Ignite Creation Date:
2025-12-25 @ 1:34 AM
Ignite Modification Date:
2025-12-25 @ 11:48 PM
Study NCT ID:
NCT03562494
Status:
COMPLETED
Last Update Posted:
2024-12-10
First Post:
2018-05-16
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
VY-AADC02 for Parkinson's Disease With Motor Fluctuations (RESTORE-1)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D010300', 'term': 'Parkinson Disease'}], 'ancestors': [{'id': 'D020734', 'term': 'Parkinsonian Disorders'}, {'id': 'D001480', 'term': 'Basal Ganglia Diseases'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D009069', 'term': 'Movement Disorders'}, {'id': 'D000080874', 'term': 'Synucleinopathies'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C005703', 'term': 'salicylhydroxamic acid'}, {'id': 'D013514', 'term': 'Surgical Procedures, Operative'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'TRIPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Randomized, Sham Surgery Controlled, Double-blind, Multi-center'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 14}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2018-10-17', 'type': 'ACTUAL'}, 'statusVerifiedDate': '2024-12', 'completionDateStruct': {'date': '2024-10-30', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-12-05', 'studyFirstSubmitDate': '2018-05-16', 'studyFirstSubmitQcDate': '2018-06-07', 'lastUpdatePostDateStruct': {'date': '2024-12-10', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2018-06-19', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2024-10-30', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of participants with treatment-emergent adverse events (TEAEs)', 'timeFrame': 'Up to 5 years after surgery'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'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'], 'conditions': ["Parkinson's Disease"]}, 'referencesModule': {'references': [{'pmid': '31127735', 'type': 'DERIVED', 'citation': "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."}]}, 'descriptionModule': {'briefSummary': "The objective of this study is to assess the safety of VY-AADC02 in participants with Parkinson's disease (PD) with motor fluctuations."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '75 Years', 'minimumAge': '40 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Key Inclusion Criteria:\n\n1. Males and females, 40 to 75 years of age (inclusive)\n2. Diagnosis of PD, consistent with United Kingdom Brain Bank Criteria\n3. Motor responsiveness to dopaminergic therapy, demonstrated by improvement in Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS III score)\n4. Disease duration from diagnosis of ≥4 years\n5. 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\n6. 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\n7. In the judgment of the Investigator, stable Parkinson's features and symptoms for at least 4 weeks prior to screening evaluation\n8. 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\n9. Ability to travel to study visits\n\nKey Exclusion Criteria:\n\n1. 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\n2. Montreal Cognitive Assessment (MoCA) score \\<26\n3. New or unstable psychiatric conditions (psychosis, depression) within 1 year of screening\n4. Brain imaging abnormalities in the striatum or other regions that would substantially increase risk of surgery\n5. Contraindication to magnetic resonance imaging (MRI) and/or gadolinium-based contrast agents\n6. 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\n7. History of malignancy other than treated carcinoma in situ within 3 years of screening evaluation\n8. 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\n9. Severe, biphasic and/or uncontrolled dyskinesia\n10. Disabling or uncontrolled impulse control disorders"}, 'identificationModule': {'nctId': 'NCT03562494', 'briefTitle': "VY-AADC02 for Parkinson's Disease With Motor Fluctuations (RESTORE-1)", 'organization': {'class': 'INDUSTRY', 'fullName': 'Neurocrine Biosciences'}, 'officialTitle': "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", 'orgStudyIdInfo': {'id': 'PD-1105'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'VY-AADC02 (NBIb-1817)', 'description': 'Single administration of up to 3.6 x 10\\^12 vector genomes (vg) of VY-AADC02', 'interventionNames': ['Biological: VY-AADC02']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Sham (Placebo) Surgery', 'description': 'Sham surgical procedure', 'interventionNames': ['Other: Sham (Placebo) Surgery']}], 'interventions': [{'name': 'VY-AADC02', 'type': 'BIOLOGICAL', 'description': 'Adeno-associated viral vector serotype 2 encoding human aromatic L-amino acid decarboxylase (AAV2- hAADC) infusion into the brain', 'armGroupLabels': ['VY-AADC02 (NBIb-1817)']}, {'name': 'Sham (Placebo) Surgery', 'type': 'OTHER', 'description': 'Bilateral partial burr/twist holes without dura penetration', 'armGroupLabels': ['Sham (Placebo) Surgery']}]}, 'contactsLocationsModule': {'locations': [{'zip': '92697', 'city': 'Irvine', 'state': 'California', 'country': 'United States', 'facility': 'UC Irvine', 'geoPoint': {'lat': 33.66946, 'lon': -117.82311}}, {'zip': '95817', 'city': 'Sacramento', 'state': 'California', 'country': 'United States', 'facility': 'UC Davis Health System', 'geoPoint': {'lat': 38.58157, 'lon': -121.4944}}, {'zip': '94143', 'city': 'San Francisco', 'state': 'California', 'country': 'United States', 'facility': 'University of California, San Francisco', 'geoPoint': {'lat': 37.77493, 'lon': -122.41942}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': 'University of Colorado', 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '30322', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': 'Emory University Hospital', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'Northwestern Medical Faculty Foundation', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '66160', 'city': 'Kansas City', 'state': 'Kansas', 'country': 'United States', 'facility': 'University of Kansas Medical Center', 'geoPoint': {'lat': 39.11417, 'lon': -94.62746}}, {'zip': '10017', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'NYU Langone Medical Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '43210', 'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'facility': 'Ohio State University Clinical Trials Management Office', 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '19107', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'University of Philadelphia, Dept of Neurology', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '15213', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'University of Pittsburgh Medical Center (UPMC)', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}], 'overallOfficials': [{'name': 'Clinical Development Lead', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Neurocrine Biosciences'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Neurocrine Biosciences', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Voyager Therapeutics', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}