Clinical trial NCT03733496: Observational, Long-term Extension Study for Participants of Prior VY-AADC01 Studies
Official title: An Observational, Long-Term Extension Study for Participants in Prior VY-AADC01 Clinical Studies
Study type: OBSERVATIONAL
Overall status: COMPLETED
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
Lead sponsor: Neurocrine Biosciences
Sponsor class: INDUSTRY
Collaborators: name: Voyager Therapeutics
First posted: 2018-11-07
Enrollment: 14
Sex: ALL
Minimum age (years): 40
Standard ages: ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants from prior VY-AADC01 clinical studies will be eligible for the extension study

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


Eligibility 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

Primary outcomes:
measure: Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability)
timeFrame: Up to 8 years from VY-AADC01 administration
Secondary outcomes:
measure: Change in AADC enzyme activity
timeFrame: Up to 8 years from VY-AADC01 administration
measure: Changes in PD medications
timeFrame: Up to 8 years from VY-AADC01 administration
measure: Changes in motor function as assessed by Unified Parkinson's Disease Rating Scale (UPDRS)
timeFrame: Up to 8 years from VY-AADC01 administration
measure: Changes in "Off" and "On" time as recorded by the participant in Parkinson's Disease (PD) diary
timeFrame: Up to 8 years from VY-AADC01 administration
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: University of California, San Francisco
state: California
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Columbus
country: United States
facility: Ohio State University
state: Ohio
Study officials:
affiliation: Neurocrine Biosciences
name: Clinical Development Lead
role: STUDY_DIRECTOR
References:
citation: 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.
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.
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.