Observational, Long-term Extension Study for Participants of Prior VY-AADC01 Studies
Sponsor
Neurocrine Biosciences (Industry)
Overall Status
Enrolling by invitation
CT.gov ID
NCT03733496
Collaborator
Voyager Therapeutics (Industry)
23
1
92.2
0.2
Study Details
Study Description
Brief Summary
An extension study for participants who have completed a prior VY-AADC01 clinical study
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
|
Study Design
Study Type:
Observational
Anticipated Enrollment
:
23 participants
Observational Model:
Cohort
Time Perspective:
Prospective
Official Title:
An Observational, Long-Term Extension Study for Participants in Prior VY-AADC01 Clinical Studies
Actual Study Start Date
:
Aug 24, 2018
Anticipated Primary Completion Date
:
Apr 1, 2026
Anticipated Study Completion Date
:
May 1, 2026
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| 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 |
Outcome Measures
Primary Outcome Measures
- Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability) [Up to 8 years from VY-AADC01 administration]
Long-term safety of VY-AADC01 as measured by Adverse Events and Serious Adverse Events
Secondary Outcome Measures
- Change in AADC enzyme activity [Up to 8 years from VY-AADC01 administration]
Continued expression of AADC as measured by [18F]-fluorodopa (F-Dopa) positron emission tomography (PET)
- Changes in PD medications [Up to 8 years from VY-AADC01 administration]
Change from baseline in PD medications, measured as levodopa equivalent dose
- Changes in motor function as assessed by Unified Parkinson's Disease Rating Scale (UPDRS) [Up to 8 years from VY-AADC01 administration]
Changes from baseline in motor function as assessed by UPDRS, in both "Off" and "On" medication states
- Changes in "Off" and "On" time as recorded by the participant in Parkinson's Disease (PD) diary [Up to 8 years from VY-AADC01 administration]
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
Eligibility Criteria
Criteria
Ages Eligible for Study:
40 Years
and Older
Sexes Eligible for Study:
All
Key Inclusion Criteria:
- Completed participation in the PD-1101 or PD-1102 clinical study of VY-AADC01 gene therapy
Key Exclusion Criteria:
None
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | University of California, San Francisco | San Francisco | California | United States | 94143 |
Sponsors and Collaborators
- Neurocrine Biosciences
- Voyager Therapeutics
Investigators
- Study Director: Clinical Development Lead, Neurocrine Biosciences
Study Documents (Full-Text)
None provided.More Information
Publications
- 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.
- 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.
- 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. Review.
Responsible Party:
Neurocrine Biosciences
ClinicalTrials.gov Identifier:
NCT03733496
Other Study ID Numbers:
- PD-1104
First Posted:
Nov 7, 2018
Last Update Posted:
Mar 21, 2022
Last Verified:
Mar 1, 2022
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Device Product:
No
Keywords provided by Neurocrine Biosciences
Additional relevant MeSH terms: