Ignite Creation Date:
2025-12-24 @ 7:32 PM
Ignite Modification Date:
2026-01-01 @ 10:01 PM
Study NCT ID:
NCT02462603
Status:
COMPLETED
Last Update Posted:
2022-05-03
First Post:
2015-05-20
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Safety and Biomarker Study of PTC-589 in Participants With Parkinson's Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': True, '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'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'medinfo@ptcbio.com', 'phone': '1-866-562-4620', 'title': 'Patient Advocacy', 'organization': 'PTC Therapeutics, Inc.'}, 'certainAgreement': {'otherDetails': 'The Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the sponsor for review. The sponsor may consult with the PI to require changes to the communication or extend the embargo.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Baseline up to 30 days after last dose of study drug (up to 4 months)', 'description': 'Safety population included any participant who received at least 1 dose of PTC589.', 'eventGroups': [{'id': 'EG000', 'title': 'PTC589', 'description': "Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues.", 'otherNumAtRisk': 41, 'deathsNumAtRisk': 41, 'otherNumAffected': 29, 'seriousNumAtRisk': 41, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 7}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Post lumbar puncture syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 3}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 5}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 3}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 4}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 8}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Abnormal sensation in eye', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Dry eye', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Eye pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Ocular discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Ocular hyperaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Dyspepsia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Eructation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Flatulence', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Salivary hypersecretion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Asthenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Malaise', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Localised infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Respiratory tract infection viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Skin infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Facial bones fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Muscle strain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Aspartate aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Blood cholesterol increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Low density lipoprotein increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Diabetes mellitus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Gout', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Muscle spasms', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Muscular weakness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Musculoskeletal stiffness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Myalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Plantar fasciitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Tendonitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Bradykinesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Dyskinesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Dystonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Hypoaesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Hypogeusia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Peroneal nerve palsy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Tremor', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Depressed mood', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Depression', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Insomnia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Nightmare', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Pollakiuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Laryngeal inflammation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}, {'term': 'Hyperhidrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.0'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants With Drug-Related Serious Adverse Events (SAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '41', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'PTC589', 'description': "Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues."}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to 30 days after last dose of study drug (up to 4 months)', 'description': "An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.", 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population included any participant who received at least 1 dose of PTC589.'}, {'type': 'SECONDARY', 'title': "Change From Baseline in Movement Disorder Society Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Score at Month 3", 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'PTC589', 'description': "Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues."}], 'classes': [{'title': 'nM-EDL Total Score: Baseline', 'categories': [{'measurements': [{'value': '5.0', 'spread': '4.51', 'groupId': 'OG000'}]}]}, {'title': 'nM-EDL Total Score: Change at Month 3', 'categories': [{'measurements': [{'value': '-0.1', 'spread': '3.93', 'groupId': 'OG000'}]}]}, {'title': 'M-EDL Total Score: Baseline', 'categories': [{'measurements': [{'value': '5.8', 'spread': '4.13', 'groupId': 'OG000'}]}]}, {'title': 'M-EDL Total Score: Change at Month 3', 'categories': [{'measurements': [{'value': '0.1', 'spread': '3.54', 'groupId': 'OG000'}]}]}, {'title': 'Motor Examination Total Score: Baseline', 'categories': [{'measurements': [{'value': '22.5', 'spread': '8.60', 'groupId': 'OG000'}]}]}, {'title': 'Motor Examination Total Score: Change at Month 3', 'categories': [{'measurements': [{'value': '-0.6', 'spread': '6.72', 'groupId': 'OG000'}]}]}, {'title': 'Motor Complications Total Score: Baseline', 'categories': [{'measurements': [{'value': '1.3', 'spread': '2.34', 'groupId': 'OG000'}]}]}, {'title': 'Motor Complications Total Score: Change at Month 3', 'categories': [{'measurements': [{'value': '-0.1', 'spread': '2.17', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Month 3', 'description': "The MDS-UPDRS is a tool for monitoring the impact of Parkinson's disease, the degree of disability caused, and complications from treatment. Part I (13 items) evaluates nonmotor experiences of daily living (nM-EDL); Part II (13 items) evaluates motor experiences of daily living (M-EDL; Part III (18 items) is a motor examination; Part IV (6 items) examines motor complications (for example, motor fluctuations and dyskinesias). Each item was rated on a 5-point scale, ranging from 0 (normal) to 4 (severe), with higher score indicating greater severity and more impairment. Total score for Part I (nM-EDL) and Part II (M-EDL) each ranges from 0-52; for Part III (motor examination) ranges from 0-72; and for Part IV (motor complications) ranges from 0-24; with higher scores in each range for all 4 parts reflecting greater severity.", 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Efficacy intent-to-treat (EITT) population included any participant who received at least 1 dose of PTC589 and had a minimum of the Month 3 assessment.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Non-motor Symptoms Scale (NMSS) Total Score at Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'PTC589', 'description': "Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues."}], 'classes': [{'title': 'Baseline', 'categories': [{'measurements': [{'value': '15.7', 'spread': '14.84', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 3', 'categories': [{'measurements': [{'value': '-0.6', 'spread': '14.79', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Month 3', 'description': 'Non-motor symptoms were evaluated using the NMSS which was divided into 30 questions in 9 different domains including such symptoms as dribbling saliva, constipation, depression, sleep disorders, apathy, hallucinations and dementia. Symptoms were quantified based on their severity (using a scale of 0 \\[none\\] to 3 \\[severe\\]) and frequency (using a scale of 0 \\[rarely\\] to 4 \\[very frequent\\]). Total score derived from adding up the product of the frequency score times severity score for each of the 30 questions. Total score ranged from 0 to 360, with a lower score indicating fewer symptoms.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'EITT population included any participant who received at least 1 dose of PTC589 and had a minimum of the Month 3 assessment.'}, {'type': 'SECONDARY', 'title': "Change From Baseline in Parkinson's Disease Questionnaire - 39 (PDQ-39) Score at Month 3", 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'PTC589', 'description': "Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues."}], 'classes': [{'title': 'Mobility: Baseline', 'categories': [{'measurements': [{'value': '7.06', 'spread': '12.543', 'groupId': 'OG000'}]}]}, {'title': 'Mobility: Change at Month 3', 'categories': [{'measurements': [{'value': '0.19', 'spread': '7.281', 'groupId': 'OG000'}]}]}, {'title': 'Activities of daily living: Baseline', 'categories': [{'measurements': [{'value': '11.56', 'spread': '10.096', 'groupId': 'OG000'}]}]}, {'title': 'Activities of daily living: Change at Month 3', 'categories': [{'measurements': [{'value': '0.21', 'spread': '10.676', 'groupId': 'OG000'}]}]}, {'title': 'Emotional well-being: Baseline', 'categories': [{'measurements': [{'value': '10.84', 'spread': '11.387', 'groupId': 'OG000'}]}]}, {'title': 'Emotional well-being: Change at Month 3', 'categories': [{'measurements': [{'value': '0.10', 'spread': '10.180', 'groupId': 'OG000'}]}]}, {'title': 'Stigma: Baseline', 'categories': [{'measurements': [{'value': '15.96', 'spread': '17.685', 'groupId': 'OG000'}]}]}, {'title': 'Stigma: Change at Month 3', 'categories': [{'measurements': [{'value': '-0.94', 'spread': '16.602', 'groupId': 'OG000'}]}]}, {'title': 'Social support: Baseline', 'categories': [{'measurements': [{'value': '1.88', 'spread': '4.811', 'groupId': 'OG000'}]}]}, {'title': 'Social support: Change at Month 3', 'categories': [{'measurements': [{'value': '3.12', 'spread': '9.750', 'groupId': 'OG000'}]}]}, {'title': 'Cognition: Baseline', 'categories': [{'measurements': [{'value': '9.70', 'spread': '12.085', 'groupId': 'OG000'}]}]}, {'title': 'Cognition: Change at Month 3', 'categories': [{'measurements': [{'value': '-1.24', 'spread': '8.747', 'groupId': 'OG000'}]}]}, {'title': 'Communication: Baseline', 'categories': [{'measurements': [{'value': '6.25', 'spread': '9.388', 'groupId': 'OG000'}]}]}, {'title': 'Communication: Change at Month 3', 'categories': [{'measurements': [{'value': '1.25', 'spread': '10.777', 'groupId': 'OG000'}]}]}, {'title': 'Bodily discomfort: Baseline', 'categories': [{'measurements': [{'value': '16.87', 'spread': '15.390', 'groupId': 'OG000'}]}]}, {'title': 'Bodily discomfort: Change at Month 3', 'categories': [{'measurements': [{'value': '0.01', 'spread': '14.501', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Month 3', 'description': "The PDQ-39 is a self-administered questionnaire for participants with Parkinson's disease that has 39 questions grouped in 8 dimensions: mobility (items 1-10), activities of daily living (items 11-16), emotional well-being (items 17-22), stigma (items 23-26), social support (items 27-29), cognitions (items 30-33), communication (items 34-36), and bodily discomfort (items 37-39). Each item was scored on a 5-point Likert scale (0 to 4) to indicate the frequency of each event; 0 = never, 1 = occasionally, 2 = sometimes, 3 = often, and 4 = always or cannot do at all. Each dimension's total score ranged from 0-100, with lower scores indicating better health, and higher scores indicating more severe symptoms.", 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'EITT population included any participant who received at least 1 dose of PTC589 and had a minimum of the Month 3 assessment.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in EuroQol-5 Dimension (EQ-5D) Score at Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'PTC589', 'description': "Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues."}], 'classes': [{'title': 'Mobility: Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.2', 'spread': '0.36', 'groupId': 'OG000'}]}]}, {'title': 'Mobility: Change at Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'spread': '0.36', 'groupId': 'OG000'}]}]}, {'title': 'Personal Care: Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.1', 'spread': '0.27', 'groupId': 'OG000'}]}]}, {'title': 'Personal Care: Change at Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'spread': '0.36', 'groupId': 'OG000'}]}]}, {'title': 'Usual Activities: Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.2', 'spread': '0.42', 'groupId': 'OG000'}]}]}, {'title': 'Usual Activities: Change at Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.1', 'spread': '0.35', 'groupId': 'OG000'}]}]}, {'title': 'Pain/Discomfort: Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.3', 'spread': '0.47', 'groupId': 'OG000'}]}]}, {'title': 'Pain/Discomfort: Change at Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.2', 'spread': '0.48', 'groupId': 'OG000'}]}]}, {'title': 'Anxiety/Depression: Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.1', 'spread': '0.27', 'groupId': 'OG000'}]}]}, {'title': 'Anxiety/Depression: Change at Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.2', 'spread': '0.50', 'groupId': 'OG000'}]}]}, {'title': 'VAS Score: Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '81.0', 'spread': '12.00', 'groupId': 'OG000'}]}]}, {'title': 'VAS Score: Change at Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '39', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-1.9', 'spread': '8.68', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Month 3', 'description': "EQ-5D is a questionnaire designed to provide measures of health-related quality of life states, consisting of 5 domains (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). Each dimension has a 3 point response scale designed to indicate the level of the problem: 1 = no problems, 2 = some problems, 3 = extreme problems. A higher score indicated an increase in the level of problem. The EQ-5D also contains a visual analog scale (EQ-VAS), which records the respondent's self-rated health status on a vertical graduated visual analog scale ranging from 0 (worst imaginable health state) to 100 (best imaginable health state). Higher score indicated improvement.", 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'EITT population included any participant who received at least 1 dose of PTC589 and had a minimum of the Month 3 assessment.'}, {'type': 'SECONDARY', 'title': 'Montreal Cognitive Assessment (MoCA) Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'PTC589', 'description': "Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues."}], 'classes': [{'title': 'Normal global cognition', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '28.5', 'spread': '1.25', 'groupId': 'OG000'}]}]}, {'title': 'Mild cognitive impairment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '23.4', 'spread': '1.40', 'groupId': 'OG000'}]}]}, {'title': 'Moderate cognitive impairment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Severe cognitive impairment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Month 3', 'description': 'MoCA is a 30-point questionnaire for cognitive dysfunction. It assesses different cognitive domains: attention and concentration, executive functions, memory, language, visuoconstructional skills, conceptual thinking, calculations, and orientation. Scores on the MoCA range from 0-30 with 26-30 indicating normal global cognition; 18-25 mild cognitive impairment; 10-17 moderate cognitive impairment; and \\<10 severe cognitive impairment.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "EITT population included any participant who received at least 1 dose of PTC589 and had a minimum of the Month 3 assessment. Here, 'Number analyzed' signifies participants with normal, mild, moderate, or severe cognition impairment. Data for a specific severity level was not collected if no evaluable participants were available."}, {'type': 'SECONDARY', 'title': 'Beck Depression Inventory (BDI) Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'PTC589', 'description': "Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues."}], 'classes': [{'title': 'Normal', 'denoms': [{'units': 'Participants', 'counts': [{'value': '24', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.6', 'spread': '2.41', 'groupId': 'OG000'}]}]}, {'title': 'Mild mood disturbance', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '13.1', 'spread': '1.83', 'groupId': 'OG000'}]}]}, {'title': 'Borderline clinical depression', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Moderate depression', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Severe depression', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Extreme depression', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Month 3', 'description': 'The BDI is a self-reporting 21-item scoring tool that measures characteristic attitudes and symptoms of depression, including physical symptoms. Each of the 21 items on BDI tool represent a depressive symptom. The symptoms are each scored on a 4-point Likert scale of 0 (symptom is absent) to 3 (symptom is severe). Scores for each symptom are added up to obtain the total scores for all 21 items, which are interpreted as follows 1-10 (normal); 11-16 (mild mood disturbance); 17-20 (borderline clinical depression); 21-30 (moderate depression); 31-40 (severe depression); and \\>40 (extreme depression). Participants with symptom score of 0 were not included in the summary.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "EITT population included any participant who received at least 1 dose of PTC589 and had a minimum of Month 3 assessment. 'Number analyzed' = participants with symptom score \\>1 for normal, mild, borderline, moderate, severe, or extreme depression. Data for a specific severity level was not collected if no evaluable participants were available."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Montgomery and Asberg Depression Rating Scale (MADRS) Total Score at Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'PTC589', 'description': "Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues."}], 'classes': [{'title': 'Baseline', 'categories': [{'measurements': [{'value': '2.4', 'spread': '2.60', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 3', 'categories': [{'measurements': [{'value': '0.1', 'spread': '3.00', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Month 3', 'description': 'The MADRS is a clinician-rated tool for measuring changes in depressive symptom severity. Ten core symptoms and cognitive features (feelings of sadness, lassitude, pessimism, inner tension, suicidality, reduced sleep or appetite, difficulty concentrating, and a lack of interest) were rated on a severity scale of 0 (no symptoms)) to 6 (symptoms of maximum severity). The total score was the sum of the scores on the 10 items, ranging from 0 to 60 with a higher score indicating increasing depressive symptoms.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'EITT population included any participant who received at least 1 dose of PTC589 and had a minimum of the Month 3 assessment.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Time to Complete Time Up and Go (TUG) Test in ON State at Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'PTC589', 'description': "Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues."}], 'classes': [{'title': 'Baseline', 'categories': [{'measurements': [{'value': '8.637', 'spread': '1.8763', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 3', 'categories': [{'measurements': [{'value': '-0.347', 'spread': '1.1056', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Month 3', 'description': "Timed motor tests are simple, objective, quantitative measures for the assessment of Parkinson's disease. They include, in on-medication and off-medication state, timed recorded physical movements. Time Up and Go Test (TUG) is one of timed motor tests which is used to assess a person's mobility and requires both static and dynamic balance. This is a walking assessment. Participants start in the seated position, stand up, walk 7 meters, turn around, and sit back down. The entire process from leaving the chair to returning to the chair was timed. The total time was summarized under ON state with participants on dopamine therapy.", 'unitOfMeasure': 'seconds', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "EITT population included any participant who received at least 1 dose of PTC589 and had a minimum of the Month 3 assessment. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure."}, {'type': 'SECONDARY', 'title': 'Maximum Observed Plasma Concentration (Cmax) of PTC589', 'denoms': [{'units': 'Participants', 'counts': [{'value': '23', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'PTC589', 'description': "Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues."}], 'classes': [{'title': 'Month 1', 'categories': [{'measurements': [{'value': '3718.3', 'spread': '311.6', 'groupId': 'OG000'}]}]}, {'title': 'Month 3', 'categories': [{'measurements': [{'value': '2903.5', 'spread': '249.0', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '0 hour (predose) and 0.5, 1, 2, 4, 6, 8, and 12 hours postdose at Month 1 and 3', 'unitOfMeasure': 'nanograms (ng)/milliliter (mL)', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': "EITT population included any participant who received at least 1 dose of PTC589 and had a minimum of the Month 3 assessment. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure."}, {'type': 'SECONDARY', 'title': 'Level of Disease-Related Biomarker (Glutathione) in Plasma', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'PTC589', 'description': "Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues."}], 'classes': [{'categories': [{'measurements': [{'value': '1.54', 'groupId': 'OG000', 'lowerLimit': '0.37', 'upperLimit': '8.33'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Month 3', 'description': 'Glutathione lowest limit of quantification (LLOQ) = 0.01 micromoles (uM) and upper limit of quantification (ULOQ) = 27.83 uM in plasma.', 'unitOfMeasure': 'µM', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'EITT population included any participant who received at least 1 dose of EPI-589.'}, {'type': 'SECONDARY', 'title': 'Level of Disease-Related Biomarker (Glutathione) in Cerebrospinal Fluid (CSF)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '36', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'PTC589', 'description': "Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues."}], 'classes': [{'categories': [{'measurements': [{'value': '0.10', 'groupId': 'OG000', 'lowerLimit': '0.04', 'upperLimit': '0.23'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Month 3', 'description': 'Glutathione LLOQ = 0.002 uM and ULOQ = 0.35 uM in CSF.', 'unitOfMeasure': 'µM', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': "EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure."}, {'type': 'SECONDARY', 'title': 'Level of Disease-Related Biomarker (Glutathione) in Urine', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'PTC589', 'description': "Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues."}], 'classes': [{'categories': [{'measurements': [{'value': '0.0000061', 'groupId': 'OG000', 'lowerLimit': '0.00000034', 'upperLimit': '0.000033'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Month 3', 'description': 'Glutathione LLOQ = 0.01 uM, and ULOQ = 1.39 uM in urine.', 'unitOfMeasure': 'µM', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'EITT population included any participant who received at least 1 dose of EPI-589.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'PTC589', 'description': "Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 milligrams (mg) (2 tablets of 250 mg each) orally twice daily (BID) for up to 3 months unless discontinued for safety or tolerability issues."}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '44'}]}, {'type': 'Received at Least 1 Dose of Study Drug', 'achievements': [{'groupId': 'FG000', 'numSubjects': '41'}]}, {'type': 'EITT Population', 'comment': 'participant who received at least 1 dose of PTC589 and had a minimum of Month 3 assessment.', 'achievements': [{'groupId': 'FG000', 'numSubjects': '40'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '40'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '4'}]}], 'dropWithdraws': [{'type': 'Investigator Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Non-Compliance', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '41', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'PTC589', 'description': "Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) received PTC589 at a dose of 500 mg (2 tablets of 250 mg each) orally BID for up to 3 months unless discontinued for safety or tolerability issues."}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '59.2', 'spread': '8.21', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '8', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '33', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '39', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '40', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'Safety population included any participant who received at least 1 dose of PTC589.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2017-09-29', 'size': 2772638, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2022-04-06T15:37', 'hasProtocol': True}, {'date': '2018-08-03', 'size': 1430954, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2022-04-06T15:40', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 44}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2016-05-17', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-04', 'dispFirstSubmitDate': '2020-05-19', 'completionDateStruct': {'date': '2019-01-08', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2022-04-06', 'studyFirstSubmitDate': '2015-05-20', 'dispFirstSubmitQcDate': '2020-05-19', 'resultsFirstSubmitDate': '2022-04-06', 'studyFirstSubmitQcDate': '2015-06-02', 'dispFirstPostDateStruct': {'date': '2020-05-21', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2022-05-03', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2022-04-06', 'studyFirstPostDateStruct': {'date': '2015-06-04', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2022-05-03', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-01-08', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants With Drug-Related Serious Adverse Events (SAEs)', 'timeFrame': 'Baseline up to 30 days after last dose of study drug (up to 4 months)', 'description': "An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'."}], 'secondaryOutcomes': [{'measure': "Change From Baseline in Movement Disorder Society Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Score at Month 3", 'timeFrame': 'Baseline, Month 3', 'description': "The MDS-UPDRS is a tool for monitoring the impact of Parkinson's disease, the degree of disability caused, and complications from treatment. Part I (13 items) evaluates nonmotor experiences of daily living (nM-EDL); Part II (13 items) evaluates motor experiences of daily living (M-EDL; Part III (18 items) is a motor examination; Part IV (6 items) examines motor complications (for example, motor fluctuations and dyskinesias). Each item was rated on a 5-point scale, ranging from 0 (normal) to 4 (severe), with higher score indicating greater severity and more impairment. Total score for Part I (nM-EDL) and Part II (M-EDL) each ranges from 0-52; for Part III (motor examination) ranges from 0-72; and for Part IV (motor complications) ranges from 0-24; with higher scores in each range for all 4 parts reflecting greater severity."}, {'measure': 'Change From Baseline in Non-motor Symptoms Scale (NMSS) Total Score at Month 3', 'timeFrame': 'Baseline, Month 3', 'description': 'Non-motor symptoms were evaluated using the NMSS which was divided into 30 questions in 9 different domains including such symptoms as dribbling saliva, constipation, depression, sleep disorders, apathy, hallucinations and dementia. Symptoms were quantified based on their severity (using a scale of 0 \\[none\\] to 3 \\[severe\\]) and frequency (using a scale of 0 \\[rarely\\] to 4 \\[very frequent\\]). Total score derived from adding up the product of the frequency score times severity score for each of the 30 questions. Total score ranged from 0 to 360, with a lower score indicating fewer symptoms.'}, {'measure': "Change From Baseline in Parkinson's Disease Questionnaire - 39 (PDQ-39) Score at Month 3", 'timeFrame': 'Baseline, Month 3', 'description': "The PDQ-39 is a self-administered questionnaire for participants with Parkinson's disease that has 39 questions grouped in 8 dimensions: mobility (items 1-10), activities of daily living (items 11-16), emotional well-being (items 17-22), stigma (items 23-26), social support (items 27-29), cognitions (items 30-33), communication (items 34-36), and bodily discomfort (items 37-39). Each item was scored on a 5-point Likert scale (0 to 4) to indicate the frequency of each event; 0 = never, 1 = occasionally, 2 = sometimes, 3 = often, and 4 = always or cannot do at all. Each dimension's total score ranged from 0-100, with lower scores indicating better health, and higher scores indicating more severe symptoms."}, {'measure': 'Change From Baseline in EuroQol-5 Dimension (EQ-5D) Score at Month 3', 'timeFrame': 'Baseline, Month 3', 'description': "EQ-5D is a questionnaire designed to provide measures of health-related quality of life states, consisting of 5 domains (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). Each dimension has a 3 point response scale designed to indicate the level of the problem: 1 = no problems, 2 = some problems, 3 = extreme problems. A higher score indicated an increase in the level of problem. The EQ-5D also contains a visual analog scale (EQ-VAS), which records the respondent's self-rated health status on a vertical graduated visual analog scale ranging from 0 (worst imaginable health state) to 100 (best imaginable health state). Higher score indicated improvement."}, {'measure': 'Montreal Cognitive Assessment (MoCA) Score', 'timeFrame': 'Month 3', 'description': 'MoCA is a 30-point questionnaire for cognitive dysfunction. It assesses different cognitive domains: attention and concentration, executive functions, memory, language, visuoconstructional skills, conceptual thinking, calculations, and orientation. Scores on the MoCA range from 0-30 with 26-30 indicating normal global cognition; 18-25 mild cognitive impairment; 10-17 moderate cognitive impairment; and \\<10 severe cognitive impairment.'}, {'measure': 'Beck Depression Inventory (BDI) Score', 'timeFrame': 'Month 3', 'description': 'The BDI is a self-reporting 21-item scoring tool that measures characteristic attitudes and symptoms of depression, including physical symptoms. Each of the 21 items on BDI tool represent a depressive symptom. The symptoms are each scored on a 4-point Likert scale of 0 (symptom is absent) to 3 (symptom is severe). Scores for each symptom are added up to obtain the total scores for all 21 items, which are interpreted as follows 1-10 (normal); 11-16 (mild mood disturbance); 17-20 (borderline clinical depression); 21-30 (moderate depression); 31-40 (severe depression); and \\>40 (extreme depression). Participants with symptom score of 0 were not included in the summary.'}, {'measure': 'Change From Baseline in Montgomery and Asberg Depression Rating Scale (MADRS) Total Score at Month 3', 'timeFrame': 'Baseline, Month 3', 'description': 'The MADRS is a clinician-rated tool for measuring changes in depressive symptom severity. Ten core symptoms and cognitive features (feelings of sadness, lassitude, pessimism, inner tension, suicidality, reduced sleep or appetite, difficulty concentrating, and a lack of interest) were rated on a severity scale of 0 (no symptoms)) to 6 (symptoms of maximum severity). The total score was the sum of the scores on the 10 items, ranging from 0 to 60 with a higher score indicating increasing depressive symptoms.'}, {'measure': 'Change From Baseline in Time to Complete Time Up and Go (TUG) Test in ON State at Month 3', 'timeFrame': 'Baseline, Month 3', 'description': "Timed motor tests are simple, objective, quantitative measures for the assessment of Parkinson's disease. They include, in on-medication and off-medication state, timed recorded physical movements. Time Up and Go Test (TUG) is one of timed motor tests which is used to assess a person's mobility and requires both static and dynamic balance. This is a walking assessment. Participants start in the seated position, stand up, walk 7 meters, turn around, and sit back down. The entire process from leaving the chair to returning to the chair was timed. The total time was summarized under ON state with participants on dopamine therapy."}, {'measure': 'Maximum Observed Plasma Concentration (Cmax) of PTC589', 'timeFrame': '0 hour (predose) and 0.5, 1, 2, 4, 6, 8, and 12 hours postdose at Month 1 and 3'}, {'measure': 'Level of Disease-Related Biomarker (Glutathione) in Plasma', 'timeFrame': 'Month 3', 'description': 'Glutathione lowest limit of quantification (LLOQ) = 0.01 micromoles (uM) and upper limit of quantification (ULOQ) = 27.83 uM in plasma.'}, {'measure': 'Level of Disease-Related Biomarker (Glutathione) in Cerebrospinal Fluid (CSF)', 'timeFrame': 'Month 3', 'description': 'Glutathione LLOQ = 0.002 uM and ULOQ = 0.35 uM in CSF.'}, {'measure': 'Level of Disease-Related Biomarker (Glutathione) in Urine', 'timeFrame': 'Month 3', 'description': 'Glutathione LLOQ = 0.01 uM, and ULOQ = 1.39 uM in urine.'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ["Parkinson's Disease", 'PD', 'EPI-589', 'EPI589'], 'conditions': ["Parkinson's Disease"]}, 'descriptionModule': {'briefSummary': 'Open-label study with 30-day run-in phase and adaptive design component to include more participants if deemed appropriate by investigators.', 'detailedDescription': "This is a within-subject, controlled open-label study seeking to determine if PTC-589 can alter the biochemical signature of Parkinson's disease as assessed by peripheral blood biomarkers, central nervous system (CNS) biomarkers, and urine biomarker analysis. In addition, data on a number of disease-relevant clinical measures will be collected."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '75 Years', 'minimumAge': '21 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Hoehn and Yahr stage ≤3.0\n* Ambulatory with or without assistance\n* Sexually active fertile participants and their partners must agree to use medically accepted methods of contraception (such as, hormonal methods, including oral, subcutaneous, and intrauterine; barrier methods, including male condom, female condom, or diaphragm with spermicidal gel) during the course of the study and for 3 months after the last dose of study treatment.\n* Willingness and ability to comply with study procedures\n* If on medications for Parkinson's disease drugs, then medication regimen must be stable for 60 days prior to enrollment\n* Abstention from use of other investigative or non-approved drugs for the duration of the trial\n\nFor Idiopathic Participants\n\n* A diagnosis of idiopathic Parkinson's disease confirmed by the presence of bradykinesia plus one or both of the following symptoms: rigidity or resting tremor; and with an abnormal DaTscan consistent with a dopaminergic deficit\n* Age 40 to 75 years\n* Within 5 years of diagnosis of Parkinson's disease\n\nFor Genetic Subtype Participants\n\n* A confirmed diagnosis of Parkinson's disease plus a genetic diagnosis consistent with Parkinson's disease, specifically PTEN-induced kinase 1 (PINK1), parkin, Leucine-rich repeat kinase 2 (LRRK2) or other mitochondrial genetic subtype\n* Age 21 to 75 years\n\nExclusion Criteria:\n\n* Allergy to PTC-589 or other components of the PTC-589 tablet formulation\n* Use of antioxidant supplements, specifically vitamins E and C beyond the recommended daily allowance\n* Other Parkinsonian disorders\n* Montreal Cognitive Assessment (MoCA) score of \\<24\n* Revised Hamilton Rating Scale for Depression ≥11\n* Parkinsonism due to drugs or toxins\n* Diagnosis of any other clinically significant neurologic disease that will confound the assessment of effect of study drug on disease progression\n* Malignancy within past 2 years\n* Pregnant or plans to become pregnant or breast feeding\n* History of stroke\n* History of brain surgery\n* Hepatic insufficiency with liver function tests (LFTs) \\>3 times upper limit of normal\n* Renal insufficiency as defined by creatinine \\>1.5 times normal\n* End stage cardiac failure\n* Participation within past 3 months and for duration of study in a trial of a device, drug, or other therapy for Parkinson's disease"}, 'identificationModule': {'nctId': 'NCT02462603', 'briefTitle': "Safety and Biomarker Study of PTC-589 in Participants With Parkinson's Disease", 'organization': {'class': 'INDUSTRY', 'fullName': 'Edison Pharmaceuticals Inc'}, 'officialTitle': "A Phase 2A Safety and Biomarker Study of EPI-589 in Mitochondrial Subtype and Idiopathic Parkinson's Disease Subjects", 'orgStudyIdInfo': {'id': 'EPI589-15-002'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'PTC589', 'description': "Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) will receive PTC589 at a dose of 500 milligrams (mg) (2 tablets of 250 mg each) orally twice daily (BID) for up to 3 months unless discontinued for safety or tolerability issues.", 'interventionNames': ['Drug: PTC-589']}], 'interventions': [{'name': 'PTC-589', 'type': 'DRUG', 'otherNames': ['(R)-Troloxamide quinone'], 'description': 'PTC-589 is a redox active molecule and will be provided in a 250 mg tablet formulation.', 'armGroupLabels': ['PTC589']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90048', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': "Cedar's Sinai", 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '94115', 'city': 'San Francisco', 'state': 'California', 'country': 'United States', 'facility': 'University of California, San Francisco', 'geoPoint': {'lat': 37.77493, 'lon': -122.41942}}, {'zip': '02215', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Beth Israel Deaconess Medical Center', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '72074', 'city': 'Tübingen', 'country': 'Germany', 'facility': 'DZNE Site', 'geoPoint': {'lat': 48.52266, 'lon': 9.05222}}, {'zip': 'NW3 2PF', 'city': 'London', 'country': 'United Kingdom', 'facility': 'University College of London,Dept. of Clinical Neuroscience', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'Matthew B Klein, MD FACS', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Edison Pharmaceuticals'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Edison Pharmaceuticals Inc', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}