Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Voyager Therapeutics | INDUSTRY |
Not provided
Not provided
Not provided
Not provided
Not provided
An extension study for participants who have completed a prior VY-AADC01 clinical study
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Participants from VY-AADC01 clinical studies: | Participants who have completed participation in VY-AADC01 clinical studies (PD-1101 or PD-1102) will be invited to participate in this extension study |
Not provided
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability) | Long-term safety of VY-AADC01 as measured by Adverse Events and Serious Adverse Events | Up to 8 years from VY-AADC01 administration |
| Measure | Description | Time Frame |
|---|---|---|
| Change in AADC enzyme activity | Continued expression of AADC as measured by [18F]-fluorodopa (F-Dopa) positron emission tomography (PET) | Up to 8 years from VY-AADC01 administration |
| Changes in PD medications |
Not provided
Key Inclusion Criteria:
1. Completed participation in the PD-1101 or PD-1102 clinical study of VY-AADC01 gene therapy
Key Exclusion Criteria:
None
Not provided
Not provided
Not provided
Not provided
Participants from prior VY-AADC01 clinical studies will be eligible for the extension study
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Clinical Development Lead | Neurocrine Biosciences | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California, San Francisco | San Francisco | California | 94143 | United States | ||
| Emory University |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 30802998 | Background | 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. | |
| 32732384 | Background |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D010300 | Parkinson Disease |
| ID | Term |
|---|---|
| D020734 | Parkinsonian Disorders |
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
Not provided
Not provided
Not provided
Not provided
Not provided
Change from baseline in PD medications, measured as levodopa equivalent dose
| Up to 8 years from VY-AADC01 administration |
| Changes in motor function as assessed by Unified Parkinson's Disease Rating Scale (UPDRS) | Changes from baseline in motor function as assessed by UPDRS, in both "Off" and "On" medication states | Up to 8 years from VY-AADC01 administration |
| Changes in "Off" and "On" time as recorded by the participant in Parkinson's Disease (PD) diary | Changes from baseline based on the PD Diary: "OFF" time; "ON" time without troublesome dyskinesia; "ON" time without dyskinesia; "ON" time with troublesome dyskinesia; and "ON" time with non-troublesome dyskinesia | Up to 8 years from VY-AADC01 administration |
| Atlanta |
| Georgia |
| 30322 |
| United States |
| Ohio State University | Columbus | Ohio | 43210 | United States |
| Richardson RM, Bankiewicz KS, Christine CW, Van Laar AD, Gross RE, Lonser R, Factor SA, Kostyk SK, Kells AP, Ravina B, Larson PS. Data-driven evolution of neurosurgical gene therapy delivery in Parkinson's disease. J Neurol Neurosurg Psychiatry. 2020 Nov;91(11):1210-1218. doi: 10.1136/jnnp-2020-322904. Epub 2020 Jul 30. |
| 32149427 | Background | Nutt JG, Curtze C, Hiller A, Anderson S, Larson PS, Van Laar AD, Richardson RM, Thompson ME, Sedkov A, Leinonen M, Ravina B, Bankiewicz KS, Christine CW. Aromatic L-Amino Acid Decarboxylase Gene Therapy Enhances Levodopa Response in Parkinson's Disease. Mov Disord. 2020 May;35(5):851-858. doi: 10.1002/mds.27993. Epub 2020 Mar 9. |
| D009422 | Nervous System Diseases |
| D009069 | Movement Disorders |
| D000080874 | Synucleinopathies |
| D019636 | Neurodegenerative Diseases |