Ignite Creation Date:
2025-12-26 @ 11:08 AM
Ignite Modification Date:
2026-02-22 @ 7:22 PM
Study NCT ID:
NCT03692312
Status:
COMPLETED
Last Update Posted:
2025-10-08
First Post:
2018-03-16
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2025-06-19', 'type': 'ACTUAL'}}}}, '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}, 'limitationsAndCaveats': {'description': 'None reported'}}, 'adverseEventsModule': {'timeFrame': 'Consent to end of study, up to 28 weeks', 'description': 'A participant assigned to placebo was found to have some tideglusib levels (due to inadvertent exposure between siblings in opposing arms), and was therefore, included in the safety analysis and full analysis sets for tideglusib.', 'eventGroups': [{'id': 'EG000', 'title': 'Tideglusib', 'description': 'Weight adjusted tideglusib, orally, once daily\n\nTideglusib for oral suspension, weight-adjusted at 400 mg for 2 weeks, then up-titrated to a weight-adjusted 600 mg for 2 weeks, after which 1000 mg was administered for the remainder of the treatment period, once daily', 'otherNumAtRisk': 28, 'deathsNumAtRisk': 28, 'otherNumAffected': 20, 'seriousNumAtRisk': 28, 'deathsNumAffected': 0, 'seriousNumAffected': 1}, {'id': 'EG001', 'title': 'Placebo', 'description': 'Matching placebo, orally, once daily\n\nPlacebo: Matching placebo formulation', 'otherNumAtRisk': 25, 'deathsNumAtRisk': 25, 'otherNumAffected': 19, 'seriousNumAtRisk': 25, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Nasopharyngytitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 4, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 25, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 4, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 25, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'COVID-19', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 25, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 25, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Rhinorrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 25, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 25, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 25, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Diarrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 5, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 25, 'numEvents': 6, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 25, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 25, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Skin abrasion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 25, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Vaccination site pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 25, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Weight decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 25, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 25, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 25, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'seriousEvents': [{'term': 'Scoliosis Surgery', 'stats': [{'groupId': 'EG000', 'numAtRisk': 28, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 25, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Surgical and medical procedures', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}, {'value': '26', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Tideglusib', 'description': 'Weight adjusted tideglusib, orally, once daily\n\nTideglusib for oral suspension, weight-adjusted at 400 mg for 2 weeks, then up-titrated to a weight-adjusted 600 mg for 2 weeks, after which 1000 mg was administered for the remainder of the treatment period, once daily'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Matching placebo, orally, once daily\n\nPlacebo: Matching placebo formulation'}], 'classes': [{'categories': [{'measurements': [{'value': '-1.65', 'spread': '0.620', 'groupId': 'OG000'}, {'value': '-3.40', 'spread': '0.618', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0514', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '1.75', 'ciLowerLimit': '-0.01', 'ciUpperLimit': '3.51', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.875', 'groupDescription': 'Analysis is a mixed model for repeated measures (MMRM) analysis with treatment, visit, age group and treatment by visit interaction as fixed effects and baseline value as a covariate. An unstructured variance-covariance matrix has been used.', 'statisticalMethod': 'MMRM', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline and week 20', 'description': "The Clinician-Completed Congenital DM1 Scale is an 11-item rating scale completed by the clinician that scores the symptom severity of domains that are clinically relevant in Congenital DM1.\n\nThe severity of the clinician's concern in each domain is scored by using a 5-point Likert Scale. Scores range from 0 = Not present to 4 = Very severe.", 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change in Clinical Global Impression- Improvement Scale (CGI-I) Scores', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}, {'value': '26', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Tideglusib', 'description': 'Weight adjusted tideglusib, orally, once daily\n\nTideglusib for oral suspension, weight-adjusted at 400 mg for 2 weeks, then up-titrated to a weight-adjusted 600 mg for 2 weeks, after which 1000 mg was administered for the remainder of the treatment period, once daily'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Matching placebo, orally, once daily\n\nPlacebo: Matching placebo formulation'}], 'classes': [{'categories': [{'measurements': [{'value': '3.11', 'spread': '0.190', 'groupId': 'OG000'}, {'value': '2.77', 'spread': '0.189', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.2124', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.34', 'ciLowerLimit': '-0.20', 'ciUpperLimit': '0.88', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.268', 'groupDescription': 'Analysis is a mixed model for repeated measures (MMRM) analysis with treatment, visit, age group and treatment by visit interaction as fixed effects and baseline value as a covariate. An unstructured variance-covariance matrix has been used.', 'statisticalMethod': 'MMRM', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline and week 20', 'description': "The clinician administered CGI-I rates how much the subject's illness has improved or worsened relative to a baseline state.\n\nA 7-point Likert type scale is used with ratings of 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, 7 = very much worse.", 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'A subject (randomized to tideglusib) withdrew consent on Day 12 of randomized treatment and was excluded from the intent-to-treat analysis, full and per-protocol analysis sets as they did not have a post-baseline efficacy assessment.'}, {'type': 'SECONDARY', 'title': 'Change in Top 3 Caregiver Concerns Visual Analogue Scale (VAS) Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}, {'value': '25', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Tideglusib', 'description': 'Weight adjusted tideglusib, orally, once daily\n\nTideglusib for oral suspension, weight-adjusted at 400 mg for 2 weeks, then up-titrated to a weight-adjusted 600 mg for 2 weeks, after which 1000 mg was administered for the remainder of the treatment period, once daily'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Matching placebo, orally, once daily\n\nPlacebo: Matching placebo formulation'}], 'classes': [{'categories': [{'measurements': [{'value': '-1.74', 'spread': '0.780', 'groupId': 'OG000'}, {'value': '-6.47', 'spread': '0.780', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '4.74', 'ciLowerLimit': '2.51', 'ciUpperLimit': '6.96', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '1.104', 'groupDescription': 'Analysis is a mixed model for repeated measures (MMRM) analysis with treatment, visit, age group and treatment by visit interaction as fixed effects and baseline value as a covariate. An unstructured variance-covariance matrix has been used.', 'statisticalMethod': 'MMRM', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline and week 20', '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. Caregivers were asked to rate three causes for concern by drawing a vertical mark on a 10 cm long VAS 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 Error', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}, {'value': '25', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Tideglusib', 'description': 'Weight adjusted tideglusib, orally, once daily\n\nTideglusib for oral suspension, weight-adjusted at 400 mg for 2 weeks, then up-titrated to a weight-adjusted 600 mg for 2 weeks, after which 1000 mg was administered for the remainder of the treatment period, once daily'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Matching placebo, orally, once daily\n\nPlacebo: Matching placebo formulation'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.18', 'spread': '0.770', 'groupId': 'OG000'}, {'value': '-3.36', 'spread': '0.782', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0059', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '3.18', 'ciLowerLimit': '0.96', 'ciUpperLimit': '5.39', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '1.099', 'groupDescription': 'Analysis is a mixed model for repeated measures (MMRM) analysis with treatment, visit, age group and treatment by visit interaction as fixed effects and baseline value as a covariate. An unstructured variance-covariance matrix has been used.', 'statisticalMethod': 'MMRM', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline and week 20', 'description': 'The Caregiver-Completed Congenital DM1 Scale is a caregiver assessment of the subject on symptoms that may occur in individuals with CDM1. There are a total of 11 clinically relevant symptoms that the caregiver is asked to rate the severity of.\n\nThe symptoms are rated on a score from 0 to 4 based on overall severity where 0 = symptom not present or is no longer present during the relevant time frame, and 4 = very severe, symptom causes pronounced and consistent impairment and is highly disruptive with regard to daily life. A total CC-CDM1-RS score for each subject was calculated as the sum of the scores where 0 = min and 44 = max. A higher score represents a worse outcome.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Clinical Global Impression - Severity Scale (CGI-S)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}, {'value': '25', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Tideglusib', 'description': 'Weight adjusted tideglusib, orally, once daily\n\nTideglusib for oral suspension, weight-adjusted at 400 mg for 2 weeks, then up-titrated to a weight-adjusted 600 mg for 2 weeks, after which 1000 mg was administered for the remainder of the treatment period, once daily'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Matching placebo, orally, once daily\n\nPlacebo: Matching placebo formulation'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.20', 'spread': '0.076', 'groupId': 'OG000'}, {'value': '-0.12', 'spread': '0.076', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.4634', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.08', 'ciLowerLimit': '-0.30', 'ciUpperLimit': '0.14', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.108', 'groupDescription': 'Analysis is a mixed model for repeated measures (MMRM) analysis with treatment, visit, age group and treatment by visit interaction as fixed effects and baseline value as a covariate. An unstructured variance-covariance matrix has been used.', 'statisticalMethod': 'MMRM', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline and week 20', 'description': "The Clinical Global Impression - Severity Scale (CGI-S) is a 7-point 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.\n\nSubjects are 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.", 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': '10-meter Walk-run Test', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}, {'value': '25', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Tideglusib', 'description': 'Weight adjusted tideglusib, orally, once daily\n\nTideglusib for oral suspension, weight-adjusted at 400 mg for 2 weeks, then up-titrated to a weight-adjusted 600 mg for 2 weeks, after which 1000 mg was administered for the remainder of the treatment period, once daily'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Matching placebo, orally, once daily\n\nPlacebo: Matching placebo formulation'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.54', 'spread': '0.393', 'groupId': 'OG000'}, {'value': '-0.19', 'spread': '0.391', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.5385', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.35', 'ciLowerLimit': '-1.49', 'ciUpperLimit': '0.79', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.564', 'groupDescription': 'Analysis is a mixed model for repeated measures (MMRM) analysis with treatment, visit, age group and treatment by visit interaction as fixed effects and baseline value as a covariate. An unstructured variance-covariance matrix has been used.', 'statisticalMethod': 'MMRM', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': '20 weeks', 'description': 'The 10-meter walk/run test is a performance measure used to assess walking speed in seconds over a short distance. It can be used as an assessment of functional mobility.', 'unitOfMeasure': 'Seconds', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of Adverse Events (AEs), Including Serious Adverse Events (SAEs), Between Screening to End of Study.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '28', 'groupId': 'OG000'}, {'value': '25', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Tideglusib', 'description': 'Weight adjusted tideglusib, orally, once daily\n\nTideglusib for oral suspension, weight-adjusted at 400 mg for 2 weeks, then up-titrated to a weight-adjusted 600 mg for 2 weeks, after which 1000 mg was administered for the remainder of the treatment period, once daily'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Matching placebo, orally, once daily\n\nPlacebo: Matching placebo formulation'}], 'classes': [{'title': 'Total number of TEAEs', 'categories': [{'measurements': [{'value': '20', 'groupId': 'OG000'}, {'value': '19', 'groupId': 'OG001'}]}]}, {'title': 'Total number of serious TEAEs', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Total number of serious TEAEs related to treatment', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Total number of subjects with TEAEs leading to discontinuation from the study', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Total number of subjects with treatment-related TEAEs leading to discontinuation from the study', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Total number of subjects with TEAEs leading to death', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Severity: Mild', 'categories': [{'measurements': [{'value': '13', 'groupId': 'OG000'}, {'value': '15', 'groupId': 'OG001'}]}]}, {'title': 'Severity: Moderate', 'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}]}, {'title': 'Severity: Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Unrelated to active treatment', 'categories': [{'measurements': [{'value': '18', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}]}, {'title': 'Related to active treatment', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Between Screening to End of Study, up to 28 weeks', 'description': 'Adverse events may be volunteered spontaneously by the subject, or discovered as a result of general, non-leading questioning by physician.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'A participant assigned to placebo was found to have some tideglusib levels (due to inadvertent exposure between siblings in opposing arms), and was therefore, included in the safety analysis and full analysis sets for tideglusib.'}, {'type': 'SECONDARY', 'title': 'Number of Abnormal Findings in Objective Assessments (e.g. Laboratory Values, ECGs, Vital Signs and Bone Mineral Density) Between Screening and End of Study.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '28', 'groupId': 'OG000'}, {'value': '25', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Tideglusib', 'description': 'Weight adjusted tideglusib, orally, once daily\n\nTideglusib for oral suspension, weight-adjusted at 400 mg for 2 weeks, then up-titrated to a weight-adjusted 600 mg for 2 weeks, after which 1000 mg was administered for the remainder of the treatment period, once daily'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Matching placebo, orally, once daily\n\nPlacebo: Matching placebo formulation'}], 'classes': [{'title': 'Alanine aminotransferase increased', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Haemoglobin decreased', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Transaminases increased', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Weight decreased', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Between Screening to End of Study, up to 28 weeks', 'description': 'Abnormal laboratory findings (e.g. hematology, liver function, biochemistry, urinalysis) or other abnormal assessments (e.g. ECGs, vital signs) that are judged by the Investigator as clinically significant will be recorded as AEs or SAEs if they meet the definition of an AE. The Investigator will exercise his or her medical and scientific judgment in deciding whether an abnormal laboratory finding or other abnormal assessment is clinically significant.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'A participant assigned to placebo was found to have some tideglusib levels (due to inadvertent exposure between siblings in opposing arms), and was therefore, included in the safety analysis and full analysis sets for tideglusib.'}, {'type': 'SECONDARY', 'title': 'CDM1-RS Independent Central Rater Score (CDM1-RS)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '20', 'groupId': 'OG000'}, {'value': '19', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Tideglusib', 'description': 'Weight adjusted tideglusib, orally, once daily\n\nTideglusib for oral suspension, weight-adjusted at 400mg for 2 weeks, then up-titrated to a weight-adjusted 600mg for 2 weeks, after which 1000 mg was administered for the remainder of the treatment period, once daily'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Matching placebo, orally, once daily\n\nPlacebo: Matching placebo formulation'}], 'classes': [{'categories': [{'measurements': [{'value': '-2.26', 'spread': '0.533', 'groupId': 'OG000'}, {'value': '-2.55', 'spread': '0.547', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.7110', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.29', 'ciLowerLimit': '-1.26', 'ciUpperLimit': '1.83', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.764', 'groupDescription': 'Analysis is a mixed model for repeated measures (MMRM) analysis with treatment, visit, age group and treatment by visit interaction as fixed effects and baseline value as a covariate. An unstructured variance-covariance matrix has been used.', 'statisticalMethod': 'MMRM', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline to week 20', 'description': "Change from baseline to end of treatment in the independent central rater CDM1-RS total score. CDM1 Rating Scale is an 11-item rating scale completed by the clinician to score the symptom severity that are clinically relevant in CDM1.\n\nThe severity of the clinician's concern in each domain is scored by using a 5-point Likert Scale. Scores range from 0 = Not present to 4 = Very severe.", 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'A subject (randomized to tideglusib) withdrew consent on Day 12 of randomized treatment and was excluded from the intent-to-treat analysis, full and per-protocol analysis sets as they did not have a post-baseline efficacy assessment.'}, {'type': 'SECONDARY', 'title': 'CGI-I Independent Central Rater Score (CGI-I)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}, {'value': '23', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Tideglusib', 'description': 'Weight adjusted tideglusib, orally, once daily\n\nTideglusib for oral suspension, weight-adjusted at 400 mg for 2 weeks, then up-titrated to a weight-adjusted 600 mg for 2 weeks, after which 1000 mg was administered for the remainder of the treatment period, once daily'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Matching placebo, orally, once daily\n\nPlacebo: Matching placebo formulation'}], 'classes': [{'categories': [{'measurements': [{'value': '2.74', 'spread': '0.227', 'groupId': 'OG000'}, {'value': '2.65', 'spread': '0.234', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.7861', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.09', 'ciLowerLimit': '-0.57', 'ciUpperLimit': '0.75', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.326', 'groupDescription': 'Analysis is a mixed model for repeated measures (MMRM) analysis with treatment, visit, age group and treatment by visit interaction as fixed effects and baseline value as a covariate. An unstructured variance-covariance matrix has been used.', 'statisticalMethod': 'MMRM', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline and week 20', 'description': "The CGI-I requires the clinician to rate how much the subject's illness has changed (improved, worsened or stayed the same) relative to a baseline state on a seven point scale.\n\nA 7-point Likert type scale is used with ratings of 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, 7 = very much worse.", 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'A subject (randomized to tideglusib) withdrew consent on Day 12 of randomized treatment and was excluded from the intent-to-treat analysis, full and per-protocol analysis sets as they did not have a post-baseline efficacy assessment.'}, {'type': 'SECONDARY', 'title': 'Independent Rater Clinical Global Impression - Severity Scale (CGI-S)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'OG000'}, {'value': '19', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Tideglusib', 'description': 'Weight adjusted tideglusib, orally, once daily\n\nTideglusib for oral suspension, weight-adjusted at 400 mg for 2 weeks, then up-titrated to a weight-adjusted 600 mg for 2 weeks, after which 1000 mg was administered for the remainder of the treatment period, once daily'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Matching placebo, orally, once daily\n\nPlacebo: Matching placebo formulation'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.15', 'spread': '0.080', 'groupId': 'OG000'}, {'value': '-0.21', 'spread': '0.093', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.6282', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.06', 'ciLowerLimit': '-0.19', 'ciUpperLimit': '0.31', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.123', 'groupDescription': 'Analysis is a mixed model for repeated measures (MMRM) analysis with treatment, visit, age group and treatment by visit interaction as fixed effects and baseline value as a covariate. A compound symmetry variance-covariance matrix has been used.', 'statisticalMethod': 'MMRM', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline and week 20', 'description': 'CGI-S is a 7-point Likert type scale. An independent central rater rated the CGI-S scales for both the in-clinic and telehealth interviews.\n\nSubjects are 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.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'A subject (randomized to tideglusib) withdrew consent on Day 12 of randomized treatment and was excluded from the intent-to-treat analysis, full and per-protocol analysis sets as they did not have a post-baseline efficacy assessment.'}, {'type': 'POST_HOC', 'title': 'MDRI Analysis', 'denoms': [{'units': 'Participants', 'counts': [{'value': '24', 'groupId': 'OG000'}, {'value': '24', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Tideglusib', 'description': 'Weight adjusted tideglusib, orally, once daily\n\nTideglusib for oral suspension, weight-adjusted at 400 mg for 2 weeks, then up-titrated to a weight-adjusted 600 mg for 2 weeks, after which 1000 mg was administered for the remainder of the treatment period, once daily'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Matching placebo, orally, once daily\n\nPlacebo: Matching placebo formulation'}], 'classes': [{'categories': [{'measurements': [{'value': '0.8', 'spread': '0.31', 'groupId': 'OG000'}, {'value': '-0.1', 'spread': '0.25', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0428', 'groupIds': ['OG000', 'OG001'], 'statisticalMethod': 't-test, 2 sided', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and week 20', 'description': 'A multi-domain responder index (MDRI) analysis was performed by combining 5 endpoints to objectively assess movement, muscle integrity and strength, cognitive skills and adaptive behavior. For each of the 5 endpoints, subjects were scored +1 point if their change exceeded or was equal to the threshold in a beneficial direction, -1 point if their change exceeded or was equal to the threshold in a detrimental direction and 0 points otherwise. The sum of these 5 scores were added together to provide an MDRI score between -5 and 5, where a higher score indicates greater overall improvement.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED'}, {'type': 'POST_HOC', 'title': 'Creatine Phosphokinase', 'denoms': [{'units': 'Participants', 'counts': [{'value': '24', 'groupId': 'OG000'}, {'value': '24', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Tideglusib', 'description': 'Weight adjusted tideglusib, orally, once daily\n\nTideglusib for oral suspension, weight-adjusted at 400 mg for 2 weeks, then up-titrated to a weight-adjusted 600 mg for 2 weeks, after which 1000 mg was administered for the remainder of the treatment period, once daily'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Matching placebo, orally, once daily\n\nPlacebo: Matching placebo formulation'}], 'classes': [{'categories': [{'measurements': [{'value': '0.81', 'groupId': 'OG000', 'lowerLimit': '0.68', 'upperLimit': '0.97'}, {'value': '1.05', 'groupId': 'OG001', 'lowerLimit': '0.89', 'upperLimit': '1.26'}]}]}], 'analyses': [{'pValue': '0.0379', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Ratio', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.77', 'ciLowerLimit': '0.60', 'ciUpperLimit': '0.98', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.094', 'groupDescription': 'Analysis is a mixed model for repeated measures (MMRM) analysis with treatment, visit, age group and treatment by visit interaction as fixed effects and baseline value as a covariate. An unstructured variance-covariance matrix has been used.', 'statisticalMethod': 'MMRM', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': '20 weeks', 'description': 'Analysis of Ratio to Baseline in Creatine Phosphokinase', 'unitOfMeasure': 'Ratio to Baseline', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'POST_HOC', 'title': 'Mean Group Difference in 10 Meter Walk/ Run', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Cmax Lower Quantiles', 'description': 'Patients who showed lower exposure levels to tideglusib'}, {'id': 'OG001', 'title': 'Cmax Upper Quantiles', 'description': 'Patients who showed higher exposure levels to tideglusib (50th percentile)'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Matching placebo, orally, once daily\n\nPlacebo: Matching placebo formulation'}], 'classes': [{'categories': [{'measurements': [{'value': '9.1', 'groupId': 'OG000', 'lowerLimit': '6.9', 'upperLimit': '16.1'}, {'value': '8.7', 'groupId': 'OG001', 'lowerLimit': '6.5', 'upperLimit': '12.2'}, {'value': '8.5', 'groupId': 'OG002', 'lowerLimit': '3.9', 'upperLimit': '18.7'}]}]}], 'paramType': 'MEAN', 'timeFrame': '20 weeks', 'description': 'Post-hoc analyses comparing the mean clinical responses of subjects with tideglusib exposures above the 50th percentile (who achieved the desired target exposure) with those subjects who showed lower exposure levels, and with the placebo group.\n\nThe 10-meter walk/run test is a performance measure used to assess walking speed in seconds over a short distance. It can be used as an assessment of functional mobility.', 'unitOfMeasure': 'Seconds', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED'}, {'type': 'POST_HOC', 'title': 'Mean Change From Baseline Group Difference in 10 Meter Walk/ Run', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Cmax Lower Quantiles', 'description': 'Patients who showed lower exposure levels to tideglusib'}, {'id': 'OG001', 'title': 'Cmax Upper Quantiles', 'description': 'Patients who showed higher exposure levels to tideglusib (50th percentile)'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Matching placebo, orally, once daily\n\nPlacebo: Matching placebo formulation'}], 'classes': [{'categories': [{'measurements': [{'value': '0.4', 'spread': '1.7', 'groupId': 'OG000'}, {'value': '-1.5', 'spread': '2.2', 'groupId': 'OG001'}, {'value': '-0.1', 'spread': '1.8', 'groupId': 'OG002'}]}]}], 'analyses': [{'pValue': '0.078', 'groupIds': ['OG001'], 'groupDescription': 'Cmax upper quantiles group compared to the placebo group for mean change from baseline; two-tailed t-test assuming unequal variance.', 'statisticalMethod': 't-test, 2 sided', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'MEAN', 'timeFrame': '20 weeks', 'description': 'Post-hoc analyses comparing the mean change from baseline from clinical responses of subjects with tideglusib exposures above the 50th percentile (who achieved the desired target exposure) with those subjects who showed lower exposure levels, and with the placebo group.\n\nThe 10-meter walk/run test is a performance measure used to assess walking speed in seconds over a short distance. It can be used as an assessment of functional mobility.', 'unitOfMeasure': 'Seconds', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'POST_HOC', 'title': 'Mean Group Difference in Peabody Picture Vocabulary Test (PPVT)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '19', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Cmax Lower Quantiles', 'description': 'Patients who showed lower exposure levels to tideglusib'}, {'id': 'OG001', 'title': 'Cmax Upper Quantiles', 'description': 'Patients who showed higher exposure levels to tideglusib (50th percentile)'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Matching placebo, orally, once daily\n\nPlacebo: Matching placebo formulation'}], 'classes': [{'categories': [{'measurements': [{'value': '109', 'groupId': 'OG000', 'lowerLimit': '35', 'upperLimit': '185'}, {'value': '134', 'groupId': 'OG001', 'lowerLimit': '53', 'upperLimit': '192'}, {'value': '130', 'groupId': 'OG002', 'lowerLimit': '51', 'upperLimit': '191'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and week 20', 'description': 'Post-hoc analyses comparing the mean clinical responses of subjects with tideglusib exposures above the 50th percentile (who achieved the desired target exposure) with those subjects who showed lower exposure levels, and with the placebo group. The PPVT-4 scale is a norm-referenced instrument for measuring the receptive (hearing) vocabulary. It contains training items and 228 test items, each consisting of four full-color pictures as response options on a page. Each test produces a raw score and a standard score. The raw score counts the number of correct responses. Raw scores are reported here as standardized scores are considered less appropriate for a pediatric population with cognitive deficits. Higher scores mean a better performance/receptive vocabulary. The lowest possible raw score is 0, the maximum raw score depends on the number of items administered so theoretically, the highest score possible on this test would be 228.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED'}, {'type': 'POST_HOC', 'title': 'Mean Change From Baseline Group Difference in Peabody Picture Vocabulary Test (PPVT)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '19', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Cmax Lower Quantiles', 'description': 'Patients who showed lower exposure levels to tideglusib'}, {'id': 'OG001', 'title': 'Cmax Upper Quantiles', 'description': 'Patients who showed higher exposure levels to tideglusib (50th percentile)'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Matching placebo, orally, once daily\n\nPlacebo: Matching placebo formulation'}], 'classes': [{'categories': [{'measurements': [{'value': '3', 'spread': '14', 'groupId': 'OG000'}, {'value': '6', 'spread': '17', 'groupId': 'OG001'}, {'value': '-2', 'spread': '10', 'groupId': 'OG002'}]}]}], 'analyses': [{'pValue': '0.29', 'groupIds': ['OG001'], 'groupDescription': 'Cmax upper quantiles group compared to the placebo group for mean change from baseline; two-tailed t-test assuming unequal variance.', 'statisticalMethod': 't-test, 2 sided', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to week 20', 'description': 'Post-hoc analyses comparing the mean change from baseline clinical responses of subjects with tideglusib exposures above the 50th percentile (who achieved the desired target exposure) with those subjects who showed lower exposure levels, and with the placebo group. The PPVT-4 scale is a norm-referenced instrument for measuring the receptive (hearing) vocabulary. It contains training items and 228 test items, each consisting of four full-color pictures as response options on a page. Each test produces a raw score and a standard score. The raw score counts the number of correct responses. Raw scores are reported here as standardized scores are considered less appropriate for a pediatric population with cognitive deficits. Higher scores mean a better performance/receptive vocabulary. The lowest possible raw score is 0, the maximum raw score depends on the number of items administered so theoretically, the highest score possible on this test would be 228.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'POST_HOC', 'title': 'Mean Group Difference in Creatine Phosphokinase', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Cmax Lower Quantiles', 'description': 'Patients who showed lower exposure levels to tideglusib'}, {'id': 'OG001', 'title': 'Cmax Upper Quantiles', 'description': 'Patients who showed higher exposure levels to tideglusib (50th percentile)'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Matching placebo, orally, once daily\n\nPlacebo: Matching placebo formulation'}], 'classes': [{'categories': [{'measurements': [{'value': '147', 'groupId': 'OG000', 'lowerLimit': '77', 'upperLimit': '273'}, {'value': '195', 'groupId': 'OG001', 'lowerLimit': '42', 'upperLimit': '460'}, {'value': '299', 'groupId': 'OG002', 'lowerLimit': '57', 'upperLimit': '987'}]}]}], 'paramType': 'MEAN', 'timeFrame': '20 weeks', 'description': 'Post-hoc analyses comparing the mean clinical responses of subjects with tideglusib exposures above the 50th percentile (who achieved the desired target exposure) with those subjects who showed lower exposure levels, and with the placebo group.\n\nConcentration of Creatine Phosphokinase (UI/L).', 'unitOfMeasure': 'IU/L', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED'}, {'type': 'POST_HOC', 'title': 'Mean Change From Baseline Group Difference in Creatine Phosphokinase', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Cmax Lower Quantiles', 'description': 'Patients who showed lower exposure levels to tideglusib'}, {'id': 'OG001', 'title': 'Cmax Upper Quantiles', 'description': 'Patients who showed higher exposure levels to tideglusib (50th percentile)'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Matching placebo, orally, once daily\n\nPlacebo: Matching placebo formulation'}], 'classes': [{'categories': [{'measurements': [{'value': '-17', 'spread': '27', 'groupId': 'OG000'}, {'value': '-65', 'spread': '147', 'groupId': 'OG001'}, {'value': '30', 'spread': '138', 'groupId': 'OG002'}]}]}], 'analyses': [{'pValue': '0.077', 'groupIds': ['OG001'], 'groupDescription': 'Cmax upper quantiles group compared to the placebo group for mean change from baseline (absolute value); two-tailed t-test assuming unequal variance.', 'statisticalMethod': 't-test, 2 sided', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'MEAN', 'timeFrame': '20 weeks', 'description': 'Post-hoc analyses comparing the mean change from baseline in clinical responses of subjects with tideglusib exposures above the 50th percentile (who achieved the desired target exposure) with those subjects who showed lower exposure levels, and with the placebo group.\n\nConcentration of Creatine Phosphokinase (UI/L).', 'unitOfMeasure': 'UI/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'POST_HOC', 'title': 'Mean Group Difference in MDRI', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Cmax Lower Quantiles', 'description': 'Patients who showed lower exposure levels to tideglusib'}, {'id': 'OG001', 'title': 'Cmax Upper Quantiles', 'description': 'Patients who showed higher exposure levels to tideglusib (50th percentile)'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Matching placebo, orally, once daily\n\nPlacebo: Matching placebo formulation'}], 'classes': [{'categories': [{'measurements': [{'value': '0.8', 'groupId': 'OG000', 'lowerLimit': '-1.0', 'upperLimit': '3.0'}, {'value': '0.9', 'groupId': 'OG001', 'lowerLimit': '-2.0', 'upperLimit': '4.0'}, {'value': '-0.2', 'groupId': 'OG002', 'lowerLimit': '-2.0', 'upperLimit': '3.0'}]}]}], 'analyses': [{'pValue': '0.071', 'groupIds': ['OG001'], 'groupDescription': 'Cmax upper quantiles group compared to placebo group for mean Raw Score; two-tailed t-test assuming unequal variance.', 'statisticalMethod': 't-test, 2 sided', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and week 20', 'description': 'Post-hoc analyses comparing the mean clinical responses of subjects above and below the 50th percentile of tideglusib exposure levels with the placebo group. A multi-domain responder index (MDRI) analysis was performed by combining 5 objective endpoints. For each of the 5 endpoints, subjects were scored +1 point if their change exceeded or was equal to the threshold in a beneficial direction, -1 point if their change exceeded or was equal to the threshold in a detrimental direction and 0 points otherwise. The sum of these 5 scores were added together to provide an MDRI score between -5 and 5, where a higher score indicates greater overall improvement.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Tideglusib', 'description': 'Weight adjusted tideglusib, orally, once daily\n\nTideglusib for oral suspension, weight-adjusted at 400 mg for 2 weeks, then up-titrated to a weight-adjusted 600 mg for 2 weeks, after which 1000 mg was administered for the remainder of the treatment period, once daily'}, {'id': 'FG001', 'title': 'Placebo', 'description': 'Matching placebo, orally, once daily\n\nPlacebo: Matching placebo formulation'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '27'}, {'groupId': 'FG001', 'numSubjects': '26'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '25'}, {'groupId': 'FG001', 'numSubjects': '25'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}], 'preAssignmentDetails': 'Children and adolescents (≥6 - ≤16 years) with a diagnosis of genetically confirmed congenital type 1 myotonic dystrophy. Subjects were to have a Clinical Global Impression-Severity score of ≥4 at Screening and V2, be ambulatory and able to complete the 10m walk/run test. Planned randomization of 56 subjects closed at 53 subjects due to fewer than expected discontinuations. A participant assigned to placebo was found to have some tideglusib levels and was included in analysis sets for tideglusib'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '28', 'groupId': 'BG000'}, {'value': '25', 'groupId': 'BG001'}, {'value': '53', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Tideglusib', 'description': 'Weight adjusted tideglusib, orally, once daily\n\nTideglusib for oral suspension, weight-adjusted at 400 mg for 2 weeks, then up-titrated to a weight-adjusted 600 mg for 2 weeks, after which 1000 mg was administered for the remainder of the treatment period, once daily'}, {'id': 'BG001', 'title': 'Placebo', 'description': 'Matching placebo, orally, once daily\n\nPlacebo: Matching placebo formulation'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '11.0', 'spread': '3.32', 'groupId': 'BG000'}, {'value': '11.0', 'spread': '3.61', 'groupId': 'BG001'}, {'value': '11.0', 'spread': '3.43', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Age, Customized', 'classes': [{'title': 'Median', 'categories': [{'measurements': [{'value': '10', 'groupId': 'BG000', 'lowerLimit': '6', 'upperLimit': '16'}, {'value': '11', 'groupId': 'BG001', 'lowerLimit': '6', 'upperLimit': '16'}, {'value': '10', 'groupId': 'BG002', 'lowerLimit': '6', 'upperLimit': '16'}]}]}], 'paramType': 'MEDIAN', 'unitOfMeasure': 'years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '9', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '18', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '19', 'groupId': 'BG000'}, {'value': '16', 'groupId': 'BG001'}, {'value': '35', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'Ethic origin n (%)', 'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '5', 'groupId': 'BG001'}, {'value': '8', 'groupId': 'BG002'}]}, {'title': 'Not Hispanic or latino', 'measurements': [{'value': '25', 'groupId': 'BG000'}, {'value': '20', 'groupId': 'BG001'}, {'value': '45', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'Race n (%)', 'categories': [{'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'American Indian or Alaska Native', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}, {'title': 'White', 'measurements': [{'value': '27', 'groupId': 'BG000'}, {'value': '18', 'groupId': 'BG001'}, {'value': '45', 'groupId': 'BG002'}]}, {'title': 'Other', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'The safety analysis set consisted of all randomized subjects who received at least one dose of study treatment. Subjects were presented by treatment actually received. A participant assigned to placebo was found to have some tideglusib levels (due to inadvertent exposure between siblings in opposing arms), and was therefore, included in the safety analysis and full analysis sets for tideglusib.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2023-02-02', 'size': 6081306, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2025-06-03T14:51', 'hasProtocol': True}, {'date': '2024-08-22', 'size': 8328946, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2025-06-04T09:52', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2', 'PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 56}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2021-03-03', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'nctId': 'NCT07119775', 'statusForNctId': 'AVAILABLE', 'hasExpandedAccess': True}, 'statusVerifiedDate': '2025-09', 'completionDateStruct': {'date': '2023-04-04', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-09-25', 'studyFirstSubmitDate': '2018-03-16', 'resultsFirstSubmitDate': '2025-06-05', 'studyFirstSubmitQcDate': '2018-09-26', 'lastUpdatePostDateStruct': {'date': '2025-10-08', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2025-08-22', 'studyFirstPostDateStruct': {'date': '2018-10-02', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2025-09-11', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2023-04-04', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)', 'timeFrame': 'Baseline and week 20', 'description': "The Clinician-Completed Congenital DM1 Scale is an 11-item rating scale completed by the clinician that scores the symptom severity of domains that are clinically relevant in Congenital DM1.\n\nThe severity of the clinician's concern in each domain is scored by using a 5-point Likert Scale. Scores range from 0 = Not present to 4 = Very severe."}], 'secondaryOutcomes': [{'measure': 'Change in Clinical Global Impression- Improvement Scale (CGI-I) Scores', 'timeFrame': 'Baseline and week 20', 'description': "The clinician administered CGI-I rates how much the subject's illness has improved or worsened relative to a baseline state.\n\nA 7-point Likert type scale is used with ratings of 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, 7 = very much worse."}, {'measure': 'Change in Top 3 Caregiver Concerns Visual Analogue Scale (VAS) Score', 'timeFrame': 'Baseline and week 20', '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. Caregivers were asked to rate three causes for concern by drawing a vertical mark on a 10 cm long VAS 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': 'Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS)', 'timeFrame': 'Baseline and week 20', 'description': 'The Caregiver-Completed Congenital DM1 Scale is a caregiver assessment of the subject on symptoms that may occur in individuals with CDM1. There are a total of 11 clinically relevant symptoms that the caregiver is asked to rate the severity of.\n\nThe symptoms are rated on a score from 0 to 4 based on overall severity where 0 = symptom not present or is no longer present during the relevant time frame, and 4 = very severe, symptom causes pronounced and consistent impairment and is highly disruptive with regard to daily life. A total CC-CDM1-RS score for each subject was calculated as the sum of the scores where 0 = min and 44 = max. A higher score represents a worse outcome.'}, {'measure': 'Clinical Global Impression - Severity Scale (CGI-S)', 'timeFrame': 'Baseline and week 20', 'description': "The Clinical Global Impression - Severity Scale (CGI-S) is a 7-point 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.\n\nSubjects are 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."}, {'measure': '10-meter Walk-run Test', 'timeFrame': '20 weeks', 'description': 'The 10-meter walk/run test is a performance measure used to assess walking speed in seconds over a short distance. It can be used as an assessment of functional mobility.'}, {'measure': 'Number of Adverse Events (AEs), Including Serious Adverse Events (SAEs), Between Screening to End of Study.', 'timeFrame': 'Between Screening to End of Study, up to 28 weeks', 'description': 'Adverse events may be volunteered spontaneously by the subject, or discovered as a result of general, non-leading questioning by physician.'}, {'measure': 'Number of Abnormal Findings in Objective Assessments (e.g. Laboratory Values, ECGs, Vital Signs and Bone Mineral Density) Between Screening and End of Study.', 'timeFrame': 'Between Screening to End of Study, up to 28 weeks', 'description': 'Abnormal laboratory findings (e.g. hematology, liver function, biochemistry, urinalysis) or other abnormal assessments (e.g. ECGs, vital signs) that are judged by the Investigator as clinically significant will be recorded as AEs or SAEs if they meet the definition of an AE. The Investigator will exercise his or her medical and scientific judgment in deciding whether an abnormal laboratory finding or other abnormal assessment is clinically significant.'}, {'measure': 'CDM1-RS Independent Central Rater Score (CDM1-RS)', 'timeFrame': 'Baseline to week 20', 'description': "Change from baseline to end of treatment in the independent central rater CDM1-RS total score. CDM1 Rating Scale is an 11-item rating scale completed by the clinician to score the symptom severity that are clinically relevant in CDM1.\n\nThe severity of the clinician's concern in each domain is scored by using a 5-point Likert Scale. Scores range from 0 = Not present to 4 = Very severe."}, {'measure': 'CGI-I Independent Central Rater Score (CGI-I)', 'timeFrame': 'Baseline and week 20', 'description': "The CGI-I requires the clinician to rate how much the subject's illness has changed (improved, worsened or stayed the same) relative to a baseline state on a seven point scale.\n\nA 7-point Likert type scale is used with ratings of 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, 7 = very much worse."}, {'measure': 'Independent Rater Clinical Global Impression - Severity Scale (CGI-S)', 'timeFrame': 'Baseline and week 20', 'description': 'CGI-S is a 7-point Likert type scale. An independent central rater rated the CGI-S scales for both the in-clinic and telehealth interviews.\n\nSubjects are 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.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Tideglusib', 'AMO-02-MD-2-003', 'Congenital Myotonic Dystrophy', 'Myotonic Dystrophy', 'Dystrophia Myotonica', 'Myotonia Atrophica', 'Myotonia Dystrophica', 'Myotonic Dystrophy, Congenital', 'Steinert Disease', 'Steinert Myotonic Dystrophy', "Steinert's Disease"], 'conditions': ['Congenital Myotonic Dystrophy']}, 'descriptionModule': {'briefSummary': 'This is a randomized, multicenter, double-blind, placebo-controlled, Phase 2/3 study of patients (aged 6 to 16 years) diagnosed with Congenital Myotonic Dystrophy (Congenital DM1).', 'detailedDescription': 'This is a randomized, double-blind, placebo controlled study of weight adjusted dose 1000 mg/day tideglusib versus placebo in the treatment of children and adolescents 6-16 years of age with Congenital DM1.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '16 Years', 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n1. Male or female children and adolescents aged ≥6 years and ≤16 years\n2. Diagnosis of Congenital DM1 (also known as Steinert's disease)\n\n * Diagnosis must be genetically confirmed\n * One or more of the following clinically relevant (e.g. requiring medical intervention) signs or symptoms was evident within the first month after birth:\n\n * Hypotonia\n * Generalized weakness\n * Respiratory insufficiency\n * Feeding difficulties\n * Clubfoot or another musculoskeletal deformity\n3. Subject must be able to walk and complete the 10-meter walk-run test (orthotics/splints allowed, forearm crutches are not allowed)\n4. Written, voluntary informed consent must be obtained before any study related procedures are conducted.\n\n * Where a parent or LAR provides consent, there must also be assent from the subject\n5. Subject's caregiver must be willing and able to support participation for duration of study\n6. Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol\n\nExclusion Criteria:\n\n1. Not able to walk; (full time wheel chair use)\n2. Body mass index (BMI) less than 13.5 kg/m² or greater than 40 kg/m²\n3. New or change in medications/therapies within 4 weeks prior to Screening\n4. Use of strong CYP3A4 inhibitors (e.g clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir) within 4 weeks prior to Baseline\n5. Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window (e.g. warfarin and digitoxin)\n6. Current enrollment in a clinical trial of an investigational drug or enrollment in a clinical trial of an investigational drug in the last 6 months\n7. Existing or historical medical conditions or complications (e.g. neurological, cardiovascular, renal, hepatic, endocrine, gastrointestinal or respiratory disease) which would cause the investigator to conclude that the subject will not be able to perform the study procedures or assessments or would confound interpretation of data obtained during assessment\n8. Hypersensitivity to tideglusib and its excipients including allergy to strawberry"}, 'identificationModule': {'nctId': 'NCT03692312', 'briefTitle': 'Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy', 'organization': {'class': 'INDUSTRY', 'fullName': 'AMO Pharma Limited'}, 'officialTitle': 'A Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents With Congenital Myotonic Dystrophy (REACH CDM)', 'orgStudyIdInfo': {'id': 'AMO-02-MD-2-003'}, 'secondaryIdInfos': [{'id': '2016-004623-23', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Tideglusib', 'description': 'Weight adjusted tideglusib, orally, once daily', 'interventionNames': ['Drug: Tideglusib', 'Drug: Placebo']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Matching placebo, orally, once daily', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'Tideglusib', 'type': 'DRUG', 'description': 'Tideglusib for oral suspension, weight-adjusted at 400mg, 600mg or 1000 mg dose levels, once daily', 'armGroupLabels': ['Tideglusib']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Matching placebo formulation', 'armGroupLabels': ['Placebo', 'Tideglusib']}]}, 'contactsLocationsModule': {'locations': [{'zip': '72202', 'city': 'Little Rock', 'state': 'Arkansas', 'country': 'United States', 'facility': "Arkansas Children's Hospital", 'geoPoint': {'lat': 34.74648, 'lon': -92.28959}}, {'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'University of California, Los Angeles (UCLA)', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '94304', 'city': 'Palo Alto', 'state': 'California', 'country': 'United States', 'facility': 'Stanford University', 'geoPoint': {'lat': 37.44188, 'lon': -122.14302}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': "Ann & Robert H. Lurie Children's Hospital of Chicago", 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '52242', 'city': 'Iowa City', 'state': 'Iowa', 'country': 'United States', 'facility': 'University of Iowa Hospitals and Clinics', 'geoPoint': {'lat': 41.66113, 'lon': -91.53017}}, {'zip': '14642', 'city': 'Rochester', 'state': 'New York', 'country': 'United States', 'facility': 'University of Rochester Medical Center', 'geoPoint': {'lat': 43.15478, 'lon': -77.61556}}, {'zip': '15213', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'University of Pittsburgh Medical Center', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '84112', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': 'University of Utah Hospital', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '23219', 'city': 'Richmond', 'state': 'Virginia', 'country': 'United States', 'facility': 'Virginia Commonwealth University - Department of Neurology. Muscular Dystrophy Translational Research Program.', 'geoPoint': {'lat': 37.55376, 'lon': -77.46026}}, {'zip': '2031', 'city': 'Randwick', 'state': 'New South Wales', 'country': 'Australia', 'facility': 'The Bright Alliance', 'geoPoint': {'lat': -33.91439, 'lon': 151.24895}}, {'zip': 'N6A4G5', 'city': 'London', 'state': 'Ontario', 'country': 'Canada', 'facility': "Children's Hospital London Health Sciences Centre (LHSC)", 'geoPoint': {'lat': 42.98339, 'lon': -81.23304}}, {'zip': 'K1H 8L1', 'city': 'Ottawa', 'state': 'Ontario', 'country': 'Canada', 'facility': "Children's Hospital of Eastern Ontario", 'geoPoint': {'lat': 45.41117, 'lon': -75.69812}}, {'zip': '1010', 'city': 'Auckland', 'country': 'New Zealand', 'facility': 'New Zealand Clinical Research (NZCR)', 'geoPoint': {'lat': -36.84853, 'lon': 174.76349}}, {'zip': 'NE2 4HH', 'city': 'Newcastle upon Tyne', 'country': 'United Kingdom', 'facility': 'Newcastle University', 'geoPoint': {'lat': 54.97328, 'lon': -1.61396}}], 'overallOfficials': [{'name': 'Joseph P Horrigan, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'AMO Pharma'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'AMO Pharma Limited', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}