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Completed

NCT Number: NCT03733496

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

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

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Key information

Conditions

Parkinson's Disease Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Movement Disorders Nervous System Diseases Neurodegenerative Diseases Parkinson Disease Parkinsonian Disorders Synucleinopathies

Age range

40 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

University of California, San Francisco, San Francisco, California, United States

Loading trial locations.

Who can participate

Only the study team can determine whether someone qualifies for participation.

Key Inclusion Criteria:

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

Key Exclusion Criteria:

None

Treatment and study plan

Primary outcomes

  1. Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability)

    Time frame: Up to 8 years from VY-AADC01 administration

    Long-term safety of VY-AADC01 as measured by Adverse Events and Serious Adverse Events

Secondary outcomes

  1. Change in AADC enzyme activity

    Time frame: Up to 8 years from VY-AADC01 administration

    Continued expression of AADC as measured by [18F]-fluorodopa (F-Dopa) positron emission tomography (PET)

  2. Changes in PD medications

    Time frame: Up to 8 years from VY-AADC01 administration

    Change from baseline in PD medications, measured as levodopa equivalent dose

  3. Changes in motor function as assessed by Unified Parkinson's Disease Rating Scale (UPDRS)

    Time frame: 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

  4. Changes in "Off" and "On" time as recorded by the participant in Parkinson's Disease (PD) diary

    Time frame: 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

Sponsors and collaborators

Lead sponsor

Neurocrine Biosciences

Industry

Collaborators

  • Voyager Therapeutics

Registry information

Official study title

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

Important dates

Study start
2018
Primary completion
2023
Study completion
2023
First posted
Nov 7, 2018
Registry last updated
Dec 22, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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