Parkinson's DiseaseNCT03733496
Observational, Long-term Extension Study for Participants of Prior VY-AADC01 Studies
An observational study of Parkinson's Disease, run by Neurocrine Biosciences at 3 sites in United States.
Trial summary
An extension study for participants who have completed a prior VY-AADC01 clinical study Read more about this study
- Who is it for?
- Ages 40 years and older
- Conditions the study is for
- Parkinson's Disease
- How many people
- 14 participants
- Where
- 3 sitesUnited States
- Study dates
- 24 Aug 2018to 18 Jul 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 40 years and older
- All sexes
- Adult, Older adult
Study population: Participants from prior VY-AADC01 clinical studies will be eligible for the extension study
Who may be able to join
- 1. Completed participation in the PD-1101 or PD-1102 clinical study of VY-AADC01 gene therapy
Who cannot join
- None
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Emory University
Atlanta, Georgia, 30322
Ohio State University
Columbus, Ohio, 43210
University of California, San Francisco
San Francisco, California, 94143
About this study
Official title: 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
- 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
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability)
Long-term safety of VY-AADC01 as measured by Adverse Events and Serious Adverse Events
Measured: Up to 8 years from VY-AADC01 administration
Secondary outcomes (4)
Change in AADC enzyme activity
Continued expression of AADC as measured by [18F]-fluorodopa (F-Dopa) positron emission tomography (PET)
Measured: Up to 8 years from VY-AADC01 administration
Changes in PD medications
Change from baseline in PD medications, measured as levodopa equivalent dose
Measured: Up to 8 years from VY-AADC01 administration
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
Measured: 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
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
Measured: Up to 8 years from VY-AADC01 administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2018
- Study start24 Aug 2018
- Primary completion18 Jul 2023
- Study completion18 Jul 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Parkinson's DiseaseStudies of the same condition, open and under way.
- More trials run by Neurocrine BiosciencesThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Neurocrine Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Voyager Therapeutics
- Responsible party
- Sponsor
- Investigators
- Clinical Development Lead, Study director, Neurocrine Biosciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03733496
- Sponsor's protocol ID
- PD-1104
- Status last verified
- Dec 2023
- First submitted
- 19 Oct 2018
- Last updated
- 22 Dec 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 30802998Richardson 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.
PubMed 32732384Nutt 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.
PubMed 32149427
Study at a glance
From the registry record, last updated by the sponsor on 22 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Observational, Long-Term Extension Study for Participants in Prior VY-AADC01 Clinical Studies
- ClinicalTrials.gov ID
- NCT03733496
- Acronym / sponsor ID
- PD-1104
- Conditions
- Parkinson's Disease
- Keywords
- Parkinson's Disease (PD), Gene Therapy, Motor Fluctuations, Neurosurgery, NBIb-1817, Aromatic L-Amino Acid Decarboxylase, Levodopa, Dopamine
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Neurocrine BiosciencesIndustry (pharma, biotech, devices)
- Collaborators
- Voyager Therapeutics
- Study start
- 24 Aug 2018 (actual)
- Primary completion
- 18 Jul 2023 (actual)
- Study completion
- 18 Jul 2023 (actual)
- First posted
- 7 Nov 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
An extension study for participants who have completed a prior VY-AADC01 clinical study
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 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 |
Who may qualify
- Ages
- 40 years and older
- Sex
- All
- Age groups
- Adult, Older adult
- Study population
- Participants from prior VY-AADC01 clinical studies will be eligible for the extension study
Key inclusion criteria (1 listed)
- 1. Completed participation in the PD-1101 or PD-1102 clinical study of VY-AADC01 gene therapy
Key exclusion criteria (1 listed)
- None
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
4 secondary outcomes
Change in AADC enzyme activity
Time frame: Up to 8 years from VY-AADC01 administration
Changes in PD medications
Time frame: 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 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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Dec 2023.
- Background: PubMed 30802998Christine 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.
- Background: PubMed 32732384Richardson 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.
- Background: PubMed 32149427Nutt 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Development Lead study director
Neurocrine Biosciences
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Parkinson's Disease, run by Neurocrine Biosciences at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability) (time frame: Up to 8 years from VY-AADC01 administration). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03733496, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Dec 2023.