Viewing Study NCT05603312

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Ignite Creation Date: 2025-12-26 @ 10:46 PM
Ignite Modification Date: 2025-12-26 @ 10:46 PM
Study NCT ID: NCT05603312
Status: COMPLETED
Last Update Posted: 2025-09-05
First Post: 2022-10-12
Is NOT Gene Therapy: False
Has Adverse Events: True
Brief Title: A Double-blind Study to Evaluate the Safety of Glutamic Acid Decarboxylase Gene Transfer in Parkinson's Participants
Sponsor:
Organization:
Overview Dates Organization Conditions Design Enrollment Locations Outcomes Modules Raw JSON

Raw JSON

{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2025-08-21', 'type': 'ACTUAL'}}}}, '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'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'patients@meiragtx.com', 'phone': '646-860-7982', 'title': 'Program Manager', 'organization': 'MeiraGTx, LLC'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'From the signing of the Informed Consent Form to Week 26', 'description': 'Safety was evaluated by vital signs, neurological and physical examinations, electrocardiograms, radiographic imaging, and clinical laboratory tests.\n\nTreatment-related AEs were reported in 1 participant. This participant (high-dose group) experienced mild confusion and a mild headache perioperatively. These events started on the day of surgery and resolved the same day and 11 days later, respectively. All other AEs and all SAEs were assessed by the Investigator as unrelated to the study drug.', 'eventGroups': [{'id': 'EG000', 'title': 'AAV-GAD Low Dose', 'description': 'Bilateral infusion of AAV-GAD Low Dose into the STN', 'otherNumAtRisk': 5, 'deathsNumAtRisk': 5, 'otherNumAffected': 5, 'seriousNumAtRisk': 5, 'deathsNumAffected': 0, 'seriousNumAffected': 2}, {'id': 'EG001', 'title': 'AAV-GAD High Dose', 'description': 'Bilateral infusion of AAV-GAD High Dose into the STN', 'otherNumAtRisk': 5, 'deathsNumAtRisk': 5, 'otherNumAffected': 5, 'seriousNumAtRisk': 5, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG002', 'title': 'Sham Surgery', 'description': 'Sham infusion', 'otherNumAtRisk': 4, 'deathsNumAtRisk': 4, 'otherNumAffected': 4, 'seriousNumAtRisk': 4, 'deathsNumAffected': 0, 'seriousNumAffected': 1}], 'otherEvents': [{'term': 'Palpitations', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Eye swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Periorbital oedema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Vision blurred', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Dry mouth', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Gait disturbance', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Peripheral swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Acute sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Arthropod bite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Confusion postoperative', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Eye contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Computerised tomogram abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Haematocrit decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Red blood cells urine positive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Reflex test abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Muscle rigidity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Muscular weakness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Musculoskeletal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Torticollis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Balance disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Cognitive disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Dyskinesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Facial spasm', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Head discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hyperreflexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hypoaesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'On and off phenomenon', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Paraesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Anxiety', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Confusional state', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hallucination, visual', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Mental status changes', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Flushing', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Thrombophlebitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'seriousEvents': [{'term': 'Catheter site haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Freezing phenomenon', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Urinary retention', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Incidence of Adverse Events Related to the Treatment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}, {'value': '4', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'AAV-GAD Low Dose', 'description': 'Bilateral infusion of AAV-GAD Low Dose into the STN'}, {'id': 'OG001', 'title': 'AAV-GAD High Dose', 'description': 'Bilateral infusion of AAV-GAD High Dose into the STN'}, {'id': 'OG002', 'title': 'Sham Surgery', 'description': 'Sham infusion'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline to Week 26', 'description': 'The primary outcome measure is the safety of treatment with AAV-GAD, assessed by the absence of IMP-related treatment-emergent adverse events.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'PRIMARY', 'title': 'Incidence of Serious Adverse Events Related to the Treatment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}, {'value': '4', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'AAV-GAD Low Dose', 'description': 'Bilateral infusion of AAV-GAD Low Dose into the STN'}, {'id': 'OG001', 'title': 'AAV-GAD High Dose', 'description': 'Bilateral infusion of AAV-GAD High Dose into the STN'}, {'id': 'OG002', 'title': 'Sham Surgery', 'description': 'Sham infusion'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline to Week 26', 'description': 'The primary outcome measure is the safety of treatment with AAV-GAD, assessed by the absence of IMP-related serious treatment-emergent adverse events.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'AAV-GAD Low Dose', 'description': 'Bilateral infusion of AAV-GAD Low Dose into the STN'}, {'id': 'FG001', 'title': 'AAV-GAD High Dose', 'description': 'Bilateral infusion of AAV-GAD High Dose into the STN'}, {'id': 'FG002', 'title': 'Sham Surgery', 'description': 'Sham infusion'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '5'}, {'groupId': 'FG001', 'numSubjects': '5'}, {'groupId': 'FG002', 'numSubjects': '4'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '5'}, {'groupId': 'FG001', 'numSubjects': '5'}, {'groupId': 'FG002', 'numSubjects': '4'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'Participants were recruited from 6 medical centers in the United States (US). A total of 14 participants were enrolled in the study.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'BG000'}, {'value': '5', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}, {'value': '14', 'groupId': 'BG003'}]}], 'groups': [{'id': 'BG000', 'title': 'AAV-GAD Low Dose', 'description': 'Bilateral infusion of AAV-GAD Low Dose into the STN'}, {'id': 'BG001', 'title': 'AAV-GAD High Dose', 'description': 'Bilateral infusion of AAV-GAD High Dose into the STN'}, {'id': 'BG002', 'title': 'Sham Surgery', 'description': 'Sham infusion'}, {'id': 'BG003', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '5', 'groupId': 'BG003'}]}, {'title': '>=65 years', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '9', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '3', 'groupId': 'BG003'}]}, {'title': 'Male', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}, {'value': '11', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '5', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}, {'value': '14', 'groupId': 'BG003'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'White', 'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '5', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}, {'value': '14', 'groupId': 'BG003'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '5', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}, {'value': '14', 'groupId': 'BG003'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2023-09-22', 'size': 3905381, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2025-08-04T08:16', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'TRIPLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Participants are enrolled in one of three groups in parallel for the duration of the study.'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 14}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2022-10-05', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-09', 'completionDateStruct': {'date': '2024-09-06', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-09-02', 'studyFirstSubmitDate': '2022-10-12', 'resultsFirstSubmitDate': '2025-08-04', 'studyFirstSubmitQcDate': '2022-10-28', 'lastUpdatePostDateStruct': {'date': '2025-09-05', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2025-09-02', 'studyFirstPostDateStruct': {'date': '2022-11-02', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2025-09-05', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2024-09-06', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence of Adverse Events Related to the Treatment', 'timeFrame': 'Baseline to Week 26', 'description': 'The primary outcome measure is the safety of treatment with AAV-GAD, assessed by the absence of IMP-related treatment-emergent adverse events.'}, {'measure': 'Incidence of Serious Adverse Events Related to the Treatment', 'timeFrame': 'Baseline to Week 26', 'description': 'The primary outcome measure is the safety of treatment with AAV-GAD, assessed by the absence of IMP-related serious treatment-emergent adverse events.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isUnapprovedDevice': True, 'isFdaRegulatedDevice': True}, 'conditionsModule': {'conditions': ["Parkinson's Disease"]}, 'descriptionModule': {'briefSummary': "The objective of this clinical trial was to evaluate the safety and tolerability of adeno-associated virus (AAV)-mediated delivery of glutamic acid decarboxylase (GAD) gene transfer into the subthalamic nuclei (STN) of participants with Parkinson's Disease.", 'detailedDescription': 'The planned length of participation in the study for each participant was approximately 7 months, including a screening period of up to 40 days, randomization, surgery, and a follow-up period of 26 weeks.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '85 Years', 'minimumAge': '25 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Confirmed Parkinson\'s Disease\n* Levodopa responsiveness for at least 12 months\n* UPDRS Part 3 score of ≥25 points in the "off" state\n\nExclusion Criteria:\n\n* History of brain surgery to treat Parkinson\'s Disease\n* Any history of cerebral insult or central nervous system infection\n* Atypical Parkinson\'s Disease\n* Focal or lateralized neurologic deficits\n* Evidence of significant medical or psychiatric disorders\n* Cognitive impairment as defined by the Montreal Cognitive Assessment (MoCA) ≤ 20\n* Beck Depression Inventory-II score of ≥ 20'}, 'identificationModule': {'nctId': 'NCT05603312', 'briefTitle': "A Double-blind Study to Evaluate the Safety of Glutamic Acid Decarboxylase Gene Transfer in Parkinson's Participants", 'organization': {'class': 'INDUSTRY', 'fullName': 'MeiraGTx UK II Ltd'}, 'officialTitle': "A Randomized, Double-Blind, Sham-Controlled Study to Evaluate the Safety and Tolerability of Glutamic Acid Decarboxylase Gene Transfer to the Subthalamic Nuclei in Participants With Parkinson's Disease", 'orgStudyIdInfo': {'id': 'MGT-GAD-025'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'AAV-GAD Low Dose', 'description': 'Eligible participants received bilateral infusion of AAV-GAD low dose into the STN', 'interventionNames': ['Genetic: AAV-GAD Low Dose']}, {'type': 'EXPERIMENTAL', 'label': 'AAV-GAD High Dose', 'description': 'Eligible participants received bilateral infusion of AAV-GAD high dose into the STN', 'interventionNames': ['Genetic: AAV-GAD High Dose']}, {'type': 'SHAM_COMPARATOR', 'label': 'Sham Surgery', 'description': 'Eligible participants underwent a sham surgical procedure', 'interventionNames': ['Procedure: Sham Surgery']}], 'interventions': [{'name': 'AAV-GAD Low Dose', 'type': 'GENETIC', 'description': 'Bilateral infusion of AAV-GAD low dose', 'armGroupLabels': ['AAV-GAD Low Dose']}, {'name': 'AAV-GAD High Dose', 'type': 'GENETIC', 'description': 'Bilateral infusion of AAV-GAD high dose', 'armGroupLabels': ['AAV-GAD High Dose']}, {'name': 'Sham Surgery', 'type': 'PROCEDURE', 'description': 'Sham infusion', 'armGroupLabels': ['Sham Surgery']}]}, 'contactsLocationsModule': {'locations': [{'zip': '06510', 'city': 'New Haven', 'state': 'Connecticut', 'country': 'United States', 'facility': 'Yale University - Yale New Haven Hospital', 'geoPoint': {'lat': 41.30815, 'lon': -72.92816}}, {'zip': '48322', 'city': 'West Bloomfield', 'state': 'Michigan', 'country': 'United States', 'facility': 'Henry Ford West Bloomfield Hospital', 'geoPoint': {'lat': 42.56891, 'lon': -83.38356}}, {'zip': '10021', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Weill Cornell Medicine', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '10032', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Columbia University Irving Medical Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '19107', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Thomas Jefferson University', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '26506', 'city': 'Morgantown', 'state': 'West Virginia', 'country': 'United States', 'facility': 'West Virginia University', 'geoPoint': {'lat': 39.62953, 'lon': -79.9559}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'MeiraGTx, LLC', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}