Observational, Long-term Extension Study for Participants of Prior VY-AADC01 Studies

Completed

• Conditions: Parkinson's Disease

• Registration Number: NCT03733496
• Lead Sponsor: Neurocrine Biosciences

Brief Summary

An extension study for participants who have completed a prior VY-AADC01 clinical study

Detailed Description

Not available

Study Timeline

• Study Start: 2018-08-24
• Study First Submitted: 2018-10-19
• Study First Submitted QC: 2018-11-05
• Study First Posted: 2018-11-07
• Primary Completion: 2023-07-18
• Study Completion: 2023-07-18
• Status Verified: 2023-12
• Last Update Submitted: 2023-12-21
• Last Update Posted: 2023-12-22

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 14

Inclusion Criteria

1. Completed participation in the PD-1101 or PD-1102 clinical study of VY-AADC01 gene therapy

Key

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Exclusion Criteria

None

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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 "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
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 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

Trial Locations

Locations (3)

Emory University; 🇺🇸 Atlanta, Georgia, United States

University of California, San Francisco; 🇺🇸 San Francisco, California, United States

Ohio State University; 🇺🇸 Columbus, Ohio, United States