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

An Observational, Long-Term Extension Study for Participants in Prior VY-AADC01 Clinical Studies

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT03733496

Enrollment

Registered

2018-11-07

Start date

2018-08-24

Completion date

2023-07-18

Last updated

2023-12-22

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Parkinson's Disease

Keywords

Parkinson's Disease (PD), Gene Therapy, Motor Fluctuations, Neurosurgery, NBIb-1817, Aromatic L-Amino Acid Decarboxylase, Levodopa, Dopamine, AADC, DDC, AAV2-hAADC, AAV, VY-AADC

Brief summary

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

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

40 Years to No maximum

Inclusion criteria

Key Inclusion Criteria: 1\. Completed participation in the PD-1101 or PD-1102 clinical study of VY-AADC01 gene therapy Key

Exclusion criteria

None

Design outcomes

Primary

Measure	Time frame	Description
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

Measure	Time frame	Description
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

Countries

United States

Outcome results