Parkinson's DiseasePhase 1NCT03562494
VY-AADC02 for Parkinson's Disease With Motor Fluctuations (RESTORE-1)
A Phase 1 study testing VY-AADC02 in people with Parkinson's Disease, run by Neurocrine Biosciences at 11 sites in United States.
Trial summary
The objective of this study is to assess the safety of VY-AADC02 in participants with Parkinson's disease (PD) with motor fluctuations. Read more about this study
- Who is it for?
- Ages 40 to 75 years
- Conditions the study is for
- Parkinson's Disease
- What is being tested?
- VY-AADC02
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 14 participants
- Where
- 11 sitesUnited States
- Study dates
- 17 Oct 2018to 30 Oct 2024
- Phase
- Phase 1
- 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 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Males and females, 40 to 75 years of age (inclusive)
- Diagnosis of PD, consistent with United Kingdom Brain Bank Criteria
- Motor responsiveness to dopaminergic therapy, demonstrated by improvement in Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS III score)
- Disease duration from diagnosis of ≥4 years
- An average of ≥3 hours of OFF time (that is, periods of insufficient control of motor PD symptoms) per day over 3 consecutive days as confirmed by the PD Diary
- A stable, optimal regimen of Parkinson's medications including levodopa for at least weeks prior to screening evaluation. Participants must have a minimum duration of levodopa treatment of ≥1 year
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- In the judgment of the Investigator, stable Parkinson's features and symptoms for at least 4 weeks prior to screening evaluation
- Agrees to defer any elective neurological surgery, including deep brain stimulation or ablation procedure for PD, levodopa or apomorphine infusion, or the addition of new dopaminergic formulations until after the study is completed, if medically appropriate
- Ability to travel to study visits
Who cannot join
- Atypical or secondary parkinsonism, including but not limited to symptoms believed to be due to trauma, brain tumor, infection, cerebrovascular disease, other neurological disease, or to drugs, chemicals, or toxins, as determined by the Investigator
- Montreal Cognitive Assessment (MoCA) score <26
- New or unstable psychiatric conditions (psychosis, depression) within 1 year of screening
- Brain imaging abnormalities in the striatum or other regions that would substantially increase risk of surgery
- Contraindication to magnetic resonance imaging (MRI) and/or gadolinium-based contrast agents
- Prior brain surgery, infusion therapies or planned treatments that could complicate the study procedure or negatively impact study evaluations as determined from participant interview, screening MRI, or medical records
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- History of malignancy other than treated carcinoma in situ within 3 years of screening evaluation
- Prior gene transfer, current treatment with any investigational agent (drug or device) within 2 months of screening evaluation, or participation or plans to participate in another research study
- Severe, biphasic and/or uncontrolled dyskinesia
- Disabling or uncontrolled impulse control disorders
Trial locations and contacts
11 sites in 1 country.
11 sites in 1 country
United States11 sites
Emory University Hospital
Atlanta, Georgia, 30322
University of Colorado
Aurora, Colorado, 80045
Northwestern Medical Faculty Foundation
Chicago, Illinois, 60611
Ohio State University Clinical Trials Management Office
Columbus, Ohio, 43210
UC Irvine
Irvine, California, 92697
University of Kansas Medical Center
Kansas City, Kansas, 66160
NYU Langone Medical Center
New York, New York, 10017
University of Philadelphia, Dept of Neurology
Philadelphia, Pennsylvania, 19107
University of Pittsburgh Medical Center (UPMC)
Pittsburgh, Pennsylvania, 15213
UC Davis Health System
Sacramento, California, 95817
University of California, San Francisco
San Francisco, California, 94143
About this study
Official title: A Randomized, Sham Surgery Controlled, Double-blind, Multi-center, Phase 1 Clinical Trial, Evaluating the Safety of VY-AADC02 in Moderate to Advanced Parkinson's Disease Patients With Motor Fluctuations
The objective of this study is to assess the safety of VY-AADC02 in participants with Parkinson's disease (PD) with motor fluctuations.
- Conditions
- Parkinson's Disease
- Being tested
- VY-AADC02, Sham (Placebo) Surgery
- Keywords
- Parkinson's Disease (PD), Aromatic L-Amino Acid Decarboxylase, AADC, AAV, VY-AADC02, AAV2-hAADC, DDC, Gene Therapy, Levodopa, Dopamine, Neurosurgery, NBIb-1817, Motor Fluctuations
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VY-AADC02Biological
Adeno-associated viral vector serotype 2 encoding human aromatic L-amino acid decarboxylase (AAV2- hAADC) infusion into the brain
Sham (Placebo) SurgeryOther
Bilateral partial burr/twist holes without dura penetration
| Study group | Role | What they receive |
|---|---|---|
| VY-AADC02 (NBIb-1817) | Experimental | Single administration of up to 3.6 x 10^12 vector genomes (vg) of VY-AADC02 Receives: Biological: VY-AADC02 |
| Sham (Placebo) Surgery | Placebo comparator | Sham surgical procedure Receives: Other: Sham (Placebo) Surgery |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment-emergent adverse events (TEAEs)
Measured: Up to 5 years after surgery
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jun 2018
- Study start17 Oct 2018
- Primary completion30 Oct 2024
- Study completion30 Oct 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)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
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Randomized, Sham Surgery Controlled, Double-blind, Multi-center
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Outcomes assessor
- 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
- NCT03562494
- Sponsor's protocol ID
- PD-1105
- Status last verified
- Dec 2024
- First submitted
- 16 May 2018
- Last updated
- 10 Dec 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
McFarthing K, Prakash N, Simuni T. CLINICAL TRIAL HIGHLIGHTS: 1. GENE THERAPY FOR PARKINSON'S, 2. PHASE 3 STUDY IN FOCUS - INTEC PHARMA'S ACCORDION PILL, 3. CLINICAL TRIALS RESOURCES. J Parkinsons Dis. 2019;9(2):251-264. doi: 10.3233/JPD-199001. No abstract available.
PubMed 31127735
Study at a glance
From the registry record, last updated by the sponsor on 10 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Sham Surgery Controlled, Double-blind, Multi-center, Phase 1 Clinical Trial, Evaluating the Safety of VY-AADC02 in Moderate to Advanced Parkinson's Disease Patients With Motor Fluctuations
- ClinicalTrials.gov ID
- NCT03562494
- Acronym / sponsor ID
- PD-1105
- Conditions
- Parkinson's Disease
- Keywords
- Parkinson's Disease (PD), Aromatic L-Amino Acid Decarboxylase, AADC, AAV, VY-AADC02, AAV2-hAADC, DDC, Gene Therapy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Neurocrine BiosciencesIndustry (pharma, biotech, devices)
- Collaborators
- Voyager Therapeutics
- Study start
- 17 Oct 2018 (actual)
- Primary completion
- 30 Oct 2024 (actual)
- Study completion
- 30 Oct 2024 (actual)
- First posted
- 19 Jun 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.
The objective of this study is to assess the safety of VY-AADC02 in participants with Parkinson's disease (PD) with motor fluctuations.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, outcomes assessor)
- Number of arms
- 2
- Model description
- Randomized, Sham Surgery Controlled, Double-blind, Multi-center
| Arm | Type | Receives |
|---|---|---|
| VY-AADC02 (NBIb-1817) | Experimental | Biological: VY-AADC02Single administration of up to 3.6 x 10^12 vector genomes (vg) of VY-AADC02 |
| Sham (Placebo) Surgery | Placebo comparator | Other: Sham (Placebo) SurgerySham surgical procedure |
Who may qualify
- Ages
- 40 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Males and females, 40 to 75 years of age (inclusive)
- Diagnosis of PD, consistent with United Kingdom Brain Bank Criteria
- Motor responsiveness to dopaminergic therapy, demonstrated by improvement in Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS III score)
- Disease duration from diagnosis of ≥4 years
- An average of ≥3 hours of OFF time (that is, periods of insufficient control of motor PD symptoms) per day over 3 consecutive days as confirmed by the PD Diary
Key exclusion criteria (10 listed)
- Atypical or secondary parkinsonism, including but not limited to symptoms believed to be due to trauma, brain tumor, infection, cerebrovascular disease, other neurological disease, or to drugs, chemicals, or toxins, as determined by the Investigator
- Montreal Cognitive Assessment (MoCA) score <26
- New or unstable psychiatric conditions (psychosis, depression) within 1 year of screening
- Brain imaging abnormalities in the striatum or other regions that would substantially increase risk of surgery
- Contraindication to magnetic resonance imaging (MRI) and/or gadolinium-based contrast agents
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VY-AADC02Adeno-associated viral vector serotype 2 encoding human aromatic L-amino acid decarboxylase (AAV2- hAADC) infusion into the brain | Biological | VY-AADC02 (NBIb-1817) |
| Sham (Placebo) SurgeryBilateral partial burr/twist holes without dura penetration | Other | Sham (Placebo) Surgery |
Primary outcome
Number of participants with treatment-emergent adverse events (TEAEs)
Time frame: Up to 5 years after surgery
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 10 Dec 2024.
- Derived from this study: PubMed 31127735McFarthing K, Prakash N, Simuni T. CLINICAL TRIAL HIGHLIGHTS: 1. GENE THERAPY FOR PARKINSON'S, 2. PHASE 3 STUDY IN FOCUS - INTEC PHARMA'S ACCORDION PILL, 3. CLINICAL TRIALS RESOURCES. J Parkinsons Dis. 2019;9(2):251-264. doi: 10.3233/JPD-199001. No abstract available.
- 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
11 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing VY-AADC02 in people with Parkinson's Disease, run by Neurocrine Biosciences at 11 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 9 inclusion and 10 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 10 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Number of participants with treatment-emergent adverse events (TEAEs) (time frame: Up to 5 years after surgery). 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 NCT03562494, which also lists 1 linked publication.
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 10 Dec 2024.