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

December 21, 2023 updated by: Neurocrine Biosciences

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

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

Study Overview

Status

Completed

Conditions

Study Type

Observational

Enrollment (Actual)

14

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • San Francisco, California, United States, 94143
        • University of California, San Francisco
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Emory University
    • Ohio
      • Columbus, Ohio, United States, 43210
        • Ohio State University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

40 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Sampling Method

Non-Probability Sample

Study Population

Participants from prior VY-AADC01 clinical studies will be eligible for the extension study

Description

Key Inclusion Criteria:

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

Key Exclusion Criteria:

None

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Up to 8 years from VY-AADC01 administration

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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)
Up to 8 years from VY-AADC01 administration
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
Up to 8 years from VY-AADC01 administration
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
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
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
Up to 8 years from VY-AADC01 administration

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Neurocrine Biosciences

Collaborators

Voyager Therapeutics

Investigators

  • Study Director: Clinical Development Lead, Neurocrine Biosciences

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 24, 2018

Primary Completion (Actual)

July 18, 2023

Study Completion (Actual)

July 18, 2023

Study Registration Dates

First Submitted

October 19, 2018

First Submitted That Met QC Criteria

November 5, 2018

First Posted (Actual)

November 7, 2018

Study Record Updates

Last Update Posted (Actual)

December 22, 2023

Last Update Submitted That Met QC Criteria

December 21, 2023

Last Verified

December 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PD-1104

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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