, Martin AJ, Nutt J, Thompson ME, Larson PS. Magnetic resonance imaging-guided phase 1 trial of putaminal AADC gene therapy for Parkinson's disease. Ann Neurol. 2019 May;85(5):704-714. doi: 10.1002/ana.25450. Epub 2019 Mar 26.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | VY-AADC01 | Gene therapy (AAV / viral vector) | 7.5e+11 vg | Other |
| Subject | VY-AADC01 | Gene therapy (AAV / viral vector) | 1.5e+12 vg | Other |
| Subject | VY-AADC01 | Gene therapy (AAV / viral vector) | 4.7e+12 vg | Other |