Ignite Creation Date:
2025-12-24 @ 3:07 PM
Ignite Modification Date:
2026-02-20 @ 6:39 PM
Study NCT ID:
NCT02579759
Status:
COMPLETED
Last Update Posted:
2020-02-27
First Post:
2015-09-28
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT)
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D002607', 'term': 'Charcot-Marie-Tooth Disease'}], 'ancestors': [{'id': 'D015417', 'term': 'Hereditary Sensory and Motor Neuropathy'}, {'id': 'D009421', 'term': 'Nervous System Malformations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D011115', 'term': 'Polyneuropathies'}, {'id': 'D010523', 'term': 'Peripheral Nervous System Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'contact@pharnext.com', 'phone': '+33 (0)1 41 09 22 30', 'title': 'Susanne Dorn', 'organization': 'Pharnext'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': True}, 'limitationsAndCaveats': {'description': 'Two events occured during the trial due to crytals in Dose 2 formulation: hold of all subjects enrolled in Germany (Jun-17) and discontinuation of Dose 2 arm by the sponsor worldwide due to discovery of crystals in the ICH stability batch in Sep-17.'}}, 'adverseEventsModule': {'timeFrame': 'The AE reporting period therefore started with the subject signing the informed consent form and ended 30 days after the end of study (corresponding to the date of "Date of completion/early discontinuation/last contact" recorded in the termination module up to 15 months)', 'description': 'This definition was extended due to the discontinuation of Dose 2 and study on hold in Germany. The period of AE reporting was extended to 1 month after the end of study, without informed consent signed for study CLN-PXT3003-03 during this period.\n\nOnly Treatment-Emergent Adverse Events (TEAE) have been reported for non-serious advers events.', 'eventGroups': [{'id': 'EG000', 'title': 'PXT3003 Dose 1', 'description': 'Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 1: 3 mg baclofen, 0.35 mg naltrexone and 105 mg sorbitol (twice a day, morning and evening with food).', 'otherNumAtRisk': 109, 'deathsNumAtRisk': 109, 'otherNumAffected': 89, 'seriousNumAtRisk': 109, 'deathsNumAffected': 0, 'seriousNumAffected': 10}, {'id': 'EG001', 'title': 'PXT3003 Dose 2', 'description': 'Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 2: 6 mg baclofen, 0.70 mg naltrexone and 210 sorbitol (twice a day, morning and evening with food).', 'otherNumAtRisk': 113, 'deathsNumAtRisk': 113, 'otherNumAffected': 87, 'seriousNumAtRisk': 113, 'deathsNumAffected': 0, 'seriousNumAffected': 3}, {'id': 'EG002', 'title': 'Placebo', 'description': 'Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 matching placebo had the same presentation, the same aspect and taste in order to be undistinguishable (twice a day, morning and evening with food).', 'otherNumAtRisk': 101, 'deathsNumAtRisk': 101, 'otherNumAffected': 83, 'seriousNumAtRisk': 101, 'deathsNumAffected': 0, 'seriousNumAffected': 5}], 'otherEvents': [{'term': 'Hypotension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Asthenia', 'notes': 'and administation site conditions', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 9, 'numAffected': 7}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Fatigue', 'notes': 'and administation site conditions', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 15, 'numAffected': 11}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Influenza like illness', 'notes': 'and administation site conditions', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 5, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 9, 'numAffected': 6}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Peripheral swelling', 'notes': 'and administation site conditions', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Anxiety', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Depression', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Insomnia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 4, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Sleep disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 15, 'numAffected': 9}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 9, 'numAffected': 7}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Laceration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Ligament sprain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 9, 'numAffected': 8}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 12, 'numAffected': 9}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Limb injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Weight increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 6, 'numAffected': 5}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Palpitations', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 6, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 8, 'numAffected': 5}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 10, 'numAffected': 9}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 5, 'numAffected': 5}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 22, 'numAffected': 17}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 20, 'numAffected': 13}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 14, 'numAffected': 11}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Hypoaesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Migraine', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Paraesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 5, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Sciatica', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Somnolence', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 5, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Tremor', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Tinnitus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Vertigo', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 4, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 5, 'numAffected': 5}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 5, 'numAffected': 4}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 9, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 8, 'numAffected': 7}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 8, 'numAffected': 7}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Dry mouth', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Dyspepsia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 17, 'numAffected': 12}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 7, 'numAffected': 7}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Toothache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 5, 'numAffected': 5}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 19, 'numAffected': 13}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 8, 'numAffected': 8}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 12, 'numAffected': 10}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 5, 'numAffected': 5}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Bone callus excessive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Joint swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Muscle spasms', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 7, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 9, 'numAffected': 8}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Musculoskeletal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Myalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Neck pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 4, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Osteoarthritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 13, 'numAffected': 10}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 6, 'numAffected': 5}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 13, 'numAffected': 9}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Rotator cuff syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Tendonitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Decreased appetite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Vitamin D deficiency', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Cystitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 4, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 6, 'numAffected': 6}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 31, 'numAffected': 24}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 26, 'numAffected': 18}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 24, 'numAffected': 15}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Pharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 4, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 5, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 8, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 6, 'numAffected': 6}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Tracheitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numEvents': 5, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 101, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}], 'seriousEvents': [{'term': 'Arthrolysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 1}], 'organSystem': 'Surgical and medical procedures', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Basal cell carcinoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 0}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Thyroid adenoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 0}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Uterine leiomyoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 0}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Suicide attempt', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Endometriosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 0}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Clavicle fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Femoral neck fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Hand fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Median nerve injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Patella fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Rib fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Palpitations', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Congenital foot malformation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 1}], 'organSystem': 'Congenital, familial and genetic disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Patent ductus arteriosus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 0}], 'organSystem': 'Congenital, familial and genetic disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Sleep apnoea syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Seizure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Proctitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Cholecystitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 0}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Hydronephrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Ureterolithiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Chest wall haematoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Appendicitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Enteritis infectious', 'stats': [{'groupId': 'EG000', 'numAtRisk': 109, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 113, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 101, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}], 'frequencyThreshold': '2'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Overall Neuropathy Limitation Scale (ONLS) Total Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '93', 'groupId': 'OG000'}, {'value': '55', 'groupId': 'OG001'}, {'value': '87', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'PXT3003 Dose 1', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 1: 3 mg baclofen, 0.35 mg naltrexone and 105 mg sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG001', 'title': 'PXT3003 Dose 2', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 2: 6 mg baclofen, 0.70 mg naltrexone and 210 sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 matching placebo had the same presentation, the same aspect and taste in order to be undistinguishable (twice a day, morning and evening with food).'}], 'classes': [{'title': 'Base', 'categories': [{'measurements': [{'value': '3.33', 'spread': '1.05', 'groupId': 'OG000'}, {'value': '3.05', 'spread': '1.13', 'groupId': 'OG001'}, {'value': '3.23', 'spread': '1.19', 'groupId': 'OG002'}]}]}, {'title': 'Fin', 'categories': [{'measurements': [{'value': '3.25', 'spread': '1.00', 'groupId': 'OG000'}, {'value': '2.82', 'spread': '1.28', 'groupId': 'OG001'}, {'value': '3.36', 'spread': '1.16', 'groupId': 'OG002'}]}]}], 'analyses': [{'pValue': '0.008', 'groupIds': ['OG001', 'OG002'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '97.5', 'paramValue': '-0.37', 'ciLowerLimit': '-0.68', 'ciUpperLimit': '-0.06', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.14', 'groupDescription': 'The main analysis was performed as follows:\n\n1. Analysis population: modified Full Analysis Set (mFAS)\n2. Statistical model: ANCOVA where the mean at 12 and 15 months of each treatment group was compared against the placebo group, adjusting for the baseline value and assuming centre as a random effect\n3. Missing value imputation: multiple imputation taking into account the reason of missingness', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.287', 'groupIds': ['OG000', 'OG002'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '97.5', 'paramValue': '-0.13', 'ciLowerLimit': '-0.39', 'ciUpperLimit': '0.14', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.12', 'groupDescription': 'The main analysis was performed as follows:\n\n1. Analysis population: modified Full Analysis Set (mFAS)\n2. Statistical model: ANCOVA where the mean at 12 and 15 months of each treatment group was compared against the placebo group, adjusting for the baseline value and assuming centre as a random effect\n3. Missing value imputation: multiple imputation taking into account the reason of missingness', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.013', 'groupIds': ['OG001', 'OG002'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '97.5', 'paramValue': '-0.31', 'ciLowerLimit': '-0.59', 'ciUpperLimit': '-0.03', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.13', 'groupDescription': 'This analysis was performed as follows:\n\n1. Analysis population: modified Full Analysis Set (mFAS)\n2. Statistical model: Longitudinal model where the effect of each treatment over time (baseline, 6, 12 and 15 months) was estimated through a mixed model with repeated measures (MMRM) assuming time from baseline (Time) and Time-by-Treatment full interaction as fixed effects and patient as random effect and considering Time as continuous linear effect\n3. Missing value imputation: No imputation', 'statisticalMethod': 'Longitudinal mixed model', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.05', 'groupIds': ['OG000', 'OG002'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '97.5', 'paramValue': '-0.19', 'ciLowerLimit': '-0.42', 'ciUpperLimit': '0.03', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.1', 'groupDescription': 'This analysis was performed as follows:\n\n1. Analysis population: modified Full Analysis Set (mFAS)\n2. Statistical model: Longitudinal model where the effect of each treatment over time (baseline, 6, 12 and 15 months) was estimated through a mixed model with repeated measures (MMRM) assuming time from baseline (Time) and Time-by-Treatment full interaction as fixed effects and patient as random effect and considering Time as continuous linear effect\n3. Missing value imputation: No imputation', 'statisticalMethod': 'Longitudinal mixed model', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.013', 'groupIds': ['OG000', 'OG001', 'OG002'], 'paramType': 'Slope', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.17', 'ciLowerLimit': '-0.31', 'ciUpperLimit': '-0.04', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.07', 'groupDescription': 'Dose effect:\n\nDose is defined as a numerical variable proportional to quantity of PXT3003 administered (0 for Placebo, 1 for Dose 1, 2 for Dose 2).\n\nThe analysis was performed as follows:\n\n1. Analysis population: modified Full Analysis Set (mFAS)\n2. Statistical model: ANCOVA assessing the dose-effect at the mean of 12 and 15 months, adjusting for baseline value and assuming centre as random effect\n3. Missing value imputation: multiple imputation taking into account the reason of missingness', 'statisticalMethod': 'Regression, Linear', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'MEAN', 'timeFrame': 'From Baseline to Month 15', 'description': 'The primary efficacy variable used in the main analysis is the mean of the available ONLS values at month 12 and month 15.\n\nThe ONLS is a disability scale that was derived and improved from the Overall Disability Sum Score (ODSS) to measure limitations in the everyday activities of the upper limbs (rated on 5 points) and the lower limbs (rated on 7 points). The total score is a 12-point scale: 0 (no disability) to 12 (maximum disability). Lower values in the ONLS indicate a better clinical condition.\n\nReported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin).', 'unitOfMeasure': 'Scores on the ONLS', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'mFAS selection'}, {'type': 'SECONDARY', 'title': 'Mean of Ten Meter Walking Test (10MWT)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '93', 'groupId': 'OG000'}, {'value': '55', 'groupId': 'OG001'}, {'value': '87', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'PXT3003 Dose 1', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 1: 3 mg baclofen, 0.35 mg naltrexone and 105 mg sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG001', 'title': 'PXT3003 Dose 2', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 2: 6 mg baclofen, 0.70 mg naltrexone and 210 sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months\n\nplacebo: Liquid oral solution, 5 ml twice a day, morning and evening with food'}], 'classes': [{'title': 'Base', 'categories': [{'measurements': [{'value': '6.93', 'spread': '1.77', 'groupId': 'OG000'}, {'value': '7.14', 'spread': '1.77', 'groupId': 'OG001'}, {'value': '7.28', 'spread': '1.91', 'groupId': 'OG002'}]}]}, {'title': 'Fin', 'categories': [{'measurements': [{'value': '6.47', 'spread': '1.59', 'groupId': 'OG000'}, {'value': '6.52', 'spread': '1.39', 'groupId': 'OG001'}, {'value': '6.91', 'spread': '1.82', 'groupId': 'OG002'}]}]}], 'analyses': [{'pValue': '0.016', 'groupIds': ['OG001', 'OG002'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '97.5', 'paramValue': '-0.47', 'ciLowerLimit': '-0.91', 'ciUpperLimit': '-0.03', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.19', 'groupDescription': 'The main analysis was performed as follows:\n\n1. Analysis population: modified Full Analysis Set (mFAS)\n2. Statistical model: ANCOVA where the mean at 12 and 15 months of each treatment group was compared against the placebo group, adjusting for the baseline value and assuming centre as a random effect\n3. Missing value imputation: multiple imputation taking into account the reason of missingness', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.084', 'groupIds': ['OG000', 'OG002'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '97.5', 'paramValue': '-0.28', 'ciLowerLimit': '-0.65', 'ciUpperLimit': '0.08', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.16', 'groupDescription': 'The main analysis was performed as follows:\n\n1. Analysis population: modified Full Analysis Set (mFAS)\n2. Statistical model: ANCOVA where the mean at 12 and 15 months of each treatment group was compared against the placebo group, adjusting for the baseline value and assuming centre as a random effect\n3. Missing value imputation: multiple imputation taking into account the reason of missingness', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.015', 'groupIds': ['OG000', 'OG001', 'OG002'], 'paramType': 'Slope', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.22', 'ciLowerLimit': '-0.41', 'ciUpperLimit': '-0.04', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.09', 'groupDescription': 'Dose effect:\n\nDose is defined as a numerical variable proportional to quantity of PXT3003 administered (0 for Placebo, 1 for Dose 1, 2 for Dose 2).\n\nThe analysis was performed as follows:\n\n1. Analysis population: modified Full Analysis Set (mFAS)\n2. Statistical model: ANCOVA assessing the dose-effect at the mean of 12 and 15 months, adjusting for baseline value and assuming centre as random effect\n3. Missing value imputation: multiple imputation taking into account the reason of missingness', 'statisticalMethod': 'Regression, Linear', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'MEAN', 'timeFrame': 'From Baseline to Month 15', 'description': 'This outcome measure is the mean of the available 10MWT values at month 12 and month 15.\n\nThe 10MWT is a simple to administer, standardized, reliable and valid evaluation of functional exercise capacity and gait that has been used to evaluate neurologic disorders and CMT patients.\n\nLower Time to Walk 10 Meters values indicate a better clinical condition.\n\nReported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin).', 'unitOfMeasure': 'Seconds (s)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'mFAS selection'}, {'type': 'SECONDARY', 'title': 'Mean of the CMTNS-v2 Sensory Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '93', 'groupId': 'OG000'}, {'value': '55', 'groupId': 'OG001'}, {'value': '87', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'PXT3003 Dose 1', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 1: 3 mg baclofen, 0.35 mg naltrexone and 105 mg sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG001', 'title': 'PXT3003 Dose 2', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 2: 6 mg baclofen, 0.70 mg naltrexone and 210 sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months\n\nPXT3003 matching placebo had the same presentation, the same aspect and taste in order to be undistinguishable (twice a day, morning and evening with food).'}], 'classes': [{'title': 'Base', 'categories': [{'measurements': [{'value': '5.00', 'spread': '2.28', 'groupId': 'OG000'}, {'value': '4.47', 'spread': '2.21', 'groupId': 'OG001'}, {'value': '4.97', 'spread': '2.04', 'groupId': 'OG002'}]}]}, {'title': 'Fin', 'categories': [{'measurements': [{'value': '4.55', 'spread': '1.96', 'groupId': 'OG000'}, {'value': '4.23', 'spread': '2.38', 'groupId': 'OG001'}, {'value': '4.68', 'spread': '2.14', 'groupId': 'OG002'}]}]}], 'analyses': [{'pValue': '0.162', 'groupIds': ['OG001', 'OG002'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '97.5', 'paramValue': '-0.39', 'ciLowerLimit': '-1.01', 'ciUpperLimit': '0.23', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.28', 'groupDescription': 'The main analysis was performed as follows:\n\n1. Analysis population: modified Full Analysis Set (mFAS)\n2. Statistical model: ANCOVA where the mean at 12 and 15 months of each treatment group was compared against the placebo group, adjusting for the baseline value and assuming centre as a random effect\n3. Missing value imputation: multiple imputation taking into account the reason of missingness', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.556', 'groupIds': ['OG000', 'OG002'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '97.5', 'paramValue': '-0.14', 'ciLowerLimit': '-0.66', 'ciUpperLimit': '0.39', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.23', 'groupDescription': 'The main analysis was performed as follows:\n\n1. Analysis population: modified Full Analysis Set (mFAS)\n2. Statistical model: ANCOVA where the mean at 12 and 15 months of each treatment group was compared against the placebo group, adjusting for the baseline value and assuming centre as a random effect\n3. Missing value imputation: multiple imputation taking into account the reason of missingness', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.204', 'groupIds': ['OG000', 'OG001', 'OG002'], 'paramType': 'Slope', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.17', 'ciLowerLimit': '-0.43', 'ciUpperLimit': '0.09', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.13', 'groupDescription': 'Dose is defined as a numerical variable proportional to quantity of PXT3003 administered (0 for Placebo, 1 for Dose 1, 2 for Dose 2).\n\nThe analysis was performed as follows:\n\n1. Analysis population: modified Full Analysis Set (mFAS)\n2. Statistical model: ANCOVA assessing the dose-effect at the mean of 12 and 15 months, adjusting for baseline value and assuming centre as random effect\n3. Missing value imputation: multiple imputation taking into account the reason of missingness', 'statisticalMethod': 'Regression, Linear', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'MEAN', 'timeFrame': 'From Baseline to Month 15', 'description': 'This outcome measure is the mean of the available CMTNS-v2 Sensory Score values at month 12 and month 15.\n\nThe CMTNS-v2 is a specific scale designed to assess severity of impairment in CMT disease. It is a 36-point scale based on nine items to quantify impairment (sensory symptoms, pin sensibility, vibration and arm and leg strength), activity limitations (motor symptoms arms and legs) and electrophysiological function (amplitudes of ulnar CMAP and SNAP). The CMTNS-v2 goes from 0 (no impairment) to 36 (maximum impairment) whom each sub-items goes from 0 to 4.\n\nThe CMTNS-v2 Sensory score is summed of items 1+4+5 of CMTNS-v2 (Sensory symptoms, Pinprick sensibility and Vibration). It is a 12-point score: 0 (no impairment) to 12 (maximum impairment).\n\nLower CMTNS-v2 Sensory Score values indicate a better clinical condition.\n\nReported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin).', 'unitOfMeasure': 'Scores on the CMTNS-v2 Sensory Score', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'mFAS selection'}, {'type': 'SECONDARY', 'title': 'Mean of the CMTNS-v2 Examination Score (CMTES-v2)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '93', 'groupId': 'OG000'}, {'value': '55', 'groupId': 'OG001'}, {'value': '87', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'PXT3003 Dose 1', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 1: 3 mg baclofen, 0.35 mg naltrexone and 105 mg sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG001', 'title': 'PXT3003 Dose 2', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 2: 6 mg baclofen, 0.70 mg naltrexone and 210 sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 matching placebo had the same presentation, the same aspect and taste in order to be undistinguishable (twice a day, morning and evening with food).'}], 'classes': [{'title': 'Base', 'categories': [{'measurements': [{'value': '9.49', 'spread': '2.80', 'groupId': 'OG000'}, {'value': '8.78', 'spread': '2.73', 'groupId': 'OG001'}, {'value': '9.51', 'spread': '2.79', 'groupId': 'OG002'}]}]}, {'title': 'Fin', 'categories': [{'measurements': [{'value': '9.01', 'spread': '2.62', 'groupId': 'OG000'}, {'value': '8.24', 'spread': '3.13', 'groupId': 'OG001'}, {'value': '9.02', 'spread': '3.05', 'groupId': 'OG002'}]}]}], 'analyses': [{'pValue': '0.232', 'groupIds': ['OG001', 'OG002'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '97.5', 'paramValue': '-0.43', 'ciLowerLimit': '-1.25', 'ciUpperLimit': '0.38', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.36', 'groupDescription': 'The main analysis was performed as follows:\n\n1. Analysis population: modified Full Analysis Set (mFAS)\n2. Statistical model: ANCOVA where the mean at 12 and 15 months of each treatment group was compared against the placebo group, adjusting for the baseline value and assuming centre as a random effect\n3. Missing value imputation: multiple imputation taking into account the reason of missingness', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.868', 'groupIds': ['OG000', 'OG002'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '97.5', 'paramValue': '-0.05', 'ciLowerLimit': '-0.74', 'ciUpperLimit': '0.64', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.31', 'groupDescription': 'The main analysis was performed as follows:\n\n1. Analysis population: modified Full Analysis Set (mFAS)\n2. Statistical model: ANCOVA where the mean at 12 and 15 months of each treatment group was compared against the placebo group, adjusting for the baseline value and assuming centre as a random effect\n3. Missing value imputation: multiple imputation taking into account the reason of missingness', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.322', 'groupIds': ['OG000', 'OG001', 'OG002'], 'paramType': 'Slope', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.18', 'ciLowerLimit': '-0.53', 'ciUpperLimit': '0.17', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.18', 'groupDescription': 'Dose is defined as a numerical variable proportional to quantity of PXT3003 administered (0 for Placebo, 1 for Dose 1, 2 for Dose 2).\n\nThe analysis was performed as follows:\n\n1. Analysis population: modified Full Analysis Set (mFAS)\n2. Statistical model: ANCOVA assessing the dose-effect at the mean of 12 and 15 months, adjusting for baseline value and assuming centre as random effect\n3. Missing value imputation: multiple imputation taking into account the reason of missingness', 'statisticalMethod': 'Regression, Linear', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'MEAN', 'timeFrame': 'From Baseline to Month 15', 'description': 'This outcome measure is the mean of the available CMTNS-v2 Examination Score values at month 12 and month 15.\n\nThe CMTNS-v2 is a specific scale designed to assess severity of impairment in CMT disease. It is a 36-point scale based on nine items to quantify impairment (sensory symptoms, pin sensibility, vibration and arm and leg strength), activity limitations (motor symptoms arms and legs) and electrophysiological function (amplitudes of ulnar CMAP and SNAP). The CMTNS-v2 goes from 0 (no impairment) to 36 (maximum impairment) whom each sub-items goes from 0 to 4.\n\nThe CMTES-v2 is summed of item 1 to 7 of the CMTNS-v2 (limited to impairment items and excluding electrophysiological items). It is a 28-point score: 0 (no impairment) to 28 (maximum impairment).\n\nLower CMTES-v2 values indicate a better clinical condition.\n\nReported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin).', 'unitOfMeasure': 'Scores on the CMTES-v2', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'mFAS selection'}, {'type': 'SECONDARY', 'title': 'Mean of the Results at the Nine-Hole Peg Test (9-HPT)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '93', 'groupId': 'OG000'}, {'value': '55', 'groupId': 'OG001'}, {'value': '87', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'PXT3003 Dose 1', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 1: 3 mg baclofen, 0.35 mg naltrexone and 105 mg sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG001', 'title': 'PXT3003 Dose 2', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 2: 6 mg baclofen, 0.70 mg naltrexone and 210 sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 matching placebo had the same presentation, the same aspect and taste in order to be undistinguishable (twice a day, morning and evening with food).'}], 'classes': [{'title': 'Base', 'categories': [{'measurements': [{'value': '25.62', 'spread': '5.60', 'groupId': 'OG000'}, {'value': '27.33', 'spread': '11.15', 'groupId': 'OG001'}, {'value': '25.18', 'spread': '4.41', 'groupId': 'OG002'}]}]}, {'title': 'Fin', 'categories': [{'measurements': [{'value': '23.85', 'spread': '4.52', 'groupId': 'OG000'}, {'value': '25.67', 'spread': '8.29', 'groupId': 'OG001'}, {'value': '24.41', 'spread': '4.01', 'groupId': 'OG002'}]}]}], 'analyses': [{'pValue': '0.377', 'groupIds': ['OG001', 'OG002'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '97.5', 'paramValue': '-0.4', 'ciLowerLimit': '-1.43', 'ciUpperLimit': '0.62', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.46', 'groupDescription': 'The main analysis was performed as follows:\n\n1. Analysis population: modified Full Analysis Set (mFAS)\n2. Statistical model: ANCOVA where the mean at 12 and 15 months of each treatment group was compared against the placebo group, adjusting for the baseline value and assuming centre as a random effect\n3. Missing value imputation: multiple imputation taking into account the reason of missingness', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.334', 'groupIds': ['OG000', 'OG002'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '97.5', 'paramValue': '-0.36', 'ciLowerLimit': '-1.21', 'ciUpperLimit': '0.48', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.38', 'groupDescription': 'The main analysis was performed as follows:\n\n1. Analysis population: modified Full Analysis Set (mFAS)\n2. Statistical model: ANCOVA where the mean at 12 and 15 months of each treatment group was compared against the placebo group, adjusting for the baseline value and assuming centre as a random effect\n3. Missing value imputation: multiple imputation taking into account the reason of missingness', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.373', 'groupIds': ['OG000', 'OG001', 'OG002'], 'paramType': 'Slope', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.19', 'ciLowerLimit': '-0.62', 'ciUpperLimit': '0.23', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.22', 'groupDescription': 'Dose is defined as a numerical variable proportional to quantity of PXT3003 administered (0 for Placebo, 1 for Dose 1, 2 for Dose 2).\n\nThe analysis was performed as follows:\n\n1. Analysis population: modified Full Analysis Set (mFAS)\n2. Statistical model: ANCOVA assessing the dose-effect at the mean of 12 and 15 months, adjusting for baseline value and assuming centre as random effect\n3. Missing value imputation: multiple imputation taking into account the reason of missingness', 'statisticalMethod': 'Regression, Linear', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'MEAN', 'timeFrame': 'From Baseline to Month 15', 'description': 'This outcome measure is the mean of the available 9-HPT values at month 12 and month 15.\n\nThe Nine-Hole Peg Test (9HPT) is a simple timed test of fine motor coordination of extremitied in the upper limbs. It measures the time needed by the patient to insert 9 pegs in nine holes and to remove them (normal required time 18 seconds).\n\nLower 9HPT values indicate a better clinical condition.\n\nReported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin).', 'unitOfMeasure': 'Seconds (s)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'mFAS selection'}, {'type': 'SECONDARY', 'title': 'Number of Subjects With at Least One TEAE', 'denoms': [{'units': 'Participants', 'counts': [{'value': '109', 'groupId': 'OG000'}, {'value': '113', 'groupId': 'OG001'}, {'value': '101', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'PXT3003 Dose 1', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 1: 3 mg baclofen, 0.35 mg naltrexone and 105 mg sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG001', 'title': 'PXT3003 Dose 2', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 2: 6 mg baclofen, 0.70 mg naltrexone and 210 sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 matching placebo had the same presentation, the same aspect and taste in order to be undistinguishable (twice a day, morning and evening with food).'}], 'classes': [{'title': 'Any TEAE', 'categories': [{'measurements': [{'value': '89', 'groupId': 'OG000'}, {'value': '87', 'groupId': 'OG001'}, {'value': '83', 'groupId': 'OG002'}]}]}, {'title': 'Any related TEAE', 'categories': [{'measurements': [{'value': '39', 'groupId': 'OG000'}, {'value': '38', 'groupId': 'OG001'}, {'value': '34', 'groupId': 'OG002'}]}]}, {'title': 'Any moderately severe or severe related TEAE', 'categories': [{'measurements': [{'value': '8', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}]}], 'paramType': 'NUMBER', 'timeFrame': "The period between the patient signing the informed consent and 30 days after the end of study (i.e. completion/early discontinuation/last contact as recorded on the 'Study Completion on Early Termination' form up to 15 months)", 'description': 'Safety selection was to include all randomized patients that have received at least one dose of study treatment.\n\nSafety and tolerability of PXT3003 were compared to placebo on the incidence of treatment-emergent adverse events (TEAEs); they were evaluated by type/nature, severity/intensity, seriousness, and relationship to study drug.', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'FAS selection'}, {'type': 'SECONDARY', 'title': 'Incidence of AE Leading to Withdrawal of Study Drug', 'denoms': [{'units': 'Participants', 'counts': [{'value': '109', 'groupId': 'OG000'}, {'value': '113', 'groupId': 'OG001'}, {'value': '101', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'PXT3003 Dose 1', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 1: 3 mg baclofen, 0.35 mg naltrexone and 105 mg sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG001', 'title': 'PXT3003 Dose 2', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 2: 6 mg baclofen, 0.70 mg naltrexone and 210 sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 matching placebo had the same presentation, the same aspect and taste in order to be undistinguishable (twice a day, morning and evening with food).'}], 'classes': [{'title': 'Any TEAE leading to drug withdrawal', 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}]}]}, {'title': 'Any related TEAE leading to drug withdrawal', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}]}], 'paramType': 'NUMBER', 'timeFrame': "The period between the patient signing the informed consent and 30 days after the end of study (i.e. completion/early discontinuation/last contact as recorded on the 'Study Completion on Early Termination' form up to 15 months)", 'description': 'Safety and tolerability of PXT3003 were compared to placebo on the incidence of TEAEs leading to withdrawal of study drug.', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'FAS selection'}, {'type': 'SECONDARY', 'title': 'Incidence of SAEs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '109', 'groupId': 'OG000'}, {'value': '113', 'groupId': 'OG001'}, {'value': '101', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'PXT3003 Dose 1', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 1: 3 mg baclofen, 0.35 mg naltrexone and 105 mg sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG001', 'title': 'PXT3003 Dose 2', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 2: 6 mg baclofen, 0.70 mg naltrexone and 210 sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 matching placebo had the same presentation, the same aspect and taste in order to be undistinguishable (twice a day, morning and evening with food).'}], 'classes': [{'title': 'Any serious TEAE', 'categories': [{'measurements': [{'value': '10', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '5', 'groupId': 'OG002'}]}]}, {'title': 'Any related serious TEAE', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Any serious TEAE leading to drug withdrawal', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}], 'paramType': 'NUMBER', 'timeFrame': "The period between the patient signing the informed consent and 30 days after the end of study (i.e. completion/early discontinuation/last contact as recorded on the 'Study Completion on Early Termination' form up to 15 months).", 'description': 'Safety and tolerability of PXT3003 were compared to placebo on the incidence of serious adverse events (SAEs).', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'FAS selection'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Mean of the CMTNS-v2 Sensory Symptoms', 'denoms': [{'units': 'Participants', 'counts': [{'value': '93', 'groupId': 'OG000'}, {'value': '55', 'groupId': 'OG001'}, {'value': '87', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'PXT3003 Dose 1', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 1: 3 mg baclofen, 0.35 mg naltrexone and 105 mg sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG001', 'title': 'PXT3003 Dose 2', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 2: 6 mg baclofen, 0.70 mg naltrexone and 210 sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 matching placebo had the same presentation, the same aspect and taste in order to be undistinguishable (twice a day, morning and evening with food).'}], 'classes': [{'title': 'Base', 'categories': [{'measurements': [{'value': '1.26', 'spread': '0.95', 'groupId': 'OG000'}, {'value': '0.96', 'spread': '0.98', 'groupId': 'OG001'}, {'value': '1.09', 'spread': '0.90', 'groupId': 'OG002'}]}]}, {'title': 'Fin', 'categories': [{'measurements': [{'value': '1.18', 'spread': '0.81', 'groupId': 'OG000'}, {'value': '0.93', 'spread': '0.96', 'groupId': 'OG001'}, {'value': '1.21', 'spread': '0.94', 'groupId': 'OG002'}]}]}], 'analyses': [{'pValue': '0.023', 'groupIds': ['OG001', 'OG002'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '97.5', 'paramValue': '-0.29', 'ciLowerLimit': '-0.58', 'ciUpperLimit': '0', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.13', 'groupDescription': 'The main analysis was performed as follows:\n\n1. Analysis population: modified Full Analysis Set (mFAS)\n2. Statistical model: ANCOVA where the mean at 12 and 15 months of each treatment group was compared against the placebo group, adjusting for the baseline value and assuming centre as a random effect\n3. Missing value imputation: multiple imputation taking into account the reason of missingness', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.162', 'groupIds': ['OG000', 'OG002'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '97.5', 'paramValue': '-0.15', 'ciLowerLimit': '-0.4', 'ciUpperLimit': '0.09', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.11', 'groupDescription': 'The main analysis was performed as follows:\n\n1. Analysis population: modified Full Analysis Set (mFAS)\n2. Statistical model: ANCOVA where the mean at 12 and 15 months of each treatment group was compared against the placebo group, adjusting for the baseline value and assuming centre as a random effect\n3. Missing value imputation: multiple imputation taking into account the reason of missingness', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'MEAN', 'timeFrame': 'From Baseline to Month 15', 'description': 'This outcome measure is the mean of the available CMTNS-v2 Sensory Symptoms values at month 12 and month 15.\n\nThe CMTNS-v2 is a specific scale designed to assess severity of impairment in CMT disease. It is a 36-point scale based on nine items to quantify impairment (sensory symptoms, pin sensibility, vibration and arm and leg strength), activity limitations (motor symptoms arms and legs) and electrophysiological function (amplitudes of ulnar CMAP and SNAP). The CMTNS-v2 goes from 0 (no impairment) to 36 (maximum impairment) whom each sub-items goes from 0 to 4.\n\nThe CMTNS-v2 Sensory Symptoms is the first item of the CMTNS-v2. It is a 4-point score: 0 (no impairment) to 4 (maximum impairment).\n\nLower CMTNS-v2 Sensory Symptoms values indicate a better clinical condition.\n\nReported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin).', 'unitOfMeasure': 'Scores on the CMTNS-v2 Sensory Symptoms', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'mFAS selection'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Plasma Concentrations of Baclofen at Trough and at 90 Min After Drug Intake', 'denoms': [{'units': 'Participants', 'counts': [{'value': '68', 'groupId': 'OG000'}, {'value': '38', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'PXT3003 Dose 1', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 1: 3 mg baclofen, 0.35 mg naltrexone and 105 mg sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG001', 'title': 'PXT3003 Dose 2', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 2: 6 mg baclofen, 0.70 mg naltrexone and 210 sorbitol (twice a day, morning and evening with food).'}], 'classes': [{'title': 'At trough, at Month 12', 'categories': [{'measurements': [{'value': '13739.3', 'spread': '20313.6', 'groupId': 'OG000'}, {'value': '11651.9', 'spread': '6151.1', 'groupId': 'OG001'}]}]}, {'title': 'At trough, at Month 15', 'categories': [{'measurements': [{'value': '9009.7', 'spread': '10910.3', 'groupId': 'OG000'}, {'value': '8686.6', 'spread': '9172.8', 'groupId': 'OG001'}]}]}, {'title': 'At 90 min after drug intake, at Month 12', 'categories': [{'measurements': [{'value': '52201.6', 'spread': '21494.6', 'groupId': 'OG000'}, {'value': '90238.7', 'spread': '29972.8', 'groupId': 'OG001'}]}]}, {'title': 'At 90 min after drug intake, at Month 15', 'categories': [{'measurements': [{'value': '47021.1', 'spread': '19834.5', 'groupId': 'OG000'}, {'value': '105825.4', 'spread': '38756.7', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'At Month 12 and Month 15', 'description': 'Plasma concentration of PXT3003 components were measured at trough (prior to dose) and 90 minutes after drug intake.\n\nThe mean plasma values of the baseline correspond to half of the administered dose.', 'unitOfMeasure': 'pg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PP selection LLOQ = 30 pg/mL The placebo arm has not been described for this endpoint.'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Plasma Concentrations of Naltrexone at Trough and at 90 Min After Drug Intake', 'denoms': [{'units': 'Participants', 'counts': [{'value': '68', 'groupId': 'OG000'}, {'value': '38', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'PXT3003 Dose 1', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 1: 3 mg baclofen, 0.35 mg naltrexone and 105 mg sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG001', 'title': 'PXT3003 Dose 2', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 2: 6 mg baclofen, 0.70 mg naltrexone and 210 sorbitol (twice a day, morning and evening with food).'}], 'classes': [{'title': 'At trough, at Month 12', 'categories': [{'measurements': [{'value': '33.0', 'spread': '15.8', 'groupId': 'OG000'}, {'value': '42.0', 'spread': '66.0', 'groupId': 'OG001'}]}]}, {'title': 'At trough, at Month 15', 'categories': [{'measurements': [{'value': '31.8', 'spread': '14.0', 'groupId': 'OG000'}, {'value': '30.0', 'spread': '0.0', 'groupId': 'OG001'}]}]}, {'title': 'At 90 min after drug intake, at Month 12', 'categories': [{'measurements': [{'value': '63.0', 'spread': '47.4', 'groupId': 'OG000'}, {'value': '107.5', 'spread': '88.6', 'groupId': 'OG001'}]}]}, {'title': 'At 90 min after drug intake, at Month 15', 'categories': [{'measurements': [{'value': '55.0', 'spread': '39.3', 'groupId': 'OG000'}, {'value': '130.9', 'spread': '81.4', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'At Month 12 and month 15', 'description': 'Plasma concentration of PXT3003 components were measured at trough (prior to dose) and 90 minutes after drug intake.\n\nThe mean plasma values of the baseline correspond to half of the administered dose.', 'unitOfMeasure': 'pg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PP selection LLOQ = 30 pg/mL The placebo arm has not been described for this endpoint.'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Plasma Concentrations of 6β-naltrexol at Trough and at 90 Min After Drug Intake', 'denoms': [{'units': 'Participants', 'counts': [{'value': '68', 'groupId': 'OG000'}, {'value': '38', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'PXT3003 Dose 1', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 1: 3 mg baclofen, 0.35 mg naltrexone and 105 mg sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG001', 'title': 'PXT3003 Dose 2', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 2: 6 mg baclofen, 0.70 mg naltrexone and 210 sorbitol (twice a day, morning and evening with food).'}], 'classes': [{'title': 'At trough, at Month 12', 'categories': [{'measurements': [{'value': '290.1', 'spread': '177.4', 'groupId': 'OG000'}, {'value': '526.4', 'spread': '245.6', 'groupId': 'OG001'}]}]}, {'title': 'At trough, at Month 15', 'categories': [{'measurements': [{'value': '260.4', 'spread': '121.8', 'groupId': 'OG000'}, {'value': '352.3', 'spread': '319.0', 'groupId': 'OG001'}]}]}, {'title': 'At 90 min after drug intake, at Month 12', 'categories': [{'measurements': [{'value': '632.5', 'spread': '230.1', 'groupId': 'OG000'}, {'value': '1257.1', 'spread': '454.3', 'groupId': 'OG001'}]}]}, {'title': 'At 90 min after drug intake, at Month 15', 'categories': [{'measurements': [{'value': '586.4', 'spread': '205.4', 'groupId': 'OG000'}, {'value': '1450.9', 'spread': '438.0', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'At Month 12 and Month 15', 'description': 'Plasma concentration of PXT3003 components were measured at trough (prior to dose) and peak (90 minutes post dose).\n\nThe mean plasma values of the baseline correspond to half of the administered dose.', 'unitOfMeasure': 'pg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PP selection LLOQ = 50 pg/mL The placebo arm has not been described for this endpoint.'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Number of Participants With ONLS Therapy Response 1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '85', 'groupId': 'OG000'}, {'value': '49', 'groupId': 'OG001'}, {'value': '80', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'PXT3003 Dose 1', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 1: 3 mg baclofen, 0.35 mg naltrexone and 105 mg sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG001', 'title': 'PXT3003 Dose 2', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 2: 6 mg baclofen, 0.70 mg naltrexone and 210 sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months\n\nplacebo: Liquid oral solution, 5 ml twice a day, morning and evening with food'}], 'classes': [{'categories': [{'measurements': [{'value': '16', 'groupId': 'OG000'}, {'value': '14', 'groupId': 'OG001'}, {'value': '14', 'groupId': 'OG002'}]}]}], 'analyses': [{'pValue': '0.097', 'groupIds': ['OG001', 'OG002'], 'paramType': 'Odds Ratio (OR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '97.5', 'paramValue': '2.09', 'ciLowerLimit': '0.77', 'ciUpperLimit': '5.68', 'groupDescription': 'The proportion of responders (on Completers selection only) at the end of treatment was assessed through a Generalized Linear Mixed Model (GLMM) featuring logistic regression including treatment as a fixed effect, adjusting for the baseline value and center as a random effect.', 'statisticalMethod': 'General Linear Mixed Model', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.865', 'groupIds': ['OG000', 'OG002'], 'paramType': 'Odds Ratio (OR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '97.5', 'paramValue': '1.07', 'ciLowerLimit': '0.42', 'ciUpperLimit': '2.7', 'groupDescription': 'The proportion of responders (on Completers selection only) at the end of treatment was assessed through a Generalized Linear Mixed Model (GLMM) featuring logistic regression including treatment as a fixed effect, adjusting for the baseline value and center as a random effect.', 'statisticalMethod': 'General Linear Mixed Model', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'NUMBER', 'timeFrame': 'From Baseline to Month 15', 'description': 'ONLS Therapy Response 1 was defined as the number of participants (responders) with an improvement on final ONLS Total Score of at least one point. A higher response rate indicate a better clinical condition.', 'unitOfMeasure': 'Number of Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Completers selection'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Number of Participants With ONLS Therapy Response 2', 'denoms': [{'units': 'Participants', 'counts': [{'value': '85', 'groupId': 'OG000'}, {'value': '89', 'groupId': 'OG001'}, {'value': '80', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'PXT3003 Dose 1', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 1: 3 mg baclofen, 0.35 mg naltrexone and 105 mg sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG001', 'title': 'PXT3003 Dose 2', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 2: 6 mg baclofen, 0.70 mg naltrexone and 210 sorbitol (twice a day, morning and evening with food).'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 matching placebo had the same presentation, the same aspect and taste in order to be undistinguishable (twice a day, morning and evening with food).'}], 'classes': [{'categories': [{'measurements': [{'value': '66', 'groupId': 'OG000'}, {'value': '42', 'groupId': 'OG001'}, {'value': '58', 'groupId': 'OG002'}]}]}], 'analyses': [{'pValue': '0.026', 'groupIds': ['OG001', 'OG002'], 'paramType': 'Odds Ratio (OR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '97.5', 'paramValue': '3.39', 'ciLowerLimit': '0.99', 'ciUpperLimit': '11.62', 'groupDescription': 'The proportion of responders (on Completers selection only) at the end of treatment was assessed through a Generalized Linear Mixed Model (GLMM) featuring logistic regression including treatment as a fixed effect, adjusting for the baseline value and center as a random effect.', 'statisticalMethod': 'General Linear Mixed Model', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.569', 'groupIds': ['OG000', 'OG002'], 'paramType': 'Odds Ratio (OR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '97.5', 'paramValue': '1.26', 'ciLowerLimit': '0.5', 'ciUpperLimit': '3.16', 'groupDescription': 'The proportion of responders (on Completers selection only) at the end of treatment was assessed through a Generalized Linear Mixed Model (GLMM) featuring logistic regression including treatment as a fixed effect, adjusting for the baseline value and center as a random effect.', 'statisticalMethod': 'General Linear Mixed Model', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'NUMBER', 'timeFrame': 'From Baseline to Month 15', 'description': 'ONLS Therapy Response 2 was defined as the number of participants with no deterioration (responders) on final ONLS Total Score.\n\nA higher response rate indicates a better clinical condition.', 'unitOfMeasure': 'Number of Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Completers selection'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'PXT3003 Dose 1', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 1: 3 mg baclofen, 0.35 mg naltrexone and 105 mg sorbitol (twice a day, morning and evening with food).'}, {'id': 'FG001', 'title': 'PXT3003 Dose 2', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 dose 2: 6 mg baclofen, 0.70 mg naltrexone and 210 sorbitol (twice a day, morning and evening with food).'}, {'id': 'FG002', 'title': 'Placebo', 'description': 'Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months.\n\nPXT3003 matching placebo had the same presentation, the same aspect and taste in order to be undistinguishable (twice a day, morning and evening with food).'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '109'}, {'groupId': 'FG001', 'numSubjects': '113'}, {'groupId': 'FG002', 'numSubjects': '101'}]}, {'type': 'COMPLETED', 'comment': 'Completed at 12 months', 'achievements': [{'groupId': 'FG000', 'numSubjects': '85'}, {'groupId': 'FG001', 'numSubjects': '49'}, {'groupId': 'FG002', 'numSubjects': '80'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '24'}, {'groupId': 'FG001', 'numSubjects': '64'}, {'groupId': 'FG002', 'numSubjects': '21'}]}], 'dropWithdraws': [{'type': 'Protocol Violation', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'Other (Sponsor stopped Dose 2)', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'BfArM hold', 'reasons': [{'groupId': 'FG000', 'numSubjects': '13'}, {'groupId': 'FG001', 'numSubjects': '12'}, {'groupId': 'FG002', 'numSubjects': '12'}]}, {'type': 'Sponsor stopped Dose 2', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '41'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'Non compliance', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'Pregnancy', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'Inclusion/exclusion criteria', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '1'}]}, {'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '4'}, {'groupId': 'FG001', 'numSubjects': '3'}, {'groupId': 'FG002', 'numSubjects': '1'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '3'}, {'groupId': 'FG002', 'numSubjects': '5'}]}, {'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '2'}, {'groupId': 'FG002', 'numSubjects': '2'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '109', 'groupId': 'BG000'}, {'value': '113', 'groupId': 'BG001'}, {'value': '101', 'groupId': 'BG002'}, {'value': '323', 'groupId': 'BG003'}]}], 'groups': [{'id': 'BG000', 'title': 'PXT3003 Dose 1', 'description': 'Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months\n\nPXT3003 dose 1: Liquid oral solution, 5 ml twice a day, morning and evening with food'}, {'id': 'BG001', 'title': 'PXT3003 Dose 2', 'description': 'Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months\n\nPXT3003 dose 2: Liquid oral solution, 5 ml twice a day, morning and evening with food'}, {'id': 'BG002', 'title': 'Placebo', 'description': 'Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months\n\nplacebo: Liquid oral solution, 5 ml twice a day, morning and evening with food'}, {'id': 'BG003', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '15', 'groupId': 'BG003'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '103', 'groupId': 'BG000'}, {'value': '104', 'groupId': 'BG001'}, {'value': '97', 'groupId': 'BG002'}, {'value': '304', 'groupId': 'BG003'}]}, {'title': '>=65 years', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '4', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '41.0', 'spread': '12.3', 'groupId': 'BG000'}, {'value': '39.6', 'spread': '13.9', 'groupId': 'BG001'}, {'value': '42.1', 'spread': '13.2', 'groupId': 'BG002'}, {'value': '40.9', 'spread': '13.2', 'groupId': 'BG003'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '60', 'groupId': 'BG000'}, {'value': '68', 'groupId': 'BG001'}, {'value': '62', 'groupId': 'BG002'}, {'value': '190', 'groupId': 'BG003'}]}, {'title': 'Male', 'measurements': [{'value': '49', 'groupId': 'BG000'}, {'value': '45', 'groupId': 'BG001'}, {'value': '39', 'groupId': 'BG002'}, {'value': '133', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}, {'value': '3', 'groupId': 'BG003'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '108', 'groupId': 'BG000'}, {'value': '111', 'groupId': 'BG001'}, {'value': '100', 'groupId': 'BG002'}, {'value': '319', 'groupId': 'BG003'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'Canada', 'categories': [{'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}, {'value': '14', 'groupId': 'BG003'}]}]}, {'title': 'Netherlands', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '8', 'groupId': 'BG003'}]}]}, {'title': 'Belgium', 'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '6', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}, {'value': '15', 'groupId': 'BG003'}]}]}, {'title': 'United States', 'categories': [{'measurements': [{'value': '21', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '18', 'groupId': 'BG002'}, {'value': '63', 'groupId': 'BG003'}]}]}, {'title': 'United Kingdom', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}, {'value': '3', 'groupId': 'BG003'}]}]}, {'title': 'France', 'categories': [{'measurements': [{'value': '31', 'groupId': 'BG000'}, {'value': '31', 'groupId': 'BG001'}, {'value': '29', 'groupId': 'BG002'}, {'value': '91', 'groupId': 'BG003'}]}]}, {'title': 'Germany', 'categories': [{'measurements': [{'value': '22', 'groupId': 'BG000'}, {'value': '23', 'groupId': 'BG001'}, {'value': '22', 'groupId': 'BG002'}, {'value': '67', 'groupId': 'BG003'}]}]}, {'title': 'Spain', 'categories': [{'measurements': [{'value': '22', 'groupId': 'BG000'}, {'value': '22', 'groupId': 'BG001'}, {'value': '18', 'groupId': 'BG002'}, {'value': '62', 'groupId': 'BG003'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2017-12-05', 'size': 2158526, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2019-11-22T08:47', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'TRIPLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 323}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2015-12', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-02', 'completionDateStruct': {'date': '2018-08', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-02-13', 'studyFirstSubmitDate': '2015-09-28', 'resultsFirstSubmitDate': '2019-11-18', 'studyFirstSubmitQcDate': '2015-10-16', 'lastUpdatePostDateStruct': {'date': '2020-02-27', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-02-13', 'studyFirstPostDateStruct': {'date': '2015-10-20', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-02-27', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2018-03', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Mean of the CMTNS-v2 Sensory Symptoms', 'timeFrame': 'From Baseline to Month 15', 'description': 'This outcome measure is the mean of the available CMTNS-v2 Sensory Symptoms values at month 12 and month 15.\n\nThe CMTNS-v2 is a specific scale designed to assess severity of impairment in CMT disease. It is a 36-point scale based on nine items to quantify impairment (sensory symptoms, pin sensibility, vibration and arm and leg strength), activity limitations (motor symptoms arms and legs) and electrophysiological function (amplitudes of ulnar CMAP and SNAP). The CMTNS-v2 goes from 0 (no impairment) to 36 (maximum impairment) whom each sub-items goes from 0 to 4.\n\nThe CMTNS-v2 Sensory Symptoms is the first item of the CMTNS-v2. It is a 4-point score: 0 (no impairment) to 4 (maximum impairment).\n\nLower CMTNS-v2 Sensory Symptoms values indicate a better clinical condition.\n\nReported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin).'}, {'measure': 'Plasma Concentrations of Baclofen at Trough and at 90 Min After Drug Intake', 'timeFrame': 'At Month 12 and Month 15', 'description': 'Plasma concentration of PXT3003 components were measured at trough (prior to dose) and 90 minutes after drug intake.\n\nThe mean plasma values of the baseline correspond to half of the administered dose.'}, {'measure': 'Plasma Concentrations of Naltrexone at Trough and at 90 Min After Drug Intake', 'timeFrame': 'At Month 12 and month 15', 'description': 'Plasma concentration of PXT3003 components were measured at trough (prior to dose) and 90 minutes after drug intake.\n\nThe mean plasma values of the baseline correspond to half of the administered dose.'}, {'measure': 'Plasma Concentrations of 6β-naltrexol at Trough and at 90 Min After Drug Intake', 'timeFrame': 'At Month 12 and Month 15', 'description': 'Plasma concentration of PXT3003 components were measured at trough (prior to dose) and peak (90 minutes post dose).\n\nThe mean plasma values of the baseline correspond to half of the administered dose.'}, {'measure': 'Number of Participants With ONLS Therapy Response 1', 'timeFrame': 'From Baseline to Month 15', 'description': 'ONLS Therapy Response 1 was defined as the number of participants (responders) with an improvement on final ONLS Total Score of at least one point. A higher response rate indicate a better clinical condition.'}, {'measure': 'Number of Participants With ONLS Therapy Response 2', 'timeFrame': 'From Baseline to Month 15', 'description': 'ONLS Therapy Response 2 was defined as the number of participants with no deterioration (responders) on final ONLS Total Score.\n\nA higher response rate indicates a better clinical condition.'}], 'primaryOutcomes': [{'measure': 'Overall Neuropathy Limitation Scale (ONLS) Total Score', 'timeFrame': 'From Baseline to Month 15', 'description': 'The primary efficacy variable used in the main analysis is the mean of the available ONLS values at month 12 and month 15.\n\nThe ONLS is a disability scale that was derived and improved from the Overall Disability Sum Score (ODSS) to measure limitations in the everyday activities of the upper limbs (rated on 5 points) and the lower limbs (rated on 7 points). The total score is a 12-point scale: 0 (no disability) to 12 (maximum disability). Lower values in the ONLS indicate a better clinical condition.\n\nReported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin).'}], 'secondaryOutcomes': [{'measure': 'Mean of Ten Meter Walking Test (10MWT)', 'timeFrame': 'From Baseline to Month 15', 'description': 'This outcome measure is the mean of the available 10MWT values at month 12 and month 15.\n\nThe 10MWT is a simple to administer, standardized, reliable and valid evaluation of functional exercise capacity and gait that has been used to evaluate neurologic disorders and CMT patients.\n\nLower Time to Walk 10 Meters values indicate a better clinical condition.\n\nReported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin).'}, {'measure': 'Mean of the CMTNS-v2 Sensory Score', 'timeFrame': 'From Baseline to Month 15', 'description': 'This outcome measure is the mean of the available CMTNS-v2 Sensory Score values at month 12 and month 15.\n\nThe CMTNS-v2 is a specific scale designed to assess severity of impairment in CMT disease. It is a 36-point scale based on nine items to quantify impairment (sensory symptoms, pin sensibility, vibration and arm and leg strength), activity limitations (motor symptoms arms and legs) and electrophysiological function (amplitudes of ulnar CMAP and SNAP). The CMTNS-v2 goes from 0 (no impairment) to 36 (maximum impairment) whom each sub-items goes from 0 to 4.\n\nThe CMTNS-v2 Sensory score is summed of items 1+4+5 of CMTNS-v2 (Sensory symptoms, Pinprick sensibility and Vibration). It is a 12-point score: 0 (no impairment) to 12 (maximum impairment).\n\nLower CMTNS-v2 Sensory Score values indicate a better clinical condition.\n\nReported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin).'}, {'measure': 'Mean of the CMTNS-v2 Examination Score (CMTES-v2)', 'timeFrame': 'From Baseline to Month 15', 'description': 'This outcome measure is the mean of the available CMTNS-v2 Examination Score values at month 12 and month 15.\n\nThe CMTNS-v2 is a specific scale designed to assess severity of impairment in CMT disease. It is a 36-point scale based on nine items to quantify impairment (sensory symptoms, pin sensibility, vibration and arm and leg strength), activity limitations (motor symptoms arms and legs) and electrophysiological function (amplitudes of ulnar CMAP and SNAP). The CMTNS-v2 goes from 0 (no impairment) to 36 (maximum impairment) whom each sub-items goes from 0 to 4.\n\nThe CMTES-v2 is summed of item 1 to 7 of the CMTNS-v2 (limited to impairment items and excluding electrophysiological items). It is a 28-point score: 0 (no impairment) to 28 (maximum impairment).\n\nLower CMTES-v2 values indicate a better clinical condition.\n\nReported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin).'}, {'measure': 'Mean of the Results at the Nine-Hole Peg Test (9-HPT)', 'timeFrame': 'From Baseline to Month 15', 'description': 'This outcome measure is the mean of the available 9-HPT values at month 12 and month 15.\n\nThe Nine-Hole Peg Test (9HPT) is a simple timed test of fine motor coordination of extremitied in the upper limbs. It measures the time needed by the patient to insert 9 pegs in nine holes and to remove them (normal required time 18 seconds).\n\nLower 9HPT values indicate a better clinical condition.\n\nReported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin).'}, {'measure': 'Number of Subjects With at Least One TEAE', 'timeFrame': "The period between the patient signing the informed consent and 30 days after the end of study (i.e. completion/early discontinuation/last contact as recorded on the 'Study Completion on Early Termination' form up to 15 months)", 'description': 'Safety selection was to include all randomized patients that have received at least one dose of study treatment.\n\nSafety and tolerability of PXT3003 were compared to placebo on the incidence of treatment-emergent adverse events (TEAEs); they were evaluated by type/nature, severity/intensity, seriousness, and relationship to study drug.'}, {'measure': 'Incidence of AE Leading to Withdrawal of Study Drug', 'timeFrame': "The period between the patient signing the informed consent and 30 days after the end of study (i.e. completion/early discontinuation/last contact as recorded on the 'Study Completion on Early Termination' form up to 15 months)", 'description': 'Safety and tolerability of PXT3003 were compared to placebo on the incidence of TEAEs leading to withdrawal of study drug.'}, {'measure': 'Incidence of SAEs', 'timeFrame': "The period between the patient signing the informed consent and 30 days after the end of study (i.e. completion/early discontinuation/last contact as recorded on the 'Study Completion on Early Termination' form up to 15 months).", 'description': 'Safety and tolerability of PXT3003 were compared to placebo on the incidence of serious adverse events (SAEs).'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['PXT3003'], 'conditions': ['Charcot-Marie-Tooth Disease Type 1A']}, 'referencesModule': {'references': [{'pmid': '25519680', 'type': 'RESULT', 'citation': 'Attarian S, Vallat JM, Magy L, Funalot B, Gonnaud PM, Lacour A, Pereon Y, Dubourg O, Pouget J, Micallef J, Franques J, Lefebvre MN, Ghorab K, Al-Moussawi M, Tiffreau V, Preudhomme M, Magot A, Leclair-Visonneau L, Stojkovic T, Bossi L, Lehert P, Gilbert W, Bertrand V, Mandel J, Milet A, Hajj R, Boudiaf L, Scart-Gres C, Nabirotchkin S, Guedj M, Chumakov I, Cohen D. An exploratory randomised double-blind and placebo-controlled phase 2 study of a combination of baclofen, naltrexone and sorbitol (PXT3003) in patients with Charcot-Marie-Tooth disease type 1A. Orphanet J Rare Dis. 2014 Dec 18;9:199. doi: 10.1186/s13023-014-0199-0.'}, {'pmid': '25491744', 'type': 'RESULT', 'citation': 'Chumakov I, Milet A, Cholet N, Primas G, Boucard A, Pereira Y, Graudens E, Mandel J, Laffaire J, Foucquier J, Glibert F, Bertrand V, Nave KA, Sereda MW, Vial E, Guedj M, Hajj R, Nabirotchkin S, Cohen D. Polytherapy with a combination of three repurposed drugs (PXT3003) down-regulates Pmp22 over-expression and improves myelination, axonal and functional parameters in models of CMT1A neuropathy. Orphanet J Rare Dis. 2014 Dec 10;9:201. doi: 10.1186/s13023-014-0201-x.'}, {'pmid': '26070802', 'type': 'RESULT', 'citation': 'Mandel J, Bertrand V, Lehert P, Attarian S, Magy L, Micallef J, Chumakov I, Scart-Gres C, Guedj M, Cohen D. A meta-analysis of randomized double-blind clinical trials in CMT1A to assess the change from baseline in CMTNS and ONLS scales after one year of treatment. Orphanet J Rare Dis. 2015 Jun 13;10:74. doi: 10.1186/s13023-015-0293-y.'}, {'pmid': '30650121', 'type': 'RESULT', 'citation': 'Prukop T, Stenzel J, Wernick S, Kungl T, Mroczek M, Adam J, Ewers D, Nabirotchkin S, Nave KA, Hajj R, Cohen D, Sereda MW. Early short-term PXT3003 combinational therapy delays disease onset in a transgenic rat model of Charcot-Marie-Tooth disease 1A (CMT1A). PLoS One. 2019 Jan 16;14(1):e0209752. doi: 10.1371/journal.pone.0209752. eCollection 2019.'}, {'type': 'RESULT', 'citation': 'Hajj R, Prukop T, Wernick S, Ewers D, Brureau A, Cholet N, Laffaire J, Nave KA, Cohen D, Sereda M. Baclofen, Naltrexone and Sorbitol all contribute to the efficacy of PXT3003 in CMT1A Rats. EMJ Neurol, 2019;7[1]:47-49'}, {'pmid': '27387831', 'type': 'RESULT', 'citation': 'Attarian S, Vallat JM, Magy L, Funalot B, Gonnaud PM, Lacour A, Pereon Y, Dubourg O, Pouget J, Micallef J, Franques J, Lefebvre MN, Ghorab K, Al-Moussawi M, Tiffreau V, Preudhomme M, Magot A, Leclair-Visonneau L, Stojkovic T, Bossi L, Lehert P, Gilbert W, Bertrand V, Mandel J, Milet A, Hajj R, Boudiaf L, Scart-Gres C, Nabirotchkin S, Guedj M, Chumakov I, Cohen D. Erratum to: An exploratory randomised double-blind and placebo-controlled phase 2 study of a combination of baclofen, naltrexone and sorbitol (PXT3003) in patients with Charcot-Marie-Tooth disease type 1A. Orphanet J Rare Dis. 2016 Jul 7;11(1):92. doi: 10.1186/s13023-016-0463-6. No abstract available.'}], 'seeAlsoLinks': [{'url': 'https://www.ncbi.nlm.nih.gov/pubmed/25519680', 'label': 'Attarian et al., 2016'}, {'url': 'https://www.ncbi.nlm.nih.gov/pubmed/?term=25491744', 'label': 'Chumakov et al., 2014'}, {'url': 'https://www.ncbi.nlm.nih.gov/pubmed/?term=26070802', 'label': 'Mandel et al., 2015'}, {'url': 'https://www.ncbi.nlm.nih.gov/pubmed/?term=30650121', 'label': 'Prukop et al., 2019'}, {'url': 'https://www.ncbi.nlm.nih.gov/pubmed/?term=27387831', 'label': 'Attarian et al., Erratum, 2016'}, {'url': 'https://www.emjreviews.com/neurology/abstract/baclofen-naltrexone-and-sorbitol-all-contribute-to-the-efficacy-of-pxt3003-in-cmt1a-rats/', 'label': 'Hajj et al., 2019'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to determine whether PXT3003 is effective and safe in the treatment of Charcot-Marie-Tooth disease - Type 1 A (CMT1A). This double-blind study will assess in parallel groups 2 doses of PXT3003 compared to Placebo in CMT1A patients treated for 15 months.', 'detailedDescription': 'PXT3003 is a fixed dose combination of (RS)-baclofen, naltrexone hydrochloride and D-sorbitol selected via a Systems Biology approach and developed by Pharnext, with the aim to limit the production of PMP22 and protect/improve axonal function in patients with CMT1A. On September 18th 2017, PXT3003 dose 2 was prematurely discontinued, due to an unexpected investigational medicinal product quality event (failed month 18 stability testing). This resulted in a large proportion of missing data that led us to reconsider the efficacy analysis that was initially planned in the protocol.The independent data safety monitoring committee did not identify any safety concern on September 5th 2017. All patients randomised to dose 2 were requested to undergo the end of study visit, and were offered to enter the extension study (CLN-PXT3003-03).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '16 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Male or female, aged from 16 to 65 years;\n* Patient with a proven genetic diagnosis of CMT1A;\n* Mild-to-moderate severity assessed by Charcot-Marie-Tooth Neuropathy Score (version 2) with a score \\>2 and ≤18;\n* Muscle weakness in at least foot dorsiflexion;\n* Motor nerve conduction of the ulnar nerve of at least 15 m/sec;\n* Providing signed written informed consent to participate in the study and willing and able to comply with all study procedures and scheduled visits.\n\nExclusion Criteria:\n\n* Any other associated cause of peripheral neuropathy such as diabetes;\n* Patient with another significant neurological disease or a concomitant major systemic disease;\n* Clinically significant history of unstable medical illness since the last 30 days (unstable angina, cancer…) that may jeopardize the participation in the study;\n* Significant hematologic disease, hepatitis or liver failure, renal failure;\n* Limb surgery within six months before randomization or planned before trial completion;\n* Clinically significant abnormalities on the pre-study laboratory evaluation, physical evaluation, electrocardiogram (ECG);\n* Elevated ASAT/ALAT (\\> 3 x ULN) and elevated serum creatinine levels (\\> 1.25 x ULN);\n* History of recent alcohol or drug abuse or non-adherence with treatment or other experimental protocols;\n* Patient using unauthorized concomitant treatments including but not limited to baclofen, naltrexone, sorbitol (pharmaceutical form), opioids, levothyroxin and potentially neurotoxic drugs such as amiodarone, chloroquine, cancer drugs susceptible to induce a peripheral neuropathy. Patient who can/agrees to stop these medications 4 weeks before randomization and during the whole study duration can be included;\n* Female of childbearing potential (apart of patient using adequate contraceptive measures), pregnant or breast feeding;\n* Known hypersensitivity to any of the individual components of PXT3003;\n* Porphyria as it is a contra indication to baclofen, and it may also induce neuropathy;\n* Suspected inability to complete the study follow-up (foreign workers, transient visitors, tourists or any others for whom follow-up evaluation is not assured);\n* Limited mental capacity or psychiatric disease rendering the subject unable to provide written informed consent or comply with evaluation procedures;\n* Patient who has participated in another trial of investigational drug(s) within the past 30 days;\n* If a patient from the same family, living in the same household, has already been included in this study, it will not be possible to include another patient from the same family to avoid mixing of therapeutic units; therefore there would be a risk of inversion of the blind treatments which could jeopardize the interpretation of study results.'}, 'identificationModule': {'nctId': 'NCT02579759', 'acronym': 'PLEO-CMT', 'briefTitle': 'Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT)', 'organization': {'class': 'OTHER', 'fullName': 'Pharnext S.C.A.'}, 'officialTitle': 'International, Multi-center, Randomized, Double-blind, Placebo-controlled Phase III Study Assessing in Parallel Groups the Efficacy and Safety of 2 Doses of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A Treated 15 Months', 'orgStudyIdInfo': {'id': 'CLN-PXT3003-02'}, 'secondaryIdInfos': [{'id': '2015-002378-19', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'ACTIVE_COMPARATOR', 'label': 'PXT3003 dose 1', 'description': 'Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months', 'interventionNames': ['Drug: PXT3003 dose 1']}, {'type': 'ACTIVE_COMPARATOR', 'label': 'PXT3003 dose 2', 'description': 'Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months', 'interventionNames': ['Drug: PXT3003 dose 2']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'placebo', 'description': 'Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months', 'interventionNames': ['Drug: placebo']}], 'interventions': [{'name': 'PXT3003 dose 1', 'type': 'DRUG', 'otherNames': ['DOSE 1'], 'description': 'Liquid oral solution, 5 ml twice a day, morning and evening with food', 'armGroupLabels': ['PXT3003 dose 1']}, {'name': 'PXT3003 dose 2', 'type': 'DRUG', 'otherNames': ['DOSE 2'], 'description': 'Liquid oral solution, 5 ml twice a day, morning and evening with food', 'armGroupLabels': ['PXT3003 dose 2']}, {'name': 'placebo', 'type': 'DRUG', 'description': 'Liquid oral solution, 5 ml twice a day, morning and evening with food', 'armGroupLabels': ['placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90048', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'Department of Neurology, Cedars-Sinai Medical Center', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '06053', 'city': 'New Britain', 'state': 'Connecticut', 'country': 'United States', 'facility': 'Hospital for Special Care, New Britain', 'geoPoint': {'lat': 41.66121, 'lon': -72.77954}}, {'zip': '32610', 'city': 'Gainesville', 'state': 'Florida', 'country': 'United States', 'facility': 'Department of Neurology, McKnight Brain Institute', 'geoPoint': {'lat': 29.65163, 'lon': -82.32483}}, {'zip': '66160', 'city': 'Kansas City', 'state': 'Kansas', 'country': 'United States', 'facility': 'University of Kansas Medical Center', 'geoPoint': {'lat': 39.11417, 'lon': -94.62746}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Brigham and Women's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '48109-5322', 'city': 'Ann Arbor', 'state': 'Michigan', 'country': 'United States', 'facility': 'University of Michigan Health System', 'geoPoint': {'lat': 42.27756, 'lon': -83.74088}}, {'zip': '55455', 'city': 'Minneapolis', 'state': 'Minnesota', 'country': 'United States', 'facility': 'Department of Neurology, University of Minnesota', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}, {'zip': '63104-1027', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Department of Neurology and Psichiatry, Saint Louis University', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '10032', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Peripheral Neuropathy Center, Neurological Institue Building, Columbia University Medical Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '43210', 'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'facility': 'Ohio State University', 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '99202-1330', 'city': 'Spokane', 'state': 'Washington', 'country': 'United States', 'facility': "Saint Luke's Rehabilitation Institute", 'geoPoint': {'lat': 47.65966, 'lon': -117.42908}}, {'city': 'Leuven', 'country': 'Belgium', 'facility': 'Departement of Neurology, UZ Leuven', 'geoPoint': {'lat': 50.87959, 'lon': 4.70093}}, {'zip': 'G1J 1Z4', 'city': 'Québec', 'state': 'Quebec', 'country': 'Canada', 'facility': 'University Hospital of Quebec', 'geoPoint': {'lat': 46.81228, 'lon': -71.21454}}, {'city': 'Lille', 'country': 'France', 'facility': 'Centre de Référence des Maladies Neuromusculaires, Hôpital Swynghedauwl, CHU de Lille', 'geoPoint': {'lat': 50.63391, 'lon': 3.05512}}, {'city': 'Limoges', 'country': 'France', 'facility': 'Centre de Référence des Neuropathies Périphériques Rares, Hôpital Dupuytren, CHU Limoges', 'geoPoint': {'lat': 45.83362, 'lon': 1.24759}}, {'city': 'Lyon', 'country': 'France', 'facility': 'Service de Neurologie et du Sommeil, CHU Lyon Sud', 'geoPoint': {'lat': 45.74906, 'lon': 4.84789}}, {'city': 'Marseille', 'country': 'France', 'facility': 'Centre de Référence des Maladies Neuromusculaires, Pôle des Neurosciences Clinique, CHU la Timone', 'geoPoint': {'lat': 43.29695, 'lon': 5.38107}}, {'city': 'Nantes', 'country': 'France', 'facility': 'Centre de Référence des Maladies Neuromusculaires; 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