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

Parkinson's Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03733496
• Other study ID #: PD-1104
• Status: Completed
• Start date: August 24, 2018
• Est. completion date: July 18, 2023

Study information

• Verified date: December 2023
• Source: Neurocrine Biosciences
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 14
• Est. completion date: July 18, 2023
• Est. primary completion date: July 18, 2023
• Gender: All
• Age group: 40 Years and older

Eligibility

Key Inclusion Criteria:

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

Key Exclusion Criteria:

None

Related Conditions & MeSH terms

• Parkinson Disease
• Parkinson's Disease

Locations

Country	Name	City	State
United States	Emory University	Atlanta	Georgia
United States	Ohio State University	Columbus	Ohio
United States	University of California, San Francisco	San Francisco	California

Sponsors (2)

Lead Sponsor	Collaborator
Neurocrine Biosciences	Voyager Therapeutics

Country where clinical trial is conducted

United States, 

References & 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. — View Citation

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. — View Citation

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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Change in AADC enzyme activity	Continued expression of AADC as measured by [18F]-fluorodopa (F-Dopa) positron emission tomography (PET)	Up to 8 years from VY-AADC01 administration
Secondary	Changes in PD medications	Change from baseline in PD medications, measured as levodopa equivalent dose	Up to 8 years from VY-AADC01 administration
Secondary	Changes in motor function as assessed by Unified Parkinson's Disease Rating Scale (UPDRS)	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
Secondary	Changes in "Off" and "On" time as recorded by the participant in Parkinson's Disease (PD) diary	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