Ignite Creation Date:
2025-12-26 @ 10:57 AM
Ignite Modification Date:
2026-01-13 @ 7:05 PM
Study NCT ID:
NCT02858908
Status:
COMPLETED
Last Update Posted:
2025-09-11
First Post:
2016-08-04
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009223', 'term': 'Myotonic Dystrophy'}], 'ancestors': [{'id': 'D009136', 'term': 'Muscular Dystrophies'}, {'id': 'D020966', 'term': 'Muscular Disorders, Atrophic'}, {'id': 'D009135', 'term': 'Muscular Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D020967', 'term': 'Myotonic Disorders'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C520571', 'term': 'tideglusib'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'mike.snape@amo-pharma.com', 'phone': '+44 (0) 7775915639', 'title': 'Dr. Mike Snape, Chief Scientific Officer', 'organization': 'AMO Pharma Ltd.'}, 'certainAgreement': {'otherDetails': "The investigator has the right to publish study results from his/her specific site. However, any publication that includes AMO Pharma confidential information cannot be submitted for publication without AMO Pharma's prior written approval.", 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Consent to end of study, up to 14 weeks', 'description': 'SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.', 'eventGroups': [{'id': 'EG000', 'title': 'Cohort 1', 'description': '1000 mg tideglusib', 'otherNumAtRisk': 8, 'deathsNumAtRisk': 8, 'otherNumAffected': 8, 'seriousNumAtRisk': 8, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Cohort 2', 'description': '400 mg tideglusib', 'otherNumAtRisk': 8, 'deathsNumAtRisk': 8, 'otherNumAffected': 6, 'seriousNumAtRisk': 8, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Back injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Joint dislocation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Laceration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Limb injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Nervous system cyst', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Somnolence', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Diarrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Post-tussive vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Dysmenorrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Eczema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Rash papular', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Axillary mass', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Myalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Vitamin D deficiency', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Lower respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Subcutaneous abscess', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Safety (Adverse Events)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1', 'description': 'Subjects were given tideglusib 1000 mg for 12 weeks (weeks 0 to 12)'}, {'id': 'OG001', 'title': 'Cohort 2', 'description': 'Subjects were given tideglusib 400 mg for 12 weeks (weeks 0 to 12)'}], 'classes': [{'title': 'Total number of TEAEs', 'categories': [{'measurements': [{'value': '8', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}]}]}, {'title': 'Total number of serious TEAEs', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Total number of TEAEs leading to discontinuation of active treatment', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Total number of TEAEs leading to withdrawal', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Total number of TEAEs leading to death', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Severity: Mild', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Severity: Moderate', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}]}, {'title': 'Severity: Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Unrelated to active treatment', 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}]}]}, {'title': 'Related to active treatment', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '12 weeks', 'description': 'Incidence of Adverse events (AEs), including serious adverse events (SAEs), between baseline to end of study.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Analysis Set'}, {'type': 'SECONDARY', 'title': 'Plasma Concentration of Tideglusib', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1', 'description': '1000 mg tideglusib'}, {'id': 'OG001', 'title': 'Cohort 2', 'description': '400 mg tideglusib'}], 'classes': [{'title': 'Maximum plasma concentration (Cmax)', 'categories': [{'measurements': [{'value': '1041.25', 'spread': '361.44', 'groupId': 'OG000'}, {'value': '496.94', 'spread': '104.88', 'groupId': 'OG001'}]}]}, {'title': 'Minimum plasma concentration (Cmin)', 'categories': [{'measurements': [{'value': '8.63', 'spread': '5.68', 'groupId': 'OG000'}, {'value': '5.14', 'spread': '4.06', 'groupId': 'OG001'}]}]}, {'title': 'Steady-state concentration (CSS)', 'categories': [{'measurements': [{'value': '133.7', 'spread': '60.79', 'groupId': 'OG000'}, {'value': '54.27', 'spread': '16.54', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': '12 weeks', 'description': 'Pharmacokinetic samples were collected to determine Tideglusib plasma concentration', 'unitOfMeasure': 'ng/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set'}, {'type': 'SECONDARY', 'title': 'Blood Pharmacokinetics of Tideglusib', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1', 'description': '1000 mg tideglusib'}, {'id': 'OG001', 'title': 'Cohort 2', 'description': '400 mg tideglusib'}], 'classes': [{'title': 'Time of the maximum plasma concentration (Tmax)', 'categories': [{'measurements': [{'value': '0.79', 'spread': '0.27', 'groupId': 'OG000'}, {'value': '0.65', 'spread': '0.29', 'groupId': 'OG001'}]}]}, {'title': 'Terminal elimination half-life (T1/2)', 'categories': [{'measurements': [{'value': '1.82', 'spread': '0.64', 'groupId': 'OG000'}, {'value': '2.68', 'spread': '1.70', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': '12 weeks', 'description': 'Pharmacokinetic samples were collected to determine time of the maximum plasma concentration and terminal elimination half-life of tideglusib', 'unitOfMeasure': 'h', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set'}, {'type': 'SECONDARY', 'title': 'Area Under the Plasma Concentration vs. Time Curve of Tideglusib', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1', 'description': '1000 mg tideglusib'}, {'id': 'OG001', 'title': 'Cohort 2', 'description': '400 mg tideglusib'}], 'classes': [{'title': 'Area under the plasma concentration vs. time curve from 0 to 12 h (AUC0-12)', 'categories': [{'measurements': [{'value': '3054.27', 'spread': '1418.30', 'groupId': 'OG000'}, {'value': '1214.88', 'spread': '390.81', 'groupId': 'OG001'}]}]}, {'title': 'Area under the plasma concentration vs. time curve from 0 to 24 h (AUC0-24)', 'categories': [{'measurements': [{'value': '3208.8', 'spread': '1459.03', 'groupId': 'OG000'}, {'value': '1302.42', 'spread': '396.96', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': '12 weeks', 'description': 'Pharmacokinetic samples were collected to determine area under the plasma concentration vs. time curve from 0 to 12 h and area under the plasma concentration vs. time curve from 0 to 24 h of tideglusib', 'unitOfMeasure': 'ng/mL.h', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set'}, {'type': 'SECONDARY', 'title': '10 Metre Walk/Run Test', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1', 'description': '1000 mg tideglusib'}, {'id': 'OG001', 'title': 'Cohort 2', 'description': '400 mg tideglusib'}], 'classes': [{'title': 'Preferred speed (observed value at week 12)', 'categories': [{'measurements': [{'value': '9.1041', 'spread': '1.34351', 'groupId': 'OG000'}, {'value': '9.1475', 'spread': '1.72651', 'groupId': 'OG001'}]}]}, {'title': 'Preferred speed (change from baseline at week 12)', 'categories': [{'measurements': [{'value': '-0.6616', 'spread': '1.96370', 'groupId': 'OG000'}, {'value': '-0.6529', 'spread': '0.86109', 'groupId': 'OG001'}]}]}, {'title': 'Fastest speed (observed value at week 12)', 'categories': [{'measurements': [{'value': '4.2515', 'spread': '2.41439', 'groupId': 'OG000'}, {'value': '4.4833', 'spread': '2.72809', 'groupId': 'OG001'}]}]}, {'title': 'Fastest speed (change from baseline at week 12)', 'categories': [{'measurements': [{'value': '-0.3894', 'spread': '0.32806', 'groupId': 'OG000'}, {'value': '-0.0650', 'spread': '0.46428', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0484', 'groupIds': ['OG000'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.6696', 'ciLowerLimit': '-1.3340', 'ciUpperLimit': '-0.0051', 'estimateComment': 'The mean difference is an adjusted Least Square Mean', 'groupDescription': 'Change from baseline to week 12 in the time taken (seconds) to complete 10-metre walk/run test at the preferred speed', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values.'}, {'pValue': '0.0565', 'groupIds': ['OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.6449', 'ciLowerLimit': '-1.3094', 'ciUpperLimit': '0.0195', 'estimateComment': 'The mean difference is an adjusted Least Square Mean', 'groupDescription': 'Change from baseline to week 12 in the time taken (seconds) to complete 10-metre walk/run test at the preferred speed', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values.'}], 'paramType': 'MEAN', 'timeFrame': '12 weeks', 'description': 'The 10-metre walk/run test is a performance measure used to assess walking speed in metres per second over a short distance and was used as an assessment of functional mobility. Time taken to complete the 10m walk/run test at fastest and preferred speed is measured.', 'unitOfMeasure': 'seconds', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set'}, {'type': 'SECONDARY', 'title': 'Computerised Handgrip Myometer Measure of Grip Strength and Muscle Relaxation Time', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1', 'description': '1000 mg tideglusib'}, {'id': 'OG001', 'title': 'Cohort 2', 'description': '400 mg tideglusib'}], 'classes': [{'title': 'Observed value (at week 12)', 'categories': [{'measurements': [{'value': '14.0899', 'spread': '7.71583', 'groupId': 'OG000'}, {'value': '14.1334', 'spread': '6.86499', 'groupId': 'OG001'}]}]}, {'title': 'Change from baseline (at week 12)', 'categories': [{'measurements': [{'value': '1.0098', 'spread': '3.09422', 'groupId': 'OG000'}, {'value': '-1.4893', 'spread': '2.89328', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.3732', 'groupIds': ['OG000'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.9102', 'ciLowerLimit': '-1.1526', 'ciUpperLimit': '2.9730', 'estimateComment': 'The mean difference is an adjusted Least Square Mean', 'groupDescription': 'Change from baseline to week 12 in grip strength (kg)', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values.'}, {'pValue': '0.1782', 'groupIds': ['OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-1.3897', 'ciLowerLimit': '-3.4525', 'ciUpperLimit': '0.6731', 'estimateComment': 'The mean difference is an adjusted Least Square Mean', 'groupDescription': 'Change from baseline to week 12 in grip strength (kg)', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values.'}], 'paramType': 'MEAN', 'timeFrame': '12 weeks', 'description': 'Handgrip myometry is used as a measure of myotonia and muscle strength for the dominant hand', 'unitOfMeasure': 'kg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set'}, {'type': 'SECONDARY', 'title': 'Respiratory Forced Vital Capacity (FVC)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1', 'description': '1000 mg tideglusib'}, {'id': 'OG001', 'title': 'Cohort 2', 'description': '400 mg tideglusib'}], 'classes': [{'title': 'Observed value (at week 12)', 'categories': [{'measurements': [{'value': '2.1509', 'spread': '0.93362', 'groupId': 'OG000'}, {'value': '2.4220', 'spread': '1.15969', 'groupId': 'OG001'}]}]}, {'title': 'Change from baseline (at week 12)', 'categories': [{'measurements': [{'value': '-0.0179', 'spread': '0.32603', 'groupId': 'OG000'}, {'value': '0.0118', 'spread': '0.36746', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.8886', 'groupIds': ['OG000'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.0213', 'ciLowerLimit': '-0.3368', 'ciUpperLimit': '0.2941', 'estimateComment': 'The mean difference is an adjusted Least Square Mean', 'groupDescription': 'Change from baseline to week 12 in FVC (litres)', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values.'}, {'pValue': '0.9204', 'groupIds': ['OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.0152', 'ciLowerLimit': '-0.3003', 'ciUpperLimit': '0.3307', 'estimateComment': 'The mean difference is an adjusted Least Square Mean', 'groupDescription': 'Change from baseline to week 12 in FVC (litres)', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values.'}], 'paramType': 'MEAN', 'timeFrame': '12 weeks', 'description': 'FVC is a measure of lung function (the total amount of air exhaled during a Forced Expiratory Volume is measured using a spirometer)', 'unitOfMeasure': 'litres', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set'}, {'type': 'SECONDARY', 'title': 'Dual-energy X-ray Absorptiometry (DXA)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1', 'description': '1000 mg tideglusib'}, {'id': 'OG001', 'title': 'Cohort 2', 'description': '400 mg tideglusib'}], 'classes': [{'title': 'Arms (observed value at week 12)', 'categories': [{'measurements': [{'value': '3102.9', 'spread': '898.69', 'groupId': 'OG000'}, {'value': '4098.5', 'spread': '1313.53', 'groupId': 'OG001'}]}]}, {'title': 'Arms (change from baseline at week 12)', 'categories': [{'measurements': [{'value': '-252.8', 'spread': '1038.49', 'groupId': 'OG000'}, {'value': '35.9', 'spread': '357.09', 'groupId': 'OG001'}]}]}, {'title': 'Legs (observed value at week 12)', 'categories': [{'measurements': [{'value': '11767.0', 'spread': '2980.78', 'groupId': 'OG000'}, {'value': '12262.8', 'spread': '3225.63', 'groupId': 'OG001'}]}]}, {'title': 'Legs (change from baseline at week 12)', 'categories': [{'measurements': [{'value': '-62.5', 'spread': '646.22', 'groupId': 'OG000'}, {'value': '95.0', 'spread': '299.82', 'groupId': 'OG001'}]}]}, {'title': 'Total (observed value at week 12)', 'categories': [{'measurements': [{'value': '38267.6', 'spread': '8274.58', 'groupId': 'OG000'}, {'value': '37740.4', 'spread': '8481.27', 'groupId': 'OG001'}]}]}, {'title': 'Total (change from baseline at week 12)', 'categories': [{'measurements': [{'value': '411.6', 'spread': '2040.84', 'groupId': 'OG000'}, {'value': '391.9', 'spread': '763.01', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '1.0000', 'groupIds': ['OG000'], 'paramType': 'Median Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-42.75', 'ciLowerLimit': '-1270.00', 'ciUpperLimit': '2178.50', 'estimateComment': 'Hodges-Lehmann estimates for the median and confidence intervals are presented', 'groupDescription': 'Changes from baseline to week 12 in the DXA scan total lean muscle mass (g)', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'The exact Wilcoxon signed rank test was used to test for a change in the DXA scan total lean muscle mass (g) from baseline to end of treatment'}, {'pValue': '0.3125', 'groupIds': ['OG001'], 'paramType': 'Median Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '426.75', 'ciLowerLimit': '-289.50', 'ciUpperLimit': '1198.50', 'estimateComment': 'Hodges-Lehmann estimates for the median and confidence intervals are presented', 'groupDescription': 'Changes from baseline to week 12 in the DXA scan total lean muscle mass (g)', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'The exact Wilcoxon signed rank test was used to test for a change in the DXA scan total lean muscle mass (g) from baseline to end of treatment'}], 'paramType': 'MEAN', 'timeFrame': '12 weeks', 'description': "DXA utilises two low energy X-ray beams, with different energy levels, which are aimed at the subject's bones. The DXA scan is more typically used to measure bone mineral density, however it can also be used to measure total lean muscle mass", 'unitOfMeasure': 'gram', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set'}, {'type': 'SECONDARY', 'title': 'Clinical Global Impressions- Severity (CGI-S)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1', 'description': '1000 mg tideglusib'}, {'id': 'OG001', 'title': 'Cohort 2', 'description': '400 mg tideglusib'}], 'classes': [{'title': 'Observed value at week 12', 'categories': [{'measurements': [{'value': '4.8', 'spread': '1.16', 'groupId': 'OG000'}, {'value': '4.9', 'spread': '0.83', 'groupId': 'OG001'}]}]}, {'title': 'Change from baseline at week 12', 'categories': [{'measurements': [{'value': '-0.1', 'spread': '0.35', 'groupId': 'OG000'}, {'value': '0.0', 'spread': '0.00', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': '12 weeks', 'description': "The CGI-S is a 7-point Likert type scale that requires the clinician to rate the severity of the subject's illness at the time of assessment, relative to the clinician's past experience with subjects who have the same diagnosis. Considering total clinical experience, a subject is assessed on severity of illness at the time of rating 1, normal, not at all ill; 2, borderline ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill. Change in CGI-S was observed in only 1 subject. Consequently statistical analysis was not conducted.", 'unitOfMeasure': 'Values on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set'}, {'type': 'SECONDARY', 'title': 'Clinical Global Impressions- Improvement (CGI-I)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1', 'description': '1000 mg tideglusib'}, {'id': 'OG001', 'title': 'Cohort 2', 'description': '400 mg tideglusib'}], 'classes': [{'categories': [{'measurements': [{'value': '3.1', 'spread': '0.83', 'groupId': 'OG000'}, {'value': '3.0', 'spread': '1.07', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0030', 'groupIds': ['OG000'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '3.1', 'ciLowerLimit': '2.6', 'ciUpperLimit': '3.7', 'pValueComment': "The p-value is presented for the comparison of adjusted Least Square Means to a score of 4, representing 'No change'", 'groupDescription': 'Observed value at week 12 in Clinical Global Impression Global Improvement Scale (CGI-I)', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': "A mixed effect model repeated measure (MMRM) was fitted to the observed values at week12. The model included dose group and visit as fixed effects, and the treatment-by-visit interaction. F-tests from PROC MIXED were based on Kenward-Roger's adjusted degrees of freedom."}, {'pValue': '0.0009', 'groupIds': ['OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '3.0', 'ciLowerLimit': '2.4', 'ciUpperLimit': '3.6', 'pValueComment': "The p-value is presented for the comparison of adjusted Least Square Means to a score of 4, representing 'No change'", 'groupDescription': 'Observed value at week 12 in Clinical Global Impression Global Improvement Scale (CGI-I)', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': "A mixed effect model repeated measure (MMRM) was fitted to the observed values at week12. The model included dose group and visit as fixed effects, and the treatment-by-visit interaction. F-tests from PROC MIXED were based on Kenward-Roger's adjusted degrees of freedom."}], 'paramType': 'MEAN', 'timeFrame': '12 weeks', 'description': "The CGI-I requires the clinician to rate how much the subject's illness has improved or worsened relative to a baseline state.\n\nA seven point Likert type scale is used from 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, 7 = very much worse.", 'unitOfMeasure': 'Scores on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set'}, {'type': 'SECONDARY', 'title': 'Actigraphy (3-minute Bouts of Activity)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1', 'description': '1000 mg tideglusib'}, {'id': 'OG001', 'title': 'Cohort 2', 'description': '400 mg tideglusib'}], 'classes': [{'title': 'Weekly total number of 3 minute bouts of activity (observed value at week 12)', 'categories': [{'measurements': [{'value': '1.465', 'spread': '0.1780', 'groupId': 'OG000'}, {'value': '0.938', 'spread': '0.5268', 'groupId': 'OG001'}]}]}, {'title': 'Weekly total number of 3 minute bouts of activity (change from baseline at week 12)', 'categories': [{'measurements': [{'value': '-0.200', 'spread': '0.3959', 'groupId': 'OG000'}, {'value': '-0.284', 'spread': '0.1805', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.1857', 'groupIds': ['OG000'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.132', 'ciLowerLimit': '-0.330', 'ciUpperLimit': '0.066', 'estimateComment': 'The mean difference is an adjusted Least Square Mean', 'groupDescription': 'Change from baseline to week 12 in the weekly total number of 3 minute bouts of activity per hour wear time', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values.'}, {'pValue': '0.0054', 'groupIds': ['OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.325', 'ciLowerLimit': '-0.549', 'ciUpperLimit': '-0.102', 'estimateComment': 'The mean difference is an adjusted Least Square Mean', 'groupDescription': 'Change from baseline to week 12 in the weekly total number of 3 minute bouts of activity per hour wear time', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values.'}], 'paramType': 'MEAN', 'timeFrame': '12 weeks', 'description': "Actigraphy is a non-invasive method of monitoring physical activity via an actigraph device. The actigraph device was worn on the waist during waking hours, according to the device's instruction manual.", 'unitOfMeasure': '3 minute bouts of activity per hour', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set'}, {'type': 'SECONDARY', 'title': 'Actigraphy (>10-minute Bouts of Activity)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1', 'description': '1000 mg tideglusib'}, {'id': 'OG001', 'title': 'Cohort 2', 'description': '400 mg tideglusib'}], 'classes': [{'title': 'Weekly total number of >10 minute bouts of activity (observed value at week 12)', 'categories': [{'measurements': [{'value': '0.048', 'spread': '0.0621', 'groupId': 'OG000'}, {'value': '0.020', 'spread': '0.0245', 'groupId': 'OG001'}]}]}, {'title': 'Weekly total number of >10 minute bouts of activity (change from baseline at week 12)', 'categories': [{'measurements': [{'value': '-0.010', 'spread': '0.0600', 'groupId': 'OG000'}, {'value': '0.006', 'spread': '0.0313', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': '12 weeks', 'description': "Actigraphy is a non-invasive method of monitoring physical activity via an actigraph device. The actigraph device was worn on the waist during waking hours, according to the device's instruction manual.", 'unitOfMeasure': '10 minute bouts of activity per hour', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set'}, {'type': 'SECONDARY', 'title': 'Actigraphy (Steps)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1', 'description': '1000 mg tideglusib'}, {'id': 'OG001', 'title': 'Cohort 2', 'description': '400 mg tideglusib'}], 'classes': [{'title': 'Weekly total number of steps (observed value at week 12)', 'categories': [{'measurements': [{'value': '556.387', 'spread': '255.6433', 'groupId': 'OG000'}, {'value': '352.518', 'spread': '263.1992', 'groupId': 'OG001'}]}]}, {'title': 'Weekly total number of steps (change from baseline at week 12)', 'categories': [{'measurements': [{'value': '-51.427', 'spread': '198.9754', 'groupId': 'OG000'}, {'value': '-53.802', 'spread': '89.0843', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': '12 weeks', 'description': "Actigraphy is a non-invasive method of monitoring physical activity via an actigraph device. The actigraph device was worn on the waist during waking hours, according to the device's instruction manual.", 'unitOfMeasure': 'steps per hour wear time', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set'}, {'type': 'SECONDARY', 'title': 'Nine Hole Peg Test (NHPT)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1', 'description': '1000 mg tideglusib'}, {'id': 'OG001', 'title': 'Cohort 2', 'description': '400 mg tideglusib'}], 'classes': [{'title': 'Observed value at week 12', 'categories': [{'measurements': [{'value': '23.590', 'spread': '8.2592', 'groupId': 'OG000'}, {'value': '24.760', 'spread': '3.8275', 'groupId': 'OG001'}]}]}, {'title': 'Change from baseline at week 12', 'categories': [{'measurements': [{'value': '-2.168', 'spread': '3.9007', 'groupId': 'OG000'}, {'value': '-0.950', 'spread': '1.7080', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0111', 'groupIds': ['OG000'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-2.162', 'ciLowerLimit': '-3.731', 'ciUpperLimit': '-0.593', 'estimateComment': 'The mean difference is an adjusted Least Square Mean', 'groupDescription': 'Change from baseline to week 12 in the time taken (seconds) to complete the nine hole peg test for the dominant arm', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values.'}, {'pValue': '0.2088', 'groupIds': ['OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.955', 'ciLowerLimit': '-2.525', 'ciUpperLimit': '0.614', 'estimateComment': 'The mean difference is an adjusted Least Square Mean', 'groupDescription': 'Change from baseline to week 12 in the time taken (seconds) to complete the nine hole peg test for the dominant arm', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values.'}], 'paramType': 'MEAN', 'timeFrame': '12 weeks', 'description': 'Measure of fine manual dexterity. Time to taken to complete the NHPT (dominant hand) is recorded.', 'unitOfMeasure': 'seconds', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set'}, {'type': 'SECONDARY', 'title': 'Top 3 Concerns Visual Analogue Scale (VAS) Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1', 'description': '1000 mg tideglusib'}, {'id': 'OG001', 'title': 'Cohort 2', 'description': '400 mg tideglusib'}], 'classes': [{'title': 'Subject Top 3 Concerns VAS total score (observed value at week 12)', 'categories': [{'measurements': [{'value': '15.66', 'spread': '6.331', 'groupId': 'OG000'}, {'value': '15.29', 'spread': '5.641', 'groupId': 'OG001'}]}]}, {'title': 'Subject Top 3 Concerns VAS total score (change from baseline at week 12)', 'categories': [{'measurements': [{'value': '-1.76', 'spread': '3.455', 'groupId': 'OG000'}, {'value': '-2.00', 'spread': '2.598', 'groupId': 'OG001'}]}]}, {'title': 'Caregiver Top 3 Concerns VAS total score (observed value at week 12)', 'categories': [{'measurements': [{'value': '18.84', 'spread': '3.583', 'groupId': 'OG000'}, {'value': '16.58', 'spread': '6.151', 'groupId': 'OG001'}]}]}, {'title': 'Caregiver Top 3 Concerns VAS total score (change from baseline at week 12)', 'categories': [{'measurements': [{'value': '-2.39', 'spread': '2.669', 'groupId': 'OG000'}, {'value': '-1.98', 'spread': '2.001', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0728', 'groupIds': ['OG000'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-1.77', 'ciLowerLimit': '-3.71', 'ciUpperLimit': '0.18', 'estimateComment': 'The mean difference is an adjusted Least Square Mean', 'groupDescription': 'Change from baseline to week 12 in the subject top three concerns VAS total score (cm)', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values.'}, {'pValue': '0.0456', 'groupIds': ['OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-1.99', 'ciLowerLimit': '-3.94', 'ciUpperLimit': '-0.04', 'estimateComment': 'The mean difference is an adjusted Least Square Mean', 'groupDescription': 'Change from baseline to week 12 in the subject top three concerns VAS total score (cm)', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values.'}, {'pValue': '0.0058', 'groupIds': ['OG000'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-2.41', 'ciLowerLimit': '-4.03', 'ciUpperLimit': '-0.80', 'estimateComment': 'The mean difference is an adjusted Least Square Mean', 'groupDescription': 'Change from baseline to week 12 in the caregiver top three concerns VAS total score (cm)', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values.'}, {'pValue': '0.0208', 'groupIds': ['OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-1.95', 'ciLowerLimit': '-3.56', 'ciUpperLimit': '-0.34', 'estimateComment': 'The mean difference is an adjusted Least Square Mean', 'groupDescription': 'Change from baseline to week 12 in the caregiver top three concerns VAS total score (cm)', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values.'}], 'paramType': 'MEAN', 'timeFrame': '12 weeks', 'description': 'The Top 3 concerns VAS allows caregivers to identify their main three causes of concern, related to the subject\'s myotonic dystrophy, rather than these being pre-specified within a scale and then rating how these concerns have changed at specific time-points during the study. Subjects, where possible, and caregivers were asked to rate three causes for concern by drawing a vertical mark on a 10 cm long visual analogue scale with anchors of "not at all severe" at the left end (0 cm) and "very severe" at the right end (10 cm). A score for each concern was to be determined by measuring the number of centimeters on the 10 cm VAS line from the anchor point on the left side of the line. A total VAS score for each subject was calculated as the sum of the scores for the 3 concerns (minimum = 0 cm, maximum = 30 cm). A higher score represents a worse outcome.', 'unitOfMeasure': 'Score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set'}, {'type': 'SECONDARY', 'title': 'Ohio State University (OSU) Autism Rating Scale (OARS)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1', 'description': '1000 mg tideglusib'}, {'id': 'OG001', 'title': 'Cohort 2', 'description': '400 mg tideglusib'}], 'classes': [{'title': 'Total impairment mean (observed value at week 12)', 'categories': [{'measurements': [{'value': '0.239', 'spread': '0.3178', 'groupId': 'OG000'}, {'value': '0.303', 'spread': '0.4946', 'groupId': 'OG001'}]}]}, {'title': 'Total impairment mean (change from baseline at week 12)', 'categories': [{'measurements': [{'value': '-0.054', 'spread': '0.0782', 'groupId': 'OG000'}, {'value': '-0.010', 'spread': '0.0283', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0068', 'groupIds': ['OG000'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.054', 'ciLowerLimit': '-0.092', 'ciUpperLimit': '-0.017', 'estimateComment': 'The mean difference is an adjusted Least Square Mean', 'groupDescription': 'Change from baseline to week 12 in the OSU Autism Rating Scale - DSM-IV (OARS-4) total impairment mean', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values.'}, {'pValue': '0.6090', 'groupIds': ['OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.009', 'ciLowerLimit': '-0.047', 'ciUpperLimit': '0.028', 'estimateComment': 'The mean difference is an adjusted Least Square Mean', 'groupDescription': 'Change from baseline to week 12 in the OSU Autism Rating Scale - DSM-IV (OARS-4) total impairment mean', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values.'}], 'paramType': 'MEAN', 'timeFrame': '12 weeks', 'description': "The OARS-4 contains the autism signs and symptoms in the Diagnostic and Statistical Manual of Mental Disorders, 4th Edition. These were to be rated with the degree of impairment the subject experiences for the given symptom. The symptoms were to be elicited in a semi-structured interview with the subject's primary caregiver. The assessor was to take both frequency/duration and degree of impairment into account and how much the item interferes with relationships, learning, and/or activities of daily living. The scores for this assessment were from 0 (Never or Rarely; Not a Problem) to 3 (Very Often; A Severe Problem): a score for each symptom of social impairment, communication impairment and restricted patterns were averaged to provide a total impairment score. A higher score represents a worse outcome (minimum = 0, maximum 3).", 'unitOfMeasure': 'Units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set'}, {'type': 'SECONDARY', 'title': 'Ohio State University (OSU) Autism Clinical Global Impression (CGI)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1', 'description': '1000 mg tideglusib'}, {'id': 'OG001', 'title': 'Cohort 2', 'description': '400 mg tideglusib'}], 'classes': [{'title': 'OSU autism CGI-Severity (observed value at week 12)', 'categories': [{'measurements': [{'value': '1.9', 'spread': '0.99', 'groupId': 'OG000'}, {'value': '2.3', 'spread': '1.83', 'groupId': 'OG001'}]}]}, {'title': 'OSU autism CGI-Severity (change from baseline at week 12)', 'categories': [{'measurements': [{'value': '-0.1', 'spread': '0.35', 'groupId': 'OG000'}, {'value': '0', 'spread': '0', 'groupId': 'OG001'}]}]}, {'title': 'OSU autism CGI-Improvement (observed value at week 12)', 'categories': [{'measurements': [{'value': '3.4', 'spread': '0.74', 'groupId': 'OG000'}, {'value': '4', 'spread': '0', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0011', 'groupIds': ['OG000'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '3.4', 'ciLowerLimit': '3.0', 'ciUpperLimit': '3.7', 'pValueComment': "p-values are presented for the comparison of adjusted Least Square Means to a score of 4, representing 'No change'", 'groupDescription': 'Observed value at week 12 in OSU global improvement scale for autism (OSU CGI-I)', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': "A mixed effect model repeated measure (MMRM) was fitted to the observed values at week12 in OSU CGI-I. The model included dose group and visit as fixed effects, and the treatment-by-visit interaction. F-tests from PROC MIXED were based on Kenward-Roger's adjusted degrees of freedom. A compound symmetry covariance matrix was used for the repeated visits within subject."}, {'pValue': '1.0000', 'groupIds': ['OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '4.0', 'ciLowerLimit': '3.6', 'ciUpperLimit': '4.4', 'pValueComment': "p-values are presented for the comparison of adjusted Least Square Means to a score of 4, representing 'No change'", 'groupDescription': 'Observed value at week 12 in OSU global improvement scale for autism (OSU CGI-I)', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': "A mixed effect model repeated measure (MMRM) was fitted to the observed values at week12 in OSU CGI-I. The model included dose group and visit as fixed effects, and the treatment-by-visit interaction. F-tests from PROC MIXED were based on Kenward-Roger's adjusted degrees of freedom. A compound symmetry covariance matrix was used for the repeated visits within subject."}], 'paramType': 'MEAN', 'timeFrame': '12 weeks', 'description': 'The OSU Autism CGI scale contains separate subscales for symptom severity and for global improvement. These are rated in a similar way to the National Institute of Mental Health (NIMH) CGI Severity scale, but it is focused on autism spectrum symptoms. OSU Autism CGI-severity and OSU Autism CGI-improvement scores use a seven point Likert rating scale from 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, 7 = very much worse.', 'unitOfMeasure': 'Units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set'}, {'type': 'SECONDARY', 'title': 'Clinician-completed Domain Specific Cause for Concern Visual Analogue Scale (VAS): Myotonic Dystrophy', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1', 'description': '1000 mg tideglusib'}, {'id': 'OG001', 'title': 'Cohort 2', 'description': '400 mg tideglusib'}], 'classes': [{'title': 'VAS total score (observed value at week 12)', 'categories': [{'measurements': [{'value': '51.19', 'spread': '8.798', 'groupId': 'OG000'}, {'value': '59.28', 'spread': '17.450', 'groupId': 'OG001'}]}]}, {'title': 'VAS total score (change from baseline at week 12)', 'categories': [{'measurements': [{'value': '-5.80', 'spread': '4.855', 'groupId': 'OG000'}, {'value': '-3.38', 'spread': '2.726', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-5.91', 'ciLowerLimit': '-8.35', 'ciUpperLimit': '-3.47', 'estimateComment': 'The mean difference is an adjusted Least Square Mean', 'groupDescription': 'Change from baseline at week 12 in the clinician-completed domain specific causes for concern VAS total score (cm)', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values.'}, {'pValue': '0.0116', 'groupIds': ['OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-3.27', 'ciLowerLimit': '-5.71', 'ciUpperLimit': '-0.83', 'estimateComment': 'The mean difference is an adjusted Least Square Mean', 'groupDescription': 'Change from baseline at week 12 in the clinician-completed domain specific causes for concern VAS total score (cm)', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'Analysis of change in efficacy variables from baseline to week 12 were performed using mixed effect model repeated measures (MMRM) with baseline value as a covariate, dose group and visit as fixed effects and a treatment-by-visit interaction. Adjusted least square mean estimates were produced by dose group and study visit and were presented with two-sided 95% confidence intervals and p-values.'}], 'paramType': 'MEAN', 'timeFrame': '12 weeks', 'description': 'The Clinician-completed Domain Specific Causes for Concern is a Visual Analogue Scale completed by the clinician that scores the severity of concerns of domains that are clinically relevant in myotonic dystrophy. The severity of the clinician\'s concern is scored by using a 10 cm visual analogue scale (VAS), with anchors of "not at all severe" at the left end (0 cm) and "very severe" at the right end (10 cm). The clinician is asked to make a vertical line indicating his/her level of concern in each domain, using a time frame of the past week for reference. A score is to be determined by measuring the number of centimeters on the 10 cm VAS line from the anchor point on the left side of the line. A total VAS score for each subject was calculated as the sum of the scores for the 17 domains (minimum = 0, maximum = 170 cm). A higher score represents a worse outcome.', 'unitOfMeasure': 'Score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set'}, {'type': 'SECONDARY', 'title': 'Peabody Picture Vocabulary Test (PPVT)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1', 'description': '1000 mg tideglusib'}, {'id': 'OG001', 'title': 'Cohort 2', 'description': '400 mg tideglusib'}], 'classes': [{'title': 'Age-based standard score (observed value at week 12)', 'categories': [{'measurements': [{'value': '63.8', 'spread': '15.81', 'groupId': 'OG000'}, {'value': '79.8', 'spread': '22.23', 'groupId': 'OG001'}]}]}, {'title': 'Age-based standard score (change from baseline at week 12)', 'categories': [{'measurements': [{'value': '-0.1', 'spread': '7.97', 'groupId': 'OG000'}, {'value': '-1.1', 'spread': '5.57', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.6631', 'groupIds': ['OG000'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-1.1', 'ciLowerLimit': '-6.5', 'ciUpperLimit': '4.3', 'estimateComment': 'The mean difference is an adjusted Least Square Mean', 'groupDescription': 'Change from baseline to week 12 in the peabody picture vocabulary test age-based standard score', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'An ANCOVA was fitted to the change in the Peabody Picture Vocabulary Test age-based standard score from baseline to end of treatment. The model included the baseline value as a covariate and dose group as a fixed effect.'}, {'pValue': '0.9546', 'groupIds': ['OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.1', 'ciLowerLimit': '-5.5', 'ciUpperLimit': '5.2', 'estimateComment': 'The mean difference is an adjusted Least Square Mean', 'groupDescription': 'Change from baseline to week 12 in the peabody picture vocabulary test age-based standard score', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'An ANCOVA was fitted to the change in the Peabody Picture Vocabulary Test age-based standard score from baseline to end of treatment. The model included the baseline value as a covariate and dose group as a fixed effect.'}], 'paramType': 'MEAN', 'timeFrame': '12 weeks', 'description': "The PPVT-4 scale is a norm-referenced instrument for measuring the receptive (hearing) vocabulary of children and adults. It contains training items and 228 test items, each consisting of four full-colour pictures as response options on a page. For each item, the examiner says a word, and the examinee responds by selecting the picture that best illustrates that word's meaning. Each administration of the test produces a raw score (number of test items answered correctly), which can be converted to a standard score (using age-based norms) with a mean of 100 and a standard deviation of 15. Higher scores mean a better performance/receptive vocabulary.", 'unitOfMeasure': 'Units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set'}, {'type': 'SECONDARY', 'title': 'Biomarker - Lymphocyte GSK3β Levels and Activity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1', 'description': '1000 mg tideglusib'}, {'id': 'OG001', 'title': 'Cohort 2', 'description': '400 mg tideglusib'}], 'classes': [{'categories': [{'measurements': [{'value': '1.42', 'spread': '0.81', 'groupId': 'OG000'}, {'value': '1.95', 'spread': '0.79', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': '12 weeks', 'description': 'Total levels of GSK3β protein was determined via x-MAP technology in a Luminex 200 platform using the AKT Pathway Total Multispecies 7-Plex Panel from ThermoFisher Scientific.', 'unitOfMeasure': 'microgram per microliter', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Cohort 1', 'description': 'Subjects were given tideglusib 1000 mg for 12 weeks (weeks 0 to 12)'}, {'id': 'FG001', 'title': 'Cohort 2', 'description': 'Subjects were given tideglusib 400 mg for 12 weeks (weeks 0 to 12)'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}, {'groupId': 'FG001', 'numSubjects': '8'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}, {'groupId': 'FG001', 'numSubjects': '8'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}], 'preAssignmentDetails': 'Subjects were to be male or female aged 12 to 45 years with a diagnosis of genetically confirmed congenital or juvenile-onset type 1 myotonic dystrophy. Subjects were to have a Clinical Global Impression- Severity (CGI-S) score of 4 or greater at Screening and Run-in (V2) and were to be ambulatory and able to complete the 10-metre walk/run test.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '16', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Cohort 1', 'description': '1000 mg tideglusib'}, {'id': 'BG001', 'title': 'Cohort 2', 'description': '400 mg tideglusib'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Customized', 'classes': [{'title': 'Mean', 'categories': [{'measurements': [{'value': '21.8', 'spread': '5.5', 'groupId': 'BG000'}, {'value': '20.3', 'spread': '6.36', 'groupId': 'BG001'}, {'value': '21', 'spread': '5.8', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '7', 'groupId': 'BG001'}, {'value': '10', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'Not Hispanic or latino', 'categories': [{'measurements': [{'value': '8', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '16', 'groupId': 'BG002'}]}]}, {'title': 'White', 'categories': [{'measurements': [{'value': '8', 'groupId': 'BG000'}, {'value': '7', 'groupId': 'BG001'}, {'value': '15', 'groupId': 'BG002'}]}]}, {'title': 'Asian', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2017-01-09', 'size': 4121349, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2025-06-05T09:41', 'hasProtocol': True}, {'date': '2017-12-01', 'size': 2063334, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2025-06-05T09:41', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'SINGLE', 'whoMasked': ['PARTICIPANT']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 16}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2016-07-20', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'nctId': 'NCT07119775', 'statusForNctId': 'AVAILABLE', 'hasExpandedAccess': True}, 'statusVerifiedDate': '2025-08', 'dispFirstSubmitDate': '2018-12-20', 'completionDateStruct': {'date': '2018-01', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-08-22', 'studyFirstSubmitDate': '2016-08-04', 'dispFirstSubmitQcDate': '2018-12-20', 'resultsFirstSubmitDate': '2025-06-05', 'studyFirstSubmitQcDate': '2016-08-04', 'dispFirstPostDateStruct': {'date': '2018-12-27', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2025-09-11', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2025-08-22', 'studyFirstPostDateStruct': {'date': '2016-08-08', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2025-09-11', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2018-01', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Safety (Adverse Events)', 'timeFrame': '12 weeks', 'description': 'Incidence of Adverse events (AEs), including serious adverse events (SAEs), between baseline to end of study.'}], 'secondaryOutcomes': [{'measure': 'Plasma Concentration of Tideglusib', 'timeFrame': '12 weeks', 'description': 'Pharmacokinetic samples were collected to determine Tideglusib plasma concentration'}, {'measure': 'Blood Pharmacokinetics of Tideglusib', 'timeFrame': '12 weeks', 'description': 'Pharmacokinetic samples were collected to determine time of the maximum plasma concentration and terminal elimination half-life of tideglusib'}, {'measure': 'Area Under the Plasma Concentration vs. Time Curve of Tideglusib', 'timeFrame': '12 weeks', 'description': 'Pharmacokinetic samples were collected to determine area under the plasma concentration vs. time curve from 0 to 12 h and area under the plasma concentration vs. time curve from 0 to 24 h of tideglusib'}, {'measure': '10 Metre Walk/Run Test', 'timeFrame': '12 weeks', 'description': 'The 10-metre walk/run test is a performance measure used to assess walking speed in metres per second over a short distance and was used as an assessment of functional mobility. Time taken to complete the 10m walk/run test at fastest and preferred speed is measured.'}, {'measure': 'Computerised Handgrip Myometer Measure of Grip Strength and Muscle Relaxation Time', 'timeFrame': '12 weeks', 'description': 'Handgrip myometry is used as a measure of myotonia and muscle strength for the dominant hand'}, {'measure': 'Respiratory Forced Vital Capacity (FVC)', 'timeFrame': '12 weeks', 'description': 'FVC is a measure of lung function (the total amount of air exhaled during a Forced Expiratory Volume is measured using a spirometer)'}, {'measure': 'Dual-energy X-ray Absorptiometry (DXA)', 'timeFrame': '12 weeks', 'description': "DXA utilises two low energy X-ray beams, with different energy levels, which are aimed at the subject's bones. The DXA scan is more typically used to measure bone mineral density, however it can also be used to measure total lean muscle mass"}, {'measure': 'Clinical Global Impressions- Severity (CGI-S)', 'timeFrame': '12 weeks', 'description': "The CGI-S is a 7-point Likert type scale that requires the clinician to rate the severity of the subject's illness at the time of assessment, relative to the clinician's past experience with subjects who have the same diagnosis. Considering total clinical experience, a subject is assessed on severity of illness at the time of rating 1, normal, not at all ill; 2, borderline ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill. Change in CGI-S was observed in only 1 subject. Consequently statistical analysis was not conducted."}, {'measure': 'Clinical Global Impressions- Improvement (CGI-I)', 'timeFrame': '12 weeks', 'description': "The CGI-I requires the clinician to rate how much the subject's illness has improved or worsened relative to a baseline state.\n\nA seven point Likert type scale is used from 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, 7 = very much worse."}, {'measure': 'Actigraphy (3-minute Bouts of Activity)', 'timeFrame': '12 weeks', 'description': "Actigraphy is a non-invasive method of monitoring physical activity via an actigraph device. The actigraph device was worn on the waist during waking hours, according to the device's instruction manual."}, {'measure': 'Actigraphy (>10-minute Bouts of Activity)', 'timeFrame': '12 weeks', 'description': "Actigraphy is a non-invasive method of monitoring physical activity via an actigraph device. The actigraph device was worn on the waist during waking hours, according to the device's instruction manual."}, {'measure': 'Actigraphy (Steps)', 'timeFrame': '12 weeks', 'description': "Actigraphy is a non-invasive method of monitoring physical activity via an actigraph device. The actigraph device was worn on the waist during waking hours, according to the device's instruction manual."}, {'measure': 'Nine Hole Peg Test (NHPT)', 'timeFrame': '12 weeks', 'description': 'Measure of fine manual dexterity. Time to taken to complete the NHPT (dominant hand) is recorded.'}, {'measure': 'Top 3 Concerns Visual Analogue Scale (VAS) Score', 'timeFrame': '12 weeks', 'description': 'The Top 3 concerns VAS allows caregivers to identify their main three causes of concern, related to the subject\'s myotonic dystrophy, rather than these being pre-specified within a scale and then rating how these concerns have changed at specific time-points during the study. Subjects, where possible, and caregivers were asked to rate three causes for concern by drawing a vertical mark on a 10 cm long visual analogue scale with anchors of "not at all severe" at the left end (0 cm) and "very severe" at the right end (10 cm). A score for each concern was to be determined by measuring the number of centimeters on the 10 cm VAS line from the anchor point on the left side of the line. A total VAS score for each subject was calculated as the sum of the scores for the 3 concerns (minimum = 0 cm, maximum = 30 cm). A higher score represents a worse outcome.'}, {'measure': 'Ohio State University (OSU) Autism Rating Scale (OARS)', 'timeFrame': '12 weeks', 'description': "The OARS-4 contains the autism signs and symptoms in the Diagnostic and Statistical Manual of Mental Disorders, 4th Edition. These were to be rated with the degree of impairment the subject experiences for the given symptom. The symptoms were to be elicited in a semi-structured interview with the subject's primary caregiver. The assessor was to take both frequency/duration and degree of impairment into account and how much the item interferes with relationships, learning, and/or activities of daily living. The scores for this assessment were from 0 (Never or Rarely; Not a Problem) to 3 (Very Often; A Severe Problem): a score for each symptom of social impairment, communication impairment and restricted patterns were averaged to provide a total impairment score. A higher score represents a worse outcome (minimum = 0, maximum 3)."}, {'measure': 'Ohio State University (OSU) Autism Clinical Global Impression (CGI)', 'timeFrame': '12 weeks', 'description': 'The OSU Autism CGI scale contains separate subscales for symptom severity and for global improvement. These are rated in a similar way to the National Institute of Mental Health (NIMH) CGI Severity scale, but it is focused on autism spectrum symptoms. OSU Autism CGI-severity and OSU Autism CGI-improvement scores use a seven point Likert rating scale from 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, 7 = very much worse.'}, {'measure': 'Clinician-completed Domain Specific Cause for Concern Visual Analogue Scale (VAS): Myotonic Dystrophy', 'timeFrame': '12 weeks', 'description': 'The Clinician-completed Domain Specific Causes for Concern is a Visual Analogue Scale completed by the clinician that scores the severity of concerns of domains that are clinically relevant in myotonic dystrophy. The severity of the clinician\'s concern is scored by using a 10 cm visual analogue scale (VAS), with anchors of "not at all severe" at the left end (0 cm) and "very severe" at the right end (10 cm). The clinician is asked to make a vertical line indicating his/her level of concern in each domain, using a time frame of the past week for reference. A score is to be determined by measuring the number of centimeters on the 10 cm VAS line from the anchor point on the left side of the line. A total VAS score for each subject was calculated as the sum of the scores for the 17 domains (minimum = 0, maximum = 170 cm). A higher score represents a worse outcome.'}, {'measure': 'Peabody Picture Vocabulary Test (PPVT)', 'timeFrame': '12 weeks', 'description': "The PPVT-4 scale is a norm-referenced instrument for measuring the receptive (hearing) vocabulary of children and adults. It contains training items and 228 test items, each consisting of four full-colour pictures as response options on a page. For each item, the examiner says a word, and the examinee responds by selecting the picture that best illustrates that word's meaning. Each administration of the test produces a raw score (number of test items answered correctly), which can be converted to a standard score (using age-based norms) with a mean of 100 and a standard deviation of 15. Higher scores mean a better performance/receptive vocabulary."}, {'measure': 'Biomarker - Lymphocyte GSK3β Levels and Activity', 'timeFrame': '12 weeks', 'description': 'Total levels of GSK3β protein was determined via x-MAP technology in a Luminex 200 platform using the AKT Pathway Total Multispecies 7-Plex Panel from ThermoFisher Scientific.'}]}, 'oversightModule': {'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Myotonic Dystrophy 1']}, 'referencesModule': {'references': [{'pmid': '32942085', 'type': 'DERIVED', 'citation': 'Horrigan J, Gomes TB, Snape M, Nikolenko N, McMorn A, Evans S, Yaroshinsky A, Della Pasqua O, Oosterholt S, Lochmuller H. A Phase 2 Study of AMO-02 (Tideglusib) in Congenital and Childhood-Onset Myotonic Dystrophy Type 1 (DM1). Pediatr Neurol. 2020 Nov;112:84-93. doi: 10.1016/j.pediatrneurol.2020.08.001. Epub 2020 Aug 5.'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to determine whether Tideglusib is safe and efficacious in the treatment of adolescents and adults with congenital and juvenile-onset Myotonic Dystrophy. The pharmacokinetics of tideglusib and its primary metabolite will also be investigated.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '45 Years', 'minimumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Adolescents or adults with diagnosis of congenital or juvenile-onset type 1 myotonic dystrophy (DM-1)\n* Diagnosis must be genetically confirmed\n* Subjects must be male or female aged 12 years to 45 years\n* Subjects must have a Clinical Global Impression - Severity (CGI-S) score of 4 or greater at Screening and Run-in (V2)\n* Subjects must be ambulatory and able to complete the 10 metre walk/run test (splints allowed)\n* Subject's legally authorized representative (LAR) must provide written informed consent and there must be written consent or assent (as age applicable and developmentally appropriate) by the subject before any study-related procedures are conducted\n\nExclusion Criteria:\n\n* Non-ambulatory (full time) wheel chair user\n* Receiving stimulant medication\n* Receiving other medications/therapies not stable (changed) within 4 weeks prior to Run-in (V2)\n* Medical illness or other concern which would cause investigator to conclude subjects will not be able to perform the study procedures or assessments or would confound interpretation of data obtained during assessment.\n* Current enrolment in a clinical trial of an investigational drug or enrolment in a clinical trial of an investigational drug in the last 6 months\n* Women of child bearing potential who are pregnant, lactating or not willing to use a protocol defined acceptable contraception method if sexually active and not surgically sterile.\n* Gastrointestinal disease which may interfere with the absorption, distribution, metabolism or excretion of the study medication and impact the interpretability of the study results\n* Current clinically significant (as determined by the investigator) cardiovascular, renal, hepatic, endocrine or respiratory disease\n* Clinically significant heart disease (in the opinion of the investigator) or second or third degree heart block, atrial flutter, atrial fibrillation, ventricular arrhythmias, or is receiving medication for treatment of a cardiac arrhythmia\n* A history of chronic liver disease with current out of range values for Alanine transaminase (ALT), clinically relevant hepatic steatosis or other clinical manifestations of ongoing liver disease\n* A history of significant drug allergy (such as Steven-Johnson syndrome, anaphylaxis)\n* A history of alcohol or substance use disorders"}, 'identificationModule': {'nctId': 'NCT02858908', 'briefTitle': 'Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy', 'organization': {'class': 'INDUSTRY', 'fullName': 'AMO Pharma Limited'}, 'officialTitle': 'A Single-Blind, Phase 2 Study To Evaluate The Safety And Efficacy Of Tideglusib 400mg Or 1000mg For The Treatment Of Adolescent And Adult Congenital And Juvenile-Onset Myotonic Dystrophy', 'orgStudyIdInfo': {'id': 'AMO-02-MD-2-001'}, 'secondaryIdInfos': [{'id': '2016-000067-16', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Cohort 1 - Tideglusib', 'description': '1000 mg tideglusib, orally, once daily', 'interventionNames': ['Drug: Tideglusib']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 2 - Tideglusib', 'description': '400 mg tideglusib, orally, once daily', 'interventionNames': ['Drug: Tideglusib']}], 'interventions': [{'name': 'Tideglusib', 'type': 'DRUG', 'description': 'Tideglusib for oral suspension,', 'armGroupLabels': ['Cohort 1 - Tideglusib', 'Cohort 2 - Tideglusib']}]}, 'contactsLocationsModule': {'locations': [{'zip': 'NE1 4LP', 'city': 'Newcastle upon Tyne', 'state': 'Tyne and Wear', 'country': 'United Kingdom', 'facility': 'Newcastle-upon-Tyne Hospitals NHS Trust', 'geoPoint': {'lat': 54.97328, 'lon': -1.61396}}], 'overallOfficials': [{'name': 'Grainne Gorman, MB BCh BAO LRCP&SI MRCP FRCP', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Institute of Neuroscience, Newcastle University.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'AMO Pharma Limited', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}