NCT03733496

Brief Summary

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

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
14

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Aug 2018

Longer than P75 for all trials

Geographic Reach
1 country

3 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 24, 2018

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

October 19, 2018

Completed
19 days until next milestone

First Posted

Study publicly available on registry

November 7, 2018

Completed
4.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 18, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 18, 2023

Completed
Last Updated

December 22, 2023

Status Verified

December 1, 2023

Enrollment Period

4.9 years

First QC Date

October 19, 2018

Last Update Submit

December 21, 2023

Conditions

Parkinson's Disease

Keywords

Parkinson's Disease (PD)Gene TherapyMotor FluctuationsNeurosurgeryNBIb-1817Aromatic L-Amino Acid DecarboxylaseLevodopaDopamineAADCDDCAAV2-hAADCAAVVY-AADC

Outcome Measures

Primary Outcomes (1)

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

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

    Up to 8 years from VY-AADC01 administration

Secondary Outcomes (4)

  • Change in AADC enzyme activity

    Up to 8 years from VY-AADC01 administration

  • Changes in PD medications

    Up to 8 years from VY-AADC01 administration

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

    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

    Up to 8 years from VY-AADC01 administration

Study Arms (1)

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

Eligibility Criteria

Age40 Years+
Sexall
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

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

You may qualify if:

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

You may not qualify if:

  • None

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

University of California, San Francisco

San Francisco, California, 94143, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

Ohio State University

Columbus, Ohio, 43210, United States

Location

Related Publications (3)

  • 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.

    PMID: 30802998BACKGROUND

  • 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.

    PMID: 32732384BACKGROUND

  • 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.

    PMID: 32149427BACKGROUND

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian DisordersBasal Ganglia DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMovement DisordersSynucleinopathiesNeurodegenerative Diseases

Study Officials

  • Clinical Development Lead

    Neurocrine Biosciences

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 19, 2018

First Posted

November 7, 2018

Study Start

August 24, 2018

Primary Completion

July 18, 2023

Study Completion

July 18, 2023

Last Updated

December 22, 2023

Record last verified: 2023-12

Data Sharing

IPD Sharing
Will not share

Locations

US(3)