Ignite Creation Date:
2025-12-24 @ 10:06 PM
Ignite Modification Date:
2026-03-15 @ 1:00 PM
Study NCT ID:
NCT03238235
Status:
COMPLETED
Last Update Posted:
2024-11-18
First Post:
2017-07-12
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular Dystrophy
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D020388', 'term': 'Muscular Dystrophy, Duchenne'}], 'ancestors': [{'id': 'D009136', 'term': 'Muscular Dystrophies'}, {'id': 'D020966', 'term': 'Muscular Disorders, Atrophic'}, {'id': 'D009135', 'term': 'Muscular Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C575255', 'term': 'givinostat'}, {'id': 'C502418', 'term': 'givinostat hydrochloride'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'm.caserini@italfarmacogroup.com', 'phone': '+ 39 02 64431', 'title': 'Maurizio Caserini, MD', 'organization': 'Italfarmaco SpA'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}}, 'adverseEventsModule': {'timeFrame': 'Throughout the study, from screening (visits 1 and 2) to EOS/early withdrawal (V11, at week 48 ± 7 days) till follow-up visit (visit 12, at week 52 ± 7 days). This means the AEs were monitored from screening to day 364 ± 7.', 'description': 'Patients who completed the study (12 months of treatment) were asked to return to the center for the Follow-up visit 4 weeks after the last dose of study treatment (4 weeks ±1). However, a patient that was discontinued from the study treatment was asked to return for the early withdrawal visit within 4 weeks of the last dose of study treatment.', 'eventGroups': [{'id': 'EG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\ngivinostat: suspension of givinostat (10 mg/mL) bid', 'otherNumAtRisk': 34, 'deathsNumAtRisk': 34, 'otherNumAffected': 30, 'seriousNumAtRisk': 34, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nplacebo: suspension manufactured to mimic givinostat', 'otherNumAtRisk': 17, 'deathsNumAtRisk': 17, 'otherNumAffected': 9, 'seriousNumAtRisk': 17, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Lymphocytosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Sinus tachycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 16, 'numAffected': 16}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Dyspepsia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Gastrointestinal disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Chest discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Hyperpyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Influenza like illness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Oedema peripheral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'COVID-19', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Cystitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Gastroenteritis viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Tonsillitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Chest injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Face injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Head injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Joint injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Ligament sprain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Rib fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Tooth injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Aspartate aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Blood bilirubin increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Blood glucose increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Blood phosphorus increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Blood potassium increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Blood sodium increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Blood triglycerides increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'C-reactive protein increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Gamma-glutamyltransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Heart rate increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'N-terminal prohormone brain natriuretic peptide increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Platelet count decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 20, 'numAffected': 17}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Weight increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'White blood cell count decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Hypertriglyceridaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 10, 'numAffected': 10}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Intervertebral disc protrusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Joint swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Muscle hypertrophy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Muscle spasms', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Muscular weakness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Myalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Neck pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Periarthritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Balance disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Dizziness postural', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Hand-arm vibration syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Paraesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Insomnia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Pneumonitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Restrictive pulmonary disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Sleep apnoea syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Dermal cyst', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Rash erythematous', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Tooth repair', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Surgical and medical procedures', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Umbilical hernia repair', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Surgical and medical procedures', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Epistaxis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Haematuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 34, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Mean Change From Baseline to Visit 11 in Total Fibrosis (%) on Log Scale, Comparing the Histology of Muscle Biopsies', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}, {'value': '15', 'groupId': 'OG001'}]}, {'units': '# fields for each biopsy sample', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nplacebo: suspension manufactured to mimic givinostat'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.017', 'groupId': 'OG000', 'lowerLimit': '-0.367', 'upperLimit': '0.333'}, {'value': '-0.054', 'groupId': 'OG001', 'lowerLimit': '-0.507', 'upperLimit': '0.399'}]}]}], 'analyses': [{'pValue': '0.8282', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Log Difference of the least square means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.04', 'ciLowerLimit': '-0.30', 'ciUpperLimit': '0.38', 'pValueComment': 'ANCOVA model was performed considering the difference between log of total fibrosis and log baseline values as dependent variable; log baseline value was included as covariate, treatment and concomitant steroid use as independent class variables.', 'groupDescription': 'total fibrosis at visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.6465', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Log Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.11', 'ciLowerLimit': '-0.59', 'ciUpperLimit': '0.37', 'groupDescription': 'A Mixed Model was fitted to the data in order to evaluate the difference in total fibrosis between biopsy sites', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY', 'nonInferiorityComment': 'Mixed model was performed considering the difference between log Visit 11 and log baseline values as dependent variable; log baseline as covariate, treatment was included as independent class variable and treatment by sequence interaction. The sequence of biopsy site (R-L, L-R) was included as a fixed effect and subject as a random effect. If the treatment by sequence interaction was p\\>0.10, then the model without the interaction term is presented.'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'after 12 months of treatment (at Visit 11)', 'description': 'The primary efficacy assessment was the mean total fibrosis (%) on log scale assessed through histological examination of bicep muscle biopsies at two timepoints (At baseline and at Visit 11). More particularly, patients underwent two biopsies of muscle from the brachial biceps: the first before starting the study treatment (Visit 2, baseline), and the second at the end of treatment (Visit 11). For each patient, the percentage of total fibrosis was calculated as log of the least square mean of the available fields at each evaluation.', 'unitOfMeasure': 'log[percentage of total fibrosis]', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': '# fields for each biopsy sample', 'denomUnitsSelected': '# fields for each biopsy sample', 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set.'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline to Visit 11 in the Percentage of Fat Fraction of Vastus Lateralis and Soleus', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': 'muscle images for timepoint', 'counts': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic Givinostat'}], 'classes': [{'title': 'Vastus Lateralis', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': 'muscle images for timepoint', 'counts': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0.017', 'groupId': 'OG000', 'lowerLimit': '-0.053', 'upperLimit': '0.086'}, {'value': '0.064', 'groupId': 'OG001', 'lowerLimit': '-0.019', 'upperLimit': '0.147'}]}]}, {'title': 'Soleus', 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}, {'value': '14', 'groupId': 'OG001'}]}, {'units': 'muscle images for timepoint', 'counts': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-4.287', 'groupId': 'OG000', 'lowerLimit': '-6.126', 'upperLimit': '-2.447'}, {'value': '-4.021', 'groupId': 'OG001', 'lowerLimit': '-6.404', 'upperLimit': '-1.637'}]}]}], 'analyses': [{'pValue': '0.1991', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Log Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.05', 'ciLowerLimit': '-0.12', 'ciUpperLimit': '0.03', 'pValueComment': 'ANCOVA model was performed considering baseline fat fraction of vastus lateralis or fat fraction in the soleus value as covariate and treatment and concomitant steroid use at baseline as independent class variables.', 'groupDescription': 'Vastus lateralis', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.7849', 'groupIds': ['OG000', 'OG001'], 'paramType': 'difference of the least square means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.27', 'ciLowerLimit': '-2.22', 'ciUpperLimit': '1.69', 'pValueComment': 'ANCOVA model was performed considering baseline fat fraction of vastus lateralis or fat fraction in the soleus value as covariate and treatment and concomitant steroid use at baseline as independent class variables.', 'groupDescription': 'Soleus', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'after 12 months of treatment (at Visit 11)', 'description': 'Evaluation was performed comparing Magnetic Resonance Spectroscopy (MRS) before and after 12 months of treatment with Givinostat versus placebo. Please note that Statistics data are expressed as log least square mean (IC 95%) for the vastus lateralis and as least square mean (IC 95%) back-transformed on the original scale for the soleus.', 'unitOfMeasure': 'log[percentage of fat in muscle]', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'muscle images for timepoint', 'denomUnitsSelected': 'muscle images for timepoint', 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set.'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline to Visit 11 in the Percentage of Fat Fraction of Lower Limb Muscles', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': 'MRI images for each timepoint', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic Givinostat'}], 'classes': [{'title': 'Whole Thigh', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': 'MRI images for each timepoint', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0.642', 'groupId': 'OG000', 'lowerLimit': '-0.328', 'upperLimit': '1.611'}, {'value': '1.996', 'groupId': 'OG001', 'lowerLimit': '0.813', 'upperLimit': '3.180'}]}]}, {'title': 'Quadriceps', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': 'MRI images for each timepoint', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0.610', 'groupId': 'OG000', 'lowerLimit': '-0.506', 'upperLimit': '1.726'}, {'value': '2.572', 'groupId': 'OG001', 'lowerLimit': '1.227', 'upperLimit': '3.918'}]}]}, {'title': 'Hamstrings', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': 'MRI images for each timepoint', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0.877', 'groupId': 'OG000', 'lowerLimit': '-0.627', 'upperLimit': '2.381'}, {'value': '1.455', 'groupId': 'OG001', 'lowerLimit': '-0.400', 'upperLimit': '3.309'}]}]}, {'title': 'Triceps Surae', 'denoms': [{'units': 'Participants', 'counts': [{'value': '22', 'groupId': 'OG000'}, {'value': '11', 'groupId': 'OG001'}]}, {'units': 'MRI images for each timepoint', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-0.276', 'groupId': 'OG000', 'lowerLimit': '-2.862', 'upperLimit': '2.310'}, {'value': '1.317', 'groupId': 'OG001', 'lowerLimit': '-1.686', 'upperLimit': '4.319'}]}]}, {'title': 'Pelvis Girdle', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': 'MRI images for each timepoint', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0.584', 'groupId': 'OG000', 'lowerLimit': '-0.509', 'upperLimit': '1.676'}, {'value': '1.471', 'groupId': 'OG001', 'lowerLimit': '0.112', 'upperLimit': '2.830'}]}]}], 'analyses': [{'pValue': '0.0149', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-1.35', 'ciLowerLimit': '-2.43', 'ciUpperLimit': '-0.28', 'pValueComment': 'ANCOVA model was performed considering baseline Fat Fraction of lower limb muscles value as covariate and treatment and concomitant steroid use at baseline as independent class variables.', 'groupDescription': 'Whole thigh at visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.0022', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-1.96', 'ciLowerLimit': '-3.18', 'ciUpperLimit': '-0.75', 'pValueComment': 'ANCOVA model was performed considering baseline Fat Fraction of lower limb muscles value as covariate and treatment and concomitant steroid use at baseline as independent class variables', 'groupDescription': 'Quadriceps at visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.4869', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of least square means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.58', 'ciLowerLimit': '-2.24', 'ciUpperLimit': '1.08', 'pValueComment': 'ANCOVA model was performed considering baseline Fat Fraction of lower limb muscles value as covariate and treatment and concomitant steroid use at baseline as independent class variables.', 'groupDescription': 'Hamstrings at visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.0939', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-1.59', 'ciLowerLimit': '-3.47', 'ciUpperLimit': '0.29', 'pValueComment': 'ANCOVA model was performed considering baseline Fat Fraction of lower limb muscles value as covariate and treatment and concomitant steroid use at baseline as independent class variables.', 'groupDescription': 'Triceps Surae at Visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.1579', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.89', 'ciLowerLimit': '-2.13', 'ciUpperLimit': '0.36', 'pValueComment': 'ANCOVA model was performed considering baseline Fat Fraction of lower limb muscles value as covariate and treatment and concomitant steroid use at baseline as independent class variables.', 'groupDescription': 'Pelvis girdle at visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'after 12 months of treatment (at Visit 11)', 'description': 'Evaluation was performed comparing Dixon Magnetic Resonance Imaging (MRI) before and after 12 months of treatment with Givinostat versus placebo.\n\nThe lower limb muscles assessed were: whole thigh, quadriceps, medial thigh, hamstrings, triceps surae and pelvic girdle. Please note that statistics data are expressed as: Least Square Means (95% CI) for whole thigh, quadriceps, hamstrings, triceps surae and pelvis girdle; while as Log Least Square Means (95% CI) for medial thigh.', 'unitOfMeasure': 'percentage of fat in muscle', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'MRI images for each timepoint', 'denomUnitsSelected': 'MRI images for each timepoint', 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set.'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline to Visit 11 in Cross-sectional Area (CSA), in cm2 of Lower Limb Muscles', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': 'MRI images for each timepoint', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic Givinostat'}], 'classes': [{'title': 'whole thigh', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': 'MRI images for each timepoint', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '2.782', 'groupId': 'OG000', 'lowerLimit': '0.151', 'upperLimit': '5.413'}, {'value': '2.377', 'groupId': 'OG001', 'lowerLimit': '-0.820', 'upperLimit': '5.575'}]}]}, {'title': 'quadriceps', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': 'MRI images for each timepoint', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0.997', 'groupId': 'OG000', 'lowerLimit': '-0.169', 'upperLimit': '2.163'}, {'value': '1.082', 'groupId': 'OG001', 'lowerLimit': '-0.329', 'upperLimit': '2.494'}]}]}, {'title': 'medial thigh', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': 'MRI images for each timepoint', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0.669', 'groupId': 'OG000', 'lowerLimit': '-0.468', 'upperLimit': '1.807'}, {'value': '0.321', 'groupId': 'OG001', 'lowerLimit': '-1.060', 'upperLimit': '1.702'}]}]}, {'title': 'hamstrings', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': 'MRI images for each timepoint', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '1.103', 'groupId': 'OG000', 'lowerLimit': '0.108', 'upperLimit': '2.098'}, {'value': '0.994', 'groupId': 'OG001', 'lowerLimit': '-0.207', 'upperLimit': '2.195'}]}]}, {'title': 'triceps surae', 'denoms': [{'units': 'Participants', 'counts': [{'value': '22', 'groupId': 'OG000'}, {'value': '11', 'groupId': 'OG001'}]}, {'units': 'MRI images for each timepoint', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-0.033', 'groupId': 'OG000', 'lowerLimit': '-3.699', 'upperLimit': '3.634'}, {'value': '0.183', 'groupId': 'OG001', 'lowerLimit': '-3.756', 'upperLimit': '4.122'}]}]}, {'title': 'pelvic girdle', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': 'MRI images for each timepoint', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '1.284', 'groupId': 'OG000', 'lowerLimit': '-0.494', 'upperLimit': '3.062'}, {'value': '0.965', 'groupId': 'OG001', 'lowerLimit': '-1.187', 'upperLimit': '3.116'}]}]}], 'analyses': [{'pValue': '0.7770', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.40', 'ciLowerLimit': '-2.45', 'ciUpperLimit': '3.26', 'pValueComment': 'ANCOVA model was performed considering baseline CSA as covariate, treatment and concomitant steroid use at baseline as independent class variables.', 'groupDescription': 'Whole Thigh at visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.8926', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.09', 'ciLowerLimit': '-1.35', 'ciUpperLimit': '1.18', 'pValueComment': 'ANCOVA model was performed considering baseline CSA as covariate, treatment and concomitant steroid use at baseline as independent class variables', 'groupDescription': 'Quadriceps at Visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.5720', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.35', 'ciLowerLimit': '-0.88', 'ciUpperLimit': '1.58', 'pValueComment': 'ANCOVA model was performed considering baseline CSA as covariate, treatment and concomitant steroid use at baseline as independent class variables.', 'groupDescription': 'Medial Thigh at Visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.8386', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.11', 'ciLowerLimit': '-0.97', 'ciUpperLimit': '1.18', 'pValueComment': 'ANCOVA model was performed considering baseline CSA as covariate, treatment and concomitant steroid use at baseline as independent class variables.', 'groupDescription': 'Hamstrings at Visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.8591', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.22', 'ciLowerLimit': '-2.68', 'ciUpperLimit': '2.25', 'pValueComment': 'ANCOVA model was performed considering baseline CSA as covariate, treatment and concomitant steroid use at baseline as independent class variables', 'groupDescription': 'Triceps Surae at Visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.7392', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.32', 'ciLowerLimit': '-1.60', 'ciUpperLimit': '2.24', 'groupDescription': 'Pelvis Girdle at Visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'Evaluation was performed comparing Dixon MRI findings before and after 12 months of treatment with Givinostat versus placebo.\n\nDixon technique has advantages in multiple applications for evaluation of musculoskeletal system diseases. It allows more robust fat suppression than do other sequences and can be used in combination with multiple different sequences (GRE and SE) and using different weightings (T1, T2, or proton density).\n\nThe lower limb muscles assessed were: whole thigh, quadriceps, medial thigh, hamstrings, triceps surae and pelvic girdle (data showed as least square mean)', 'unitOfMeasure': 'centimeters^2', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'MRI images for each timepoint', 'denomUnitsSelected': 'MRI images for each timepoint', 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set.'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline to Visit 11 in Contractile Area of Lower Limb Muscles (MRI)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': 'MRI muscle images for each timepoint', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic Givinostat'}], 'classes': [{'title': 'Contractile Area in the Whole Thigh', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': 'MRI muscle images for each timepoint', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0.309', 'groupId': 'OG000', 'lowerLimit': '-0.853', 'upperLimit': '1.470'}, {'value': '-1.057', 'groupId': 'OG001', 'lowerLimit': '-2.466', 'upperLimit': '0.352'}]}]}, {'title': 'Contractile Area in the Quadriceps', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': 'MRI muscle images for each timepoint', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0.139', 'groupId': 'OG000', 'lowerLimit': '-0.458', 'upperLimit': '0.735'}, {'value': '-0.493', 'groupId': 'OG001', 'lowerLimit': '-1.195', 'upperLimit': '0.210'}]}]}, {'title': 'Contractile Area in the Medial Thigh', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': 'MRI muscle images for each timepoint', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0.162', 'groupId': 'OG000', 'lowerLimit': '-0.342', 'upperLimit': '0.665'}, {'value': '-0.197', 'groupId': 'OG001', 'lowerLimit': '-0.813', 'upperLimit': '0.420'}]}]}, {'title': 'Contractile Area in the Triceps Surae', 'denoms': [{'units': 'Participants', 'counts': [{'value': '22', 'groupId': 'OG000'}, {'value': '11', 'groupId': 'OG001'}]}, {'units': 'MRI muscle images for each timepoint', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-0.542', 'groupId': 'OG000', 'lowerLimit': '-3.598', 'upperLimit': '2.514'}, {'value': '-1.291', 'groupId': 'OG001', 'lowerLimit': '-4.632', 'upperLimit': '2.050'}]}]}, {'title': 'Contractile Area in the Pelvis Girdle', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': 'MRI muscle images for each timepoint', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0.257', 'groupId': 'OG000', 'lowerLimit': '-0.420', 'upperLimit': '0.933'}, {'value': '-0.287', 'groupId': 'OG001', 'lowerLimit': '-1.133', 'upperLimit': '0.559'}]}]}], 'analyses': [{'pValue': '0.0375', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '1.37', 'ciLowerLimit': '0.08', 'ciUpperLimit': '2.65', 'pValueComment': 'ANCOVA model was performed considering baseline Contractile Area value as covariate, treatment and concomitant steroid use at baseline as independent class variables.', 'groupDescription': 'Whole Thigh at Visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.0528', 'groupIds': ['OG000', 'OG001'], 'paramType': 'difference of least square means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.63', 'ciLowerLimit': '-0.01', 'ciUpperLimit': '1.27', 'pValueComment': 'ANCOVA model was performed considering baseline Contractile Area value as covariate, treatment and concomitant steroid use at baseline as independent class variables.', 'groupDescription': 'Quadriceps at Visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.2012', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.36', 'ciLowerLimit': '-0.20', 'ciUpperLimit': '0.91', 'pValueComment': 'ANCOVA model was performed considering baseline Contractile Area value as covariate, treatment and concomitant steroid use at baseline as independent class variables', 'groupDescription': 'Medial Thigh at Visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.4676', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.75', 'ciLowerLimit': '-1.33', 'ciUpperLimit': '2.83', 'pValueComment': 'ANCOVA model was performed considering baseline Contractile Area value as covariate, treatment and concomitant steroid use at baseline as independent class variables.', 'groupDescription': 'Triceps Surae at Visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.1549', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.54', 'ciLowerLimit': '-0.21', 'ciUpperLimit': '1.30', 'pValueComment': 'ANCOVA model was performed considering baseline Contractile Area value as covariate, treatment and concomitant steroid use at baseline as independent class variables.', 'groupDescription': 'Pelvis Girdle at Visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'after 12 months of treatment', 'description': 'A summary of mean change in the contractile area of lower limb muscles comparing MRI findings before and after 12 months of treatment in the ITT set is reported. The lower limb muscles considered are the same as the outcome 3. Please note that statistic data are expressed as Least Square Means (95% CI) except for Hamstrings which is expressed as Log Least Square Means.', 'unitOfMeasure': 'centimeters^2', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'MRI muscle images for each timepoint', 'denomUnitsSelected': 'MRI muscle images for each timepoint', 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set.'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline to Visit 11 in Biopsy Histology Parameters: CSA by Type (I or II Fibers), Total CSA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': '# fields for each biopsy sample', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic Givinostat'}], 'classes': [{'title': 'CSA type II fibers', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': '# fields for each biopsy sample', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-25.320', 'groupId': 'OG000', 'lowerLimit': '-1438.395', 'upperLimit': '1387.755'}, {'value': '593.875', 'groupId': 'OG001', 'lowerLimit': '-1177.854', 'upperLimit': '2365.604'}]}]}, {'title': 'Total CSA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': '# fields for each biopsy sample', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '642.505', 'groupId': 'OG000', 'lowerLimit': '-603.081', 'upperLimit': '1888.092'}, {'value': '1215.047', 'groupId': 'OG001', 'lowerLimit': '-359.764', 'upperLimit': '2789.857'}]}]}], 'analyses': [{'pValue': '0.3240', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-619.20', 'ciLowerLimit': '-1872.37', 'ciUpperLimit': '633.98', 'pValueComment': 'ANCOVA model was performed considering baseline biopsy histological parameters (Slide III) value as covariate, treatment and concomitant steroid use at baseline as independent class variables', 'groupDescription': 'CSA Type II at Visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.3070', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-572.54', 'ciLowerLimit': '-1690.73', 'ciUpperLimit': '545.64', 'pValueComment': 'ANCOVA model was performed considering baseline biopsy histological parameters (Slide III) value as covariate, treatment and concomitant steroid use at baseline as independent class variables', 'groupDescription': 'Total CSA at Visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'After 12 months of treatment (Visit 11)', 'description': 'A summary of the mean change in cross-sectional area (CSA) by type after 12 months of treatment in the ITT set is reported. Please note that descriptive statistic data are expressed as Log Least Square Means (95% CI) for CSA type I fibers, and as Least Square Means (95% CI) for CSA type II fibers and Total CSA.', 'unitOfMeasure': 'micrometers^2', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': '# fields for each biopsy sample', 'denomUnitsSelected': '# fields for each biopsy sample', 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set.'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline to Visit 11 in Percentage for the Following Histology Parameters: Fibers With Nuclear Centralizations (%), Total Number of Fibers (%), Regenerative Fibers (%).', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': '# fields for each biopsy sample', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic Pivinostat'}], 'classes': [{'title': 'Fibers with nuclear centralizations', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}, {'value': '15', 'groupId': 'OG001'}]}, {'units': '# fields for each biopsy sample', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0.196', 'groupId': 'OG000', 'lowerLimit': '-0.183', 'upperLimit': '0.576'}, {'value': '0.463', 'groupId': 'OG001', 'lowerLimit': '0.006', 'upperLimit': '0.921'}]}]}, {'title': 'Total number of fibers (Slide II)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}, {'value': '15', 'groupId': 'OG001'}]}, {'units': '# fields for each biopsy sample', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-24.573', 'groupId': 'OG000', 'lowerLimit': '-59.132', 'upperLimit': '9.986'}, {'value': '-22.346', 'groupId': 'OG001', 'lowerLimit': '-64.799', 'upperLimit': '20.106'}]}]}, {'title': 'Total number of fibers (Slide III)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}, {'value': '15', 'groupId': 'OG001'}]}, {'units': '# fields for each biopsy sample', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-9.804', 'groupId': 'OG000', 'lowerLimit': '-22.511', 'upperLimit': '2.904'}, {'value': '-14.521', 'groupId': 'OG001', 'lowerLimit': '-30.335', 'upperLimit': '1.293'}]}]}], 'analyses': [{'pValue': '0.1055', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Log Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.27', 'ciLowerLimit': '-0.59', 'ciUpperLimit': '0.06', 'pValueComment': 'ANCOVA model was performed considering baseline Biopsy histological parameters (Slide I) value as covariate, treatment and concomitant steroid use at baseline as independent class variables.', 'groupDescription': 'Fibers with nuclear centralizations (%) at Visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.8846', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-2.23', 'ciLowerLimit': '-33.05', 'ciUpperLimit': '28.59', 'pValueComment': 'ANCOVA model was performed considering baseline biopsy histological parameters (Slide II) value as covariate, treatment and concomitant steroid use at baseline as independent class variables.', 'groupDescription': 'Total number of fibers at Visit 11 (slide II)', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.4054', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '4.72', 'ciLowerLimit': '-6.62', 'ciUpperLimit': '16.06', 'pValueComment': 'ANCOVA model was performed considering baseline biopsy histological parameters (Slide III) value as covariate, treatment and concomitant steroid use at baseline as independent class variables', 'groupDescription': 'Total number of fibers (Slide III)', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'Evaluation of histologic parameters such as Fiber with nuclear centralizations (%), Total number of fibers (%), and Regenerative fibers (%) were performed comparing muscle biopsies after 12 months of treatment with Givinostat. Please note that descriptive statistic data are expressed as log least square mean for Regenerative fibers (%), and total number of fibers (Slides I), while are expressed as least square mean for Fibers with nuclear centralizations (%) and total number of fibers (Slides II and III).', 'unitOfMeasure': 'percentage of fibers', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': '# fields for each biopsy sample', 'denomUnitsSelected': '# fields for each biopsy sample', 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned.'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline to Visit 11 in Percentage for the Biopsy Histology Parameter MFA(%)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}, {'value': '15', 'groupId': 'OG001'}]}, {'units': '# fields for each biopsy sample', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic Pivinostat'}], 'classes': [{'categories': [{'measurements': [{'value': '0.422', 'groupId': 'OG000', 'lowerLimit': '-10.200', 'upperLimit': '11.043'}, {'value': '-2.028', 'groupId': 'OG001', 'lowerLimit': '-16.254', 'upperLimit': '12.199'}]}]}], 'analyses': [{'pValue': '0.6340', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '2.45', 'ciLowerLimit': '-7.87', 'ciUpperLimit': '12.77', 'pValueComment': 'ANCOVA model was performed considering baseline Biopsy histological parameters (Slide I) value as covariate, treatment and concomitant steroid use at baseline as independent class variables', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'Evaluation of histology parameters such as Muscle Fibers Area \\[MFA\\](%), were performed comparing muscle biopsies after 12 months of treatment with Givinostat. MFA fraction was determined from biopsies using the mean of available fields. Please note that descriptive statistic data are expressed as least square mean.', 'unitOfMeasure': 'percentage of muscle fiber area', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': '# fields for each biopsy sample', 'denomUnitsSelected': '# fields for each biopsy sample', 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set.'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline to Visit 11 in Percentage for the Biopsy Histology Parameters Adipose Tissue (%)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}, {'value': '15', 'groupId': 'OG001'}]}, {'units': '# fields for each biopsy sample', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic Pivinostat'}], 'classes': [{'categories': [{'measurements': [{'value': '0.088', 'groupId': 'OG000', 'lowerLimit': '-0.364', 'upperLimit': '0.540'}, {'value': '0.226', 'groupId': 'OG001', 'lowerLimit': '-0.346', 'upperLimit': '0.798'}]}]}], 'analyses': [{'pValue': '0.4893', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Log Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.14', 'ciLowerLimit': '-0.54', 'ciUpperLimit': '0.26', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'Evaluation of histology parameters such as Adipose tissue (%) were performed comparing muscle biopsies after 12 months of treatment with Givinostat. Please note that descriptive statistic data are expressed as log least square mean.', 'unitOfMeasure': 'percentage of total tissue', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': '# fields for each biopsy sample', 'denomUnitsSelected': '# fields for each biopsy sample', 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set.'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline to Visit 11 in Other Histological Structures', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}, {'value': '15', 'groupId': 'OG001'}]}, {'units': '# fields for each biopsy sample', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic Pivinostat'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.297', 'groupId': 'OG000', 'lowerLimit': '-0.832', 'upperLimit': '0.238'}, {'value': '0.045', 'groupId': 'OG001', 'lowerLimit': '-0.611', 'upperLimit': '0.701'}]}]}], 'analyses': [{'pValue': '0.1498', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Log Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.34', 'ciLowerLimit': '-0.81', 'ciUpperLimit': '0.13', 'pValueComment': 'This model was performed considering baseline biopsy histological parameters (Slide I) value as covariate, treatment and concomitant steroid use at baseline as independent class variables.', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'Evaluation of other histological structures like necrotic cells, vessels and any other nonconnective tissue present in the microscopic field were performed comparing muscle biopsies after 12 months of treatment with Givinostat. The value is shown as % compared to the arithmetic mean of the two positive control (i.e., healthy subjects) bands present in the same western blot. Please note that descriptive statistic data are expressed as log least square mean.', 'unitOfMeasure': 'percentage of total tissue', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': '# fields for each biopsy sample', 'denomUnitsSelected': '# fields for each biopsy sample', 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set.'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline to Visit 11 in Motor Function Measurement (MFM, Expressed as Log Least Square Mean)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic Givinostat'}], 'classes': [{'title': 'Standing and transfers (D1)', 'categories': [{'measurements': [{'value': '-0.038', 'groupId': 'OG000', 'lowerLimit': '-0.087', 'upperLimit': '0.011'}, {'value': '-0.100', 'groupId': 'OG001', 'lowerLimit': '-0.165', 'upperLimit': '-0.035'}]}]}, {'title': 'Axial and proximal motor function (D2)', 'categories': [{'measurements': [{'value': '-0.004', 'groupId': 'OG000', 'lowerLimit': '-0.012', 'upperLimit': '0.004'}, {'value': '-0.007', 'groupId': 'OG001', 'lowerLimit': '-0.018', 'upperLimit': '0.003'}]}]}, {'title': 'Distal motor function (D3)', 'categories': [{'measurements': [{'value': '-0.003', 'groupId': 'OG000', 'lowerLimit': '-0.013', 'upperLimit': '0.006'}, {'value': '-0.001', 'groupId': 'OG001', 'lowerLimit': '-0.014', 'upperLimit': '0.011'}]}]}, {'title': 'Total Score', 'categories': [{'measurements': [{'value': '-0.011', 'groupId': 'OG000', 'lowerLimit': '-0.025', 'upperLimit': '0.002'}, {'value': '-0.026', 'groupId': 'OG001', 'lowerLimit': '-0.044', 'upperLimit': '-0.008'}]}]}], 'analyses': [{'pValue': '0.0602', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Log Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.06', 'ciLowerLimit': '-0.00', 'ciUpperLimit': '0.13', 'pValueComment': 'Least squares mean is obtained from the mixed effects model for repeated measures with treatment, visit, visit by treatment interaction and concomitant steroid use at baseline as fixed effect; baseline value is included as a covariate.', 'groupDescription': 'Standing and transfers (D1) at Visit 11', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.5906', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Log Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.00', 'ciLowerLimit': '-0.01', 'ciUpperLimit': '0.01', 'pValueComment': 'Least squares mean is obtained from the mixed effects model for repeated measures with treatment, visit, visit by treatment interaction and concomitant steroid use at baseline as fixed effect; baseline value is included as a covariate.', 'groupDescription': 'Axial and proximal motor function (D2) at Visit 11', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.7799', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Log Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.00', 'ciLowerLimit': '-0.01', 'ciUpperLimit': '0.01', 'pValueComment': 'Least squares mean is obtained from the mixed effects model for repeated measures with treatment, visit, visit by treatment interaction and concomitant steroid use at baseline as fixed effect; baseline value is included as a covariate.', 'groupDescription': 'Distal motor function (D3) at Visit 11', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.1116', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Log Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.01', 'ciLowerLimit': '-0.00', 'ciUpperLimit': '0.03', 'pValueComment': 'Least squares mean is obtained from the mixed effects model for repeated measures with treatment, visit, visit by treatment interaction and concomitant steroid use at baseline as fixed effect; baseline value is included as a covariate.', 'groupDescription': 'Total Score at Visit 11', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': "Evaluation were performed using the MFM32 scale, that is a tool designed for neuromuscular diseases and is applicable to all degrees of disease severity. It was validated in terms of reproducibility, construct validity, and concurrent validity. It consists of 32 items (tasks) classified into three dimensions: D1, standing and transfers; D2, axial and proximal motor capacity; and D3, distal motor capacity. Each item is scored on a four-point Likert scale. The generic grading is measured as follows: 0, cannot initiate the task or cannot maintain the starting position; 1, partially performs the task; 2, performs the task with compensatory movements (position maintained for an insufficient period of time, slowness, uncontrolled movements, etc.); and 3, performs the task fully and 'normally', the movement being controlled, mastered, directed, and performed at a constant speed. The overall total score ranging from 0 (severe functional impairment) to 100 (no functional impairment).", 'unitOfMeasure': 'score on a scale', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set.'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline to Visit 11 in Time Function Test (TFT): Time to Walk/Run 10 Meters', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic Givinostat'}], 'classes': [{'categories': [{'measurements': [{'value': '0.013', 'groupId': 'OG000', 'lowerLimit': '-0.100', 'upperLimit': '0.125'}, {'value': '0.078', 'groupId': 'OG001', 'lowerLimit': '-0.072', 'upperLimit': '0.228'}]}]}], 'analyses': [{'pValue': '0.4346', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Log Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.07', 'ciLowerLimit': '-0.23', 'ciUpperLimit': '0.10', 'pValueComment': 'Least squares mean is obtained from the mixed effects model for repeated measures with treatment, visit, visit by treatment interaction and concomitant steroid use at baseline as fixed effect; baseline value is included as a covariate', 'groupDescription': 'Time to Walk/Run 10 meters at Visit 11', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': "TFT is assessed through 3 different parameters one of which is time to walk/run 10 m. The test was performed with or without orthoses as the patient preferred. He/she was not asked to run, but rather to arrive at the finish line as soon as possible leaving him the choice on how to do so. The assessor walked alongside the patient for safety but couldn't help her/him in any way. The walk/run speed was calculated as 10/time in seconds taken to run/walk 10 m. The test was graded as follows:\n\n1. Unable to walk independently.\n2. Unable to walk independently but can walk with full leg calipers (KAFOs) or with support of a person.\n3. Highly adapted wide-based lordotic gait. Can't increase walking speed.\n4. Moderately adapted gait. Can pick up speed but can't run.\n5. Able to pick up speed but runs with a double stance phase, i.e., cannot achieve both feet off the ground.\n6. Runs and gets both feet off the ground (with no double stance phase). The higher the grade, the better the outcome.", 'unitOfMeasure': 'sec', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set.'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline to Visit 11 in Time Function Test (TFT) Via Time to Climb 4 Standard Steps', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}, {'value': '15', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic Givinostat'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.144', 'groupId': 'OG000', 'lowerLimit': '-0.339', 'upperLimit': '0.051'}, {'value': '-0.123', 'groupId': 'OG001', 'lowerLimit': '-0.396', 'upperLimit': '0.149'}]}]}], 'analyses': [{'pValue': '0.8914', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Log Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.02', 'ciLowerLimit': '-0.32', 'ciUpperLimit': '0.28', 'groupDescription': 'Time to climb 4 standard steps (sec) at visit 11', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'TFT is assessed through 3 parameters, one of which is "time to climb 4 standard steps".\n\nThe patient was asked to stand at the bottom of the stairs with his arms by his sides. At the "go" signal, he/she had to walk up the stairs as quickly and safely as possible (using handrails if needed) until reaching an erect position on the top stair. The stair climb speed was calculated as 4/time in seconds taken to climb the 4 standard stairs.\n\nGrading of the 4-step ascent was:1. Unable to climb 4 stairs.2. Climbs 4 stairs "marking time" (climbs one foot at a time with both feet on a step before moving to next step), using both arms on one or both handrails.3. Climbs 4 stairs "marking time" (idem as up), using one arm on one handrail.4. Climbs 4 stairs "marking time" (idem as up), not needing handrail.5. Climbs 4 stairs alternating feet, needs handrail for support.6. Climbs 4 stairs alternating feet, not needing handrail support.The higher the grade, the better the outcome.', 'unitOfMeasure': 'sec', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set. Please consider the data are showed in Log Least Square Mean'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline to Visit 11 in Time Function Test Via Time to Rise From Floor', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}, {'value': '10', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic Givinostat'}], 'classes': [{'categories': [{'measurements': [{'value': '1.392', 'groupId': 'OG000', 'lowerLimit': '-0.968', 'upperLimit': '3.753'}, {'value': '0.774', 'groupId': 'OG001', 'lowerLimit': '-3.014', 'upperLimit': '4.562'}]}]}], 'analyses': [{'pValue': '0.7629', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.62', 'ciLowerLimit': '-3.51', 'ciUpperLimit': '4.75', 'pValueComment': 'Least squares mean is obtained from the mixed effects model for repeated measures with treatment, visit, visit by treatment interaction and concomitant steroid use at baseline as fixed effect; baseline value is included as a covariate.', 'groupDescription': 'Time to rise from floor at Visit 11', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'Time function test (TFT) is assessed through 3 different parameters, one of which was "time to rise from the floor".\n\nThe patient started the test lying on his back with arms at his sides and was asked to get up as quickly as possible. The rise from floor velocity was calculated as 1/time in seconds taken to stand up. Grading was as follows:\n\n1. Unable to stand, even with use of a chair.\n2. Assisted Gowers\' sign - requires furniture to assist in rising to full upright posture.\n3. Full Gowers\' sign - rolls over, stands up with both hands "climbing up" the legs to achieve full upright posture.\n4. Half Gowers\' sign - rolls over, stands up with one hand support on leg.\n5. Rolls to the side and/or stands up with one hand or both hands on the floor to start to rise up but does not touch legs.\n6. Stands up without rolling over or using hands. The higher the grade, the better the outcome.', 'unitOfMeasure': 'sec', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline to Visit 11 in Distance Performed Via 6-minute Walk/Run Test (6MWT, Expressed as Log Least Square Mean)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic Givinostat'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.058', 'groupId': 'OG000', 'lowerLimit': '-0.118', 'upperLimit': '0.002'}, {'value': '-0.046', 'groupId': 'OG001', 'lowerLimit': '-0.128', 'upperLimit': '0.036'}]}]}], 'analyses': [{'pValue': '0.8106', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Log Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.01', 'ciLowerLimit': '-0.11', 'ciUpperLimit': '0.08', 'pValueComment': 'Least squares mean is obtained from the mixed effects model for repeated measures with treatment, visit, visit by treatment interaction and concomitant steroid use at baseline as fixed effect; baseline value is included as a covariate', 'groupDescription': 'Maximum distance walked after 6 minutes at Visit 11', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'The 6-minute walk/run test (6MWT) assessed the distance walked in 6 minutes. The 6MWT was performed indoors on a flat, smooth path, at least 30 m long and 3 m wide, with a cone at each end around which the patient had to walk. Six progressively numbered markers were used to mark the distance travelled at each minute. Five progressively lettered markers were used to indicate any falls.\n\nTwo staff members were present during the test; the physiotherapist, to give the patient instructions, and a staff member who followed the patient giving encouragements. The patient was instructed to go from cone to cone as fast as he could but without running, and that he could stop to rest whenever he/she wanted. The time walked, distance walked after each minute, time of each fall and distance walked before each fall were registered.\n\nA summary of the 6-minute maximum distance walked/run after 12 months of treatment is reported in minutes.', 'unitOfMeasure': 'log [meters]', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set.'}, {'type': 'SECONDARY', 'title': 'Percentage of Patients With < 10% Worsening in 6MWT After 12 Months of Treatment.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic Givinostat'}], 'classes': [{'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.1626', 'groupIds': ['OG000', 'OG001'], 'pValueComment': 'P-value is obtained from the two-sided Cochran-Mantel-Haenszel chi-squared test, stratified for concomitant steroid use at baseline', 'groupDescription': '\\<10% worsening at visit 11', 'statisticalMethod': 'Cochran-Mantel-Haenszel', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'Percentage of patients with \\< 10% worsening in 6-Minute Walking Test after 12 months of treatment with Givinostat comparing to placebo patients.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Patients with missing data such that \\<10% worsening cannot be determined are included in the analysis as having worsened'}, {'type': 'SECONDARY', 'title': 'Count of Participant Who Lose Ambulation During the Study (From Baseline to Month 12)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic Givinostat'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'from baseline to the end of month 12', 'description': 'Percentage of patients losing the ability to ambulate from baseline to the end of study (Visit 11 or Month 12)', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set.'}, {'type': 'SECONDARY', 'title': 'Percentage of Patients Who Fell During the 6MWT', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic Givinostat'}], 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'A summary of the number of patients who fell and the number of falls occurring during the 6MWT is shown.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set.'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline to Visit 11 in Muscle Strength Evaluated by Knee Extension, Elbow Flexion', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': '# of measurements', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic Givinostat'}], 'classes': [{'title': 'Left Knee Extension (N)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}, {'value': '14', 'groupId': 'OG001'}]}, {'units': '# of measurements', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-1.479', 'groupId': 'OG000', 'lowerLimit': '-10.208', 'upperLimit': '7.251'}, {'value': '-5.051', 'groupId': 'OG001', 'lowerLimit': '-15.952', 'upperLimit': '5.851'}]}]}, {'title': 'Right Knee Extension (N)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}]}, {'units': '# of measurements', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0.703', 'groupId': 'OG000', 'lowerLimit': '-5.212', 'upperLimit': '6.618'}, {'value': '-0.460', 'groupId': 'OG001', 'lowerLimit': '-7.705', 'upperLimit': '6.784'}]}]}, {'title': 'Left Elbow Flexion (N)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}, {'value': '14', 'groupId': 'OG001'}]}, {'units': '# of measurements', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '1.551', 'groupId': 'OG000', 'lowerLimit': '-10.046', 'upperLimit': '13.148'}, {'value': '-2.373', 'groupId': 'OG001', 'lowerLimit': '-17.487', 'upperLimit': '12.742'}]}]}, {'title': 'Right Elbow Flexion (N)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}, {'value': '15', 'groupId': 'OG001'}]}, {'units': '# of measurements', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-1.277', 'groupId': 'OG000', 'lowerLimit': '-13.384', 'upperLimit': '10.830'}, {'value': '-5.381', 'groupId': 'OG001', 'lowerLimit': '-21.270', 'upperLimit': '10.508'}]}]}], 'analyses': [{'pValue': '0.5099', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '3.57', 'ciLowerLimit': '-7.27', 'ciUpperLimit': '14.41', 'pValueComment': 'Least squares mean is obtained from the mixed effects model for repeated measures with treatment, visit, visit by treatment interaction and concomitant steroid use at baseline as fixed effect; baseline value is included as a covariate.', 'groupDescription': 'Left Knee Extension (N) at Visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.7355', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '1.16', 'ciLowerLimit': '-5.73', 'ciUpperLimit': '8.06', 'pValueComment': 'Least squares mean is obtained from the mixed effects model for repeated measures with treatment, visit, visit by treatment interaction and concomitant steroid use at baseline as fixed effect; baseline value is included as a covariate.', 'groupDescription': 'Right Knee Extension (N) at Visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.6037', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '3.92', 'ciLowerLimit': '-11.22', 'ciUpperLimit': '19.06', 'pValueComment': 'Least squares mean is obtained from the mixed effects model for repeated measures with treatment, visit, visit by treatment interaction and concomitant steroid use at baseline as fixed effect; baseline value is included as a covariate.', 'groupDescription': 'Left Elbow Flexion (N) at Visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.6178', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '4.10', 'ciLowerLimit': '-12.35', 'ciUpperLimit': '20.55', 'pValueComment': 'Least squares mean is obtained from the mixed effects model for repeated measures with treatment, visit, visit by treatment interaction and concomitant steroid use at baseline as fixed effect; baseline value is included as a covariate.', 'groupDescription': 'Right Elbow Flexion (N) at Visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'Evaluation was performed by mean left and right knee extension, mean left and right elbow flexion using the Hand Held Myometry (HHM). Using it, the muscle strength of the knee extensor and elbow flexor were measured via standardized procedures; 3 measurements were recorded from each muscle group on each side. The mean of the 3 measurements was calculated.\n\nKnee Extension: the pt was sitting, pelvis and knee at a 90° angle. His/her feet were above the ground with the femur in neutral rotation. He/she could hold on to the edge of the bed/chair and, in case, additional stabilization was provided at the distal third of the thigh, just above the knee. The HHM was placed on the anterior surface of the lower third of the tibia, proximal to the ankle joint.\n\nElbow flexion: pt lying on his back, arm by his side, elbow flexed at a 90° angle, forearm in supine position. The MMH was placed between the middle and distal third of the forearm, proximal to the radial styloid process.', 'unitOfMeasure': 'Newton', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': '# of measurements', 'denomUnitsSelected': '# of measurements', 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set.'}, {'type': 'SECONDARY', 'title': 'Mean Changes From Baseline to Visit 11 in Quality of Life (QoL, Assessed by the 36-item Short Form Survey [SF36])', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic Givinostat'}], 'classes': [{'title': 'Physical Functioning', 'categories': [{'measurements': [{'value': '-1.03', 'spread': '16.274', 'groupId': 'OG000'}, {'value': '-5.00', 'spread': '11.989', 'groupId': 'OG001'}]}]}, {'title': 'Role-Physical', 'categories': [{'measurements': [{'value': '-2.21', 'spread': '19.697', 'groupId': 'OG000'}, {'value': '0.37', 'spread': '16.006', 'groupId': 'OG001'}]}]}, {'title': 'Bodily Pain', 'categories': [{'measurements': [{'value': '5.12', 'spread': '19.384', 'groupId': 'OG000'}, {'value': '2.12', 'spread': '15.227', 'groupId': 'OG001'}]}]}, {'title': 'General Health', 'categories': [{'measurements': [{'value': '0.82', 'spread': '14.532', 'groupId': 'OG000'}, {'value': '1.47', 'spread': '13.267', 'groupId': 'OG001'}]}]}, {'title': 'Vitality', 'categories': [{'measurements': [{'value': '0.55', 'spread': '11.654', 'groupId': 'OG000'}, {'value': '3.68', 'spread': '12.705', 'groupId': 'OG001'}]}]}, {'title': 'Social Functioning', 'categories': [{'measurements': [{'value': '2.94', 'spread': '22.415', 'groupId': 'OG000'}, {'value': '8.82', 'spread': '20.139', 'groupId': 'OG001'}]}]}, {'title': 'Role-Emotional', 'categories': [{'measurements': [{'value': '0.00', 'spread': '20.205', 'groupId': 'OG000'}, {'value': '4.90', 'spread': '15.607', 'groupId': 'OG001'}]}]}, {'title': 'Mental Health', 'categories': [{'measurements': [{'value': '4.12', 'spread': '15.997', 'groupId': 'OG000'}, {'value': '4.41', 'spread': '13.793', 'groupId': 'OG001'}]}]}, {'title': 'Physical Component Summary', 'categories': [{'measurements': [{'value': '-0.17', 'spread': '5.633', 'groupId': 'OG000'}, {'value': '-1.20', 'spread': '5.535', 'groupId': 'OG001'}]}]}, {'title': 'Mental Component Summary', 'categories': [{'measurements': [{'value': '1.41', 'spread': '7.345', 'groupId': 'OG000'}, {'value': '3.70', 'spread': '7.177', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'A summary of QoL (SF-36, Single items Short Form survey) in the ITT set is shown.\n\nQoL (SF-36) is assessed considering:\n\n* Physical Functioning (PF)\n* Role-Physical (RP)\n* Bodily Pain (BP)\n* General Health (GH)\n* Vitality (VT)\n* Social Functioning (SF)\n* Role-Emotional (RE)\n* Mental Health (MH)\n* Physical Component Summary (PCS)\n* Mental Component Summary (MCS) The SF-36 consists of eight scaled scores, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. The lower the score, the greater the disability. The SF-36 is a set of generic, coherent, and easily administered quality-of-life measures. These measures rely upon patient self-reporting and are widely utilized by managed care organizations for routine monitoring and assessment of care outcomes in adult patients.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set.'}, {'type': 'SECONDARY', 'title': 'Number of Patients Experiencing Any Kind of Severity of TEAEs, Serious and Non Serious) From Baseline Through End of Study (EOS).', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic Givinostat'}], 'classes': [{'title': 'Number of patients with any TEAEs', 'categories': [{'measurements': [{'value': '30', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}]}]}, {'title': 'Number of patients with serious TEAEs', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Number of patients with TEAEs leading to interruption of study treatment', 'categories': [{'measurements': [{'value': '11', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Number of patients with fatal TEAEs', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Number of patients with mild TEAEs', 'categories': [{'measurements': [{'value': '30', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}]}]}, {'title': 'Number of patients with moderate TEAEs', 'categories': [{'measurements': [{'value': '12', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Number of patients with severe TEAEs', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Number of patients with TEAEs related to study treatment', 'categories': [{'measurements': [{'value': '29', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}]}, {'title': 'Number of patients with TEAEs not related to study treatment', 'categories': [{'measurements': [{'value': '25', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Throughout the study, till week 52 +/-7 days', 'description': 'Treatment-emergent adverse events (TEAEs) are defined as those events with an onset date after study treatment initiation. An AE is an untoward medical occurrence after exposure to a medicine, which is not necessarily caused by that medicine. A serious AE is an adverse reaction that results in death, is life-threatening, requires hospitalisation or prolongation of existing hospitalisation, results in persistent or significant disability or incapacity, or is a birth defect.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Set: all patients who received at least one dose of study medication. Patients were analyzed according to the study treatment actually received. The safety set was used for the safety analysis.'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline to Visit 11 in the Percentage of Fat Fraction of Lower Limb Muscles (Log)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': 'MRI images for each timepoint', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic Givinostat'}], 'classes': [{'categories': [{'measurements': [{'value': '0.012', 'groupId': 'OG000', 'lowerLimit': '-0.017', 'upperLimit': '0.041'}, {'value': '0.038', 'groupId': 'OG001', 'lowerLimit': '0.003', 'upperLimit': '0.073'}]}]}], 'analyses': [{'pValue': '0.1165', 'groupIds': ['OG000', 'OG001'], 'paramType': 'log difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.03', 'ciLowerLimit': '-0.06', 'ciUpperLimit': '0.01', 'pValueComment': 'ANCOVA model was performed considering baseline fat fraction of lower limb muscles value as covariate and treatment and concomitant steroids use at baseline as independent class variables', 'groupDescription': 'Medial Thigh at visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'after 12 months of treatment (at Visit 11)', 'description': 'Evaluation was performed comparing Dixon Magnetic Resonance Imaging (MRI) before and after 12 months of treatment with Givinostat versus placebo.\n\nThe lower limb muscles assessed were: whole thigh, quadriceps, medial thigh, hamstrings, triceps surae and pelvic girdle. Please note that statistics data are expressed as: Least Square Means (95% CI) for whole thigh, quadriceps, hamstrings, triceps surae and pelvis girdle; while as Log Least Square Means (95% CI) for medial thigh.', 'unitOfMeasure': 'log[percentage of fat in muscle]', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'MRI images for each timepoint', 'denomUnitsSelected': 'MRI images for each timepoint', 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set.'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline to Visit 11 in Contractile Area of Lower Limb Muscles (MRI) (Log)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': 'MRI muscle images for each timepoint', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic Givinostat'}], 'classes': [{'categories': [{'measurements': [{'value': '0.025', 'groupId': 'OG000', 'lowerLimit': '-0.042', 'upperLimit': '0.092'}, {'value': '-0.022', 'groupId': 'OG001', 'lowerLimit': '-0.104', 'upperLimit': '0.059'}]}]}], 'analyses': [{'pValue': '0.1939', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Log difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.05', 'ciLowerLimit': '-0.02', 'ciUpperLimit': '0.12', 'pValueComment': 'ANCOVA model was performed considering baseline CSA as covariate and treatment and concomitant steroids use at baseline as independent class variables', 'groupDescription': 'Hamstrings at Visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'after 12 months of treatment', 'description': 'A summary of mean change in the contractile area of lower limb muscles comparing MRI findings before and after 12 months of treatment in the ITT set is reported. The lower limb muscles considered are the same as the outcome 3. Please note that statistic data are expressed as Least Square Means (95% CI) except for Hamstrings which is expressed as Log Least Square Means.', 'unitOfMeasure': 'centimeters^2', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'MRI muscle images for each timepoint', 'denomUnitsSelected': 'MRI muscle images for each timepoint', 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set.'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline to Visit 11 in Biopsy Histology Parameters: CSA by Type (I or II Fibers), Total CSA (Log)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': '# fields for each biopsy sample', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic Givinostat'}], 'classes': [{'categories': [{'measurements': [{'value': '0.255', 'groupId': 'OG000', 'lowerLimit': '-0.069', 'upperLimit': '0.579'}, {'value': '0.246', 'groupId': 'OG001', 'lowerLimit': '-0.166', 'upperLimit': '0.658'}]}]}], 'analyses': [{'pValue': '0.9493', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Log difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.01', 'ciLowerLimit': '-0.29', 'ciUpperLimit': '0.30', 'pValueComment': 'ANCOVA model was performed considering baseline biopsy histological parameters (slide III) value as covariate, treatment and concomitant steroids use at baseline as independent class variables', 'groupDescription': 'CSA Type I', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'After 12 months of treatment (Visit 11)', 'description': 'A summary of the mean change in cross-sectional area (CSA) by type after 12 months of treatment in the ITT set is reported. Please note that descriptive statistic data are expressed as Log Least Square Means (95% CI) for CSA type I fibers, and as Least Square Means (95% CI) for CSA type II fibers and Total CSA.', 'unitOfMeasure': 'log [micrometers^2]', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': '# fields for each biopsy sample', 'denomUnitsSelected': '# fields for each biopsy sample', 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set.'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline to Visit 11 in Percentage for the Following Histology Parameters: Fibers With Nuclear Centralizations (%), Total Number of Fibers (%), Regenerative Fibers (%). (Log)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}, {'units': '# fields for each biopsy sample', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic Pivinostat'}], 'classes': [{'title': 'Total number of fibers (Slide I)', 'categories': [{'measurements': [{'value': '-0.207', 'groupId': 'OG000', 'lowerLimit': '-0.446', 'upperLimit': '0.032'}, {'value': '-0.257', 'groupId': 'OG001', 'lowerLimit': '-0.552', 'upperLimit': '0.037'}]}]}, {'title': 'Regenerative fibers', 'categories': [{'measurements': [{'value': '0.667', 'groupId': 'OG000', 'lowerLimit': '-0.031', 'upperLimit': '1.365'}, {'value': '1.104', 'groupId': 'OG001', 'lowerLimit': '0.248', 'upperLimit': '1.961'}]}]}], 'analyses': [{'pValue': '0.6265', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Log difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.05', 'ciLowerLimit': '-0.16', 'ciUpperLimit': '0.26', 'pValueComment': 'ANCOVA model was performed considering baseline biopsy histological parameters (slide I) value as covariate, treatment and concomitant steroids use at baseline as independent class variables', 'groupDescription': 'Total number of Fibers at Visit 11 (slide I)', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.1562', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Log difference of Least Square Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.44', 'ciLowerLimit': '-1.05', 'ciUpperLimit': '0.17', 'pValueComment': 'ANCOVA model was performed considering baseline biopsy histological parameters (slide II) value as covariate, treatment and concomitant steroids use at baseline as independent class variables', 'groupDescription': 'Regenerative Fibers (%) at Visit 11', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'Evaluation of histologic parameters such as Fiber with nuclear centralizations (%), Total number of fibers (%), and Regenerative fibers (%) were performed comparing muscle biopsies after 12 months of treatment with Givinostat. Please note that descriptive statistic data are expressed as log least square mean for Regenerative fibers (%), and total number of fibers (Slides I), while are expressed as least square mean for Fibers with nuclear centralizations (%) and total number of fibers (Slides II and III).', 'unitOfMeasure': 'log[percentage of fibers]', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': '# fields for each biopsy sample', 'denomUnitsSelected': '# fields for each biopsy sample', 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\nGivinostat: suspension of Givinostat (10 mg/mL)'}, {'id': 'FG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nPlacebo: suspension manufactured to mimic givinostat'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '34'}, {'groupId': 'FG001', 'numSubjects': '17'}]}, {'type': 'Per Protocol', 'achievements': [{'groupId': 'FG000', 'numSubjects': '25'}, {'groupId': 'FG001', 'numSubjects': '14'}]}, {'type': 'ITT', 'achievements': [{'groupId': 'FG000', 'numSubjects': '34'}, {'groupId': 'FG001', 'numSubjects': '17'}]}, {'type': 'Safety', 'achievements': [{'groupId': 'FG000', 'numSubjects': '34'}, {'groupId': 'FG001', 'numSubjects': '17'}]}, {'type': 'PK', 'achievements': [{'groupId': 'FG000', 'numSubjects': '34'}, {'groupId': 'FG001', 'numSubjects': '17'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '30'}, {'groupId': 'FG001', 'numSubjects': '17'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '4'}, {'groupId': 'FG001', 'numSubjects': '0'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'patients unable to travel to the site due to the COVID-19 pandemic', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'Seventy patients provided written informed consent to participate in this study. Fifty-one patients (72.86%) completed screening successfully and were randomized; 34 patients (66.67%) were assigned to the Givinostat group and 17 (33.33%) to the placebo group.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'BG000'}, {'value': '17', 'groupId': 'BG001'}, {'value': '51', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state\n\ngivinostat: suspension of givinostat (10 mg/mL)'}, {'id': 'BG001', 'title': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state\n\nplacebo: suspension manufactured to mimic givinostat'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '34', 'groupId': 'BG000'}, {'value': '17', 'groupId': 'BG001'}, {'value': '51', 'groupId': 'BG002'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '36.5', 'spread': '11.56', 'groupId': 'BG000'}, {'value': '39.2', 'spread': '9.84', 'groupId': 'BG001'}, {'value': '37.4', 'spread': '10.99', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '34', 'groupId': 'BG000'}, {'value': '17', 'groupId': 'BG001'}, {'value': '51', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Black or African American', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '17', 'groupId': 'BG001'}, {'value': '18', 'groupId': 'BG002'}]}, {'title': 'White', 'measurements': [{'value': '33', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '33', 'groupId': 'BG002'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'Netherlands', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}]}]}, {'title': 'Italy', 'categories': [{'measurements': [{'value': '31', 'groupId': 'BG000'}, {'value': '14', 'groupId': 'BG001'}, {'value': '45', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}], 'populationDescription': 'Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2017-06-20', 'size': 11299442, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2023-05-24T04:09', 'hasProtocol': True}, {'date': '2021-04-27', 'size': 33134975, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2023-05-24T04:10', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR'], 'maskingDescription': "Placebo was indistinguishable from the active product in color, appearance, smell and taste. Personnel involved in the study (Investigators, nurses, all other site personnel, clinical research associates \\[CRA\\], medical monitors, project managers, data managers and statisticians) were blinded at all times unless knowledge of the study treatment was necessary for the patient's safety."}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 51}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2018-01-09', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-03', 'completionDateStruct': {'date': '2021-03-19', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-09-13', 'studyFirstSubmitDate': '2017-07-12', 'resultsFirstSubmitDate': '2023-05-24', 'studyFirstSubmitQcDate': '2017-07-31', 'lastUpdatePostDateStruct': {'date': '2024-11-18', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2024-09-13', 'studyFirstPostDateStruct': {'date': '2017-08-03', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2024-11-18', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-03-19', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Mean Change From Baseline to Visit 11 in Total Fibrosis (%) on Log Scale, Comparing the Histology of Muscle Biopsies', 'timeFrame': 'after 12 months of treatment (at Visit 11)', 'description': 'The primary efficacy assessment was the mean total fibrosis (%) on log scale assessed through histological examination of bicep muscle biopsies at two timepoints (At baseline and at Visit 11). More particularly, patients underwent two biopsies of muscle from the brachial biceps: the first before starting the study treatment (Visit 2, baseline), and the second at the end of treatment (Visit 11). For each patient, the percentage of total fibrosis was calculated as log of the least square mean of the available fields at each evaluation.'}], 'secondaryOutcomes': [{'measure': 'Mean Change From Baseline to Visit 11 in the Percentage of Fat Fraction of Vastus Lateralis and Soleus', 'timeFrame': 'after 12 months of treatment (at Visit 11)', 'description': 'Evaluation was performed comparing Magnetic Resonance Spectroscopy (MRS) before and after 12 months of treatment with Givinostat versus placebo. Please note that Statistics data are expressed as log least square mean (IC 95%) for the vastus lateralis and as least square mean (IC 95%) back-transformed on the original scale for the soleus.'}, {'measure': 'Mean Change From Baseline to Visit 11 in the Percentage of Fat Fraction of Lower Limb Muscles', 'timeFrame': 'after 12 months of treatment (at Visit 11)', 'description': 'Evaluation was performed comparing Dixon Magnetic Resonance Imaging (MRI) before and after 12 months of treatment with Givinostat versus placebo.\n\nThe lower limb muscles assessed were: whole thigh, quadriceps, medial thigh, hamstrings, triceps surae and pelvic girdle. Please note that statistics data are expressed as: Least Square Means (95% CI) for whole thigh, quadriceps, hamstrings, triceps surae and pelvis girdle; while as Log Least Square Means (95% CI) for medial thigh.'}, {'measure': 'Mean Change From Baseline to Visit 11 in Cross-sectional Area (CSA), in cm2 of Lower Limb Muscles', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'Evaluation was performed comparing Dixon MRI findings before and after 12 months of treatment with Givinostat versus placebo.\n\nDixon technique has advantages in multiple applications for evaluation of musculoskeletal system diseases. It allows more robust fat suppression than do other sequences and can be used in combination with multiple different sequences (GRE and SE) and using different weightings (T1, T2, or proton density).\n\nThe lower limb muscles assessed were: whole thigh, quadriceps, medial thigh, hamstrings, triceps surae and pelvic girdle (data showed as least square mean)'}, {'measure': 'Mean Change From Baseline to Visit 11 in Contractile Area of Lower Limb Muscles (MRI)', 'timeFrame': 'after 12 months of treatment', 'description': 'A summary of mean change in the contractile area of lower limb muscles comparing MRI findings before and after 12 months of treatment in the ITT set is reported. The lower limb muscles considered are the same as the outcome 3. Please note that statistic data are expressed as Least Square Means (95% CI) except for Hamstrings which is expressed as Log Least Square Means.'}, {'measure': 'Mean Change From Baseline to Visit 11 in Biopsy Histology Parameters: CSA by Type (I or II Fibers), Total CSA', 'timeFrame': 'After 12 months of treatment (Visit 11)', 'description': 'A summary of the mean change in cross-sectional area (CSA) by type after 12 months of treatment in the ITT set is reported. Please note that descriptive statistic data are expressed as Log Least Square Means (95% CI) for CSA type I fibers, and as Least Square Means (95% CI) for CSA type II fibers and Total CSA.'}, {'measure': 'Mean Change From Baseline to Visit 11 in Percentage for the Following Histology Parameters: Fibers With Nuclear Centralizations (%), Total Number of Fibers (%), Regenerative Fibers (%).', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'Evaluation of histologic parameters such as Fiber with nuclear centralizations (%), Total number of fibers (%), and Regenerative fibers (%) were performed comparing muscle biopsies after 12 months of treatment with Givinostat. Please note that descriptive statistic data are expressed as log least square mean for Regenerative fibers (%), and total number of fibers (Slides I), while are expressed as least square mean for Fibers with nuclear centralizations (%) and total number of fibers (Slides II and III).'}, {'measure': 'Mean Change From Baseline to Visit 11 in Percentage for the Biopsy Histology Parameter MFA(%)', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'Evaluation of histology parameters such as Muscle Fibers Area \\[MFA\\](%), were performed comparing muscle biopsies after 12 months of treatment with Givinostat. MFA fraction was determined from biopsies using the mean of available fields. Please note that descriptive statistic data are expressed as least square mean.'}, {'measure': 'Mean Change From Baseline to Visit 11 in Percentage for the Biopsy Histology Parameters Adipose Tissue (%)', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'Evaluation of histology parameters such as Adipose tissue (%) were performed comparing muscle biopsies after 12 months of treatment with Givinostat. Please note that descriptive statistic data are expressed as log least square mean.'}, {'measure': 'Mean Change From Baseline to Visit 11 in Other Histological Structures', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'Evaluation of other histological structures like necrotic cells, vessels and any other nonconnective tissue present in the microscopic field were performed comparing muscle biopsies after 12 months of treatment with Givinostat. The value is shown as % compared to the arithmetic mean of the two positive control (i.e., healthy subjects) bands present in the same western blot. Please note that descriptive statistic data are expressed as log least square mean.'}, {'measure': 'Mean Change From Baseline to Visit 11 in Motor Function Measurement (MFM, Expressed as Log Least Square Mean)', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': "Evaluation were performed using the MFM32 scale, that is a tool designed for neuromuscular diseases and is applicable to all degrees of disease severity. It was validated in terms of reproducibility, construct validity, and concurrent validity. It consists of 32 items (tasks) classified into three dimensions: D1, standing and transfers; D2, axial and proximal motor capacity; and D3, distal motor capacity. Each item is scored on a four-point Likert scale. The generic grading is measured as follows: 0, cannot initiate the task or cannot maintain the starting position; 1, partially performs the task; 2, performs the task with compensatory movements (position maintained for an insufficient period of time, slowness, uncontrolled movements, etc.); and 3, performs the task fully and 'normally', the movement being controlled, mastered, directed, and performed at a constant speed. The overall total score ranging from 0 (severe functional impairment) to 100 (no functional impairment)."}, {'measure': 'Mean Change From Baseline to Visit 11 in Time Function Test (TFT): Time to Walk/Run 10 Meters', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': "TFT is assessed through 3 different parameters one of which is time to walk/run 10 m. The test was performed with or without orthoses as the patient preferred. He/she was not asked to run, but rather to arrive at the finish line as soon as possible leaving him the choice on how to do so. The assessor walked alongside the patient for safety but couldn't help her/him in any way. The walk/run speed was calculated as 10/time in seconds taken to run/walk 10 m. The test was graded as follows:\n\n1. Unable to walk independently.\n2. Unable to walk independently but can walk with full leg calipers (KAFOs) or with support of a person.\n3. Highly adapted wide-based lordotic gait. Can't increase walking speed.\n4. Moderately adapted gait. Can pick up speed but can't run.\n5. Able to pick up speed but runs with a double stance phase, i.e., cannot achieve both feet off the ground.\n6. Runs and gets both feet off the ground (with no double stance phase). The higher the grade, the better the outcome."}, {'measure': 'Mean Change From Baseline to Visit 11 in Time Function Test (TFT) Via Time to Climb 4 Standard Steps', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'TFT is assessed through 3 parameters, one of which is "time to climb 4 standard steps".\n\nThe patient was asked to stand at the bottom of the stairs with his arms by his sides. At the "go" signal, he/she had to walk up the stairs as quickly and safely as possible (using handrails if needed) until reaching an erect position on the top stair. The stair climb speed was calculated as 4/time in seconds taken to climb the 4 standard stairs.\n\nGrading of the 4-step ascent was:1. Unable to climb 4 stairs.2. Climbs 4 stairs "marking time" (climbs one foot at a time with both feet on a step before moving to next step), using both arms on one or both handrails.3. Climbs 4 stairs "marking time" (idem as up), using one arm on one handrail.4. Climbs 4 stairs "marking time" (idem as up), not needing handrail.5. Climbs 4 stairs alternating feet, needs handrail for support.6. Climbs 4 stairs alternating feet, not needing handrail support.The higher the grade, the better the outcome.'}, {'measure': 'Mean Change From Baseline to Visit 11 in Time Function Test Via Time to Rise From Floor', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'Time function test (TFT) is assessed through 3 different parameters, one of which was "time to rise from the floor".\n\nThe patient started the test lying on his back with arms at his sides and was asked to get up as quickly as possible. The rise from floor velocity was calculated as 1/time in seconds taken to stand up. Grading was as follows:\n\n1. Unable to stand, even with use of a chair.\n2. Assisted Gowers\' sign - requires furniture to assist in rising to full upright posture.\n3. Full Gowers\' sign - rolls over, stands up with both hands "climbing up" the legs to achieve full upright posture.\n4. Half Gowers\' sign - rolls over, stands up with one hand support on leg.\n5. Rolls to the side and/or stands up with one hand or both hands on the floor to start to rise up but does not touch legs.\n6. Stands up without rolling over or using hands. The higher the grade, the better the outcome.'}, {'measure': 'Change From Baseline to Visit 11 in Distance Performed Via 6-minute Walk/Run Test (6MWT, Expressed as Log Least Square Mean)', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'The 6-minute walk/run test (6MWT) assessed the distance walked in 6 minutes. The 6MWT was performed indoors on a flat, smooth path, at least 30 m long and 3 m wide, with a cone at each end around which the patient had to walk. Six progressively numbered markers were used to mark the distance travelled at each minute. Five progressively lettered markers were used to indicate any falls.\n\nTwo staff members were present during the test; the physiotherapist, to give the patient instructions, and a staff member who followed the patient giving encouragements. The patient was instructed to go from cone to cone as fast as he could but without running, and that he could stop to rest whenever he/she wanted. The time walked, distance walked after each minute, time of each fall and distance walked before each fall were registered.\n\nA summary of the 6-minute maximum distance walked/run after 12 months of treatment is reported in minutes.'}, {'measure': 'Percentage of Patients With < 10% Worsening in 6MWT After 12 Months of Treatment.', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'Percentage of patients with \\< 10% worsening in 6-Minute Walking Test after 12 months of treatment with Givinostat comparing to placebo patients.'}, {'measure': 'Count of Participant Who Lose Ambulation During the Study (From Baseline to Month 12)', 'timeFrame': 'from baseline to the end of month 12', 'description': 'Percentage of patients losing the ability to ambulate from baseline to the end of study (Visit 11 or Month 12)'}, {'measure': 'Percentage of Patients Who Fell During the 6MWT', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'A summary of the number of patients who fell and the number of falls occurring during the 6MWT is shown.'}, {'measure': 'Mean Change From Baseline to Visit 11 in Muscle Strength Evaluated by Knee Extension, Elbow Flexion', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'Evaluation was performed by mean left and right knee extension, mean left and right elbow flexion using the Hand Held Myometry (HHM). Using it, the muscle strength of the knee extensor and elbow flexor were measured via standardized procedures; 3 measurements were recorded from each muscle group on each side. The mean of the 3 measurements was calculated.\n\nKnee Extension: the pt was sitting, pelvis and knee at a 90° angle. His/her feet were above the ground with the femur in neutral rotation. He/she could hold on to the edge of the bed/chair and, in case, additional stabilization was provided at the distal third of the thigh, just above the knee. The HHM was placed on the anterior surface of the lower third of the tibia, proximal to the ankle joint.\n\nElbow flexion: pt lying on his back, arm by his side, elbow flexed at a 90° angle, forearm in supine position. The MMH was placed between the middle and distal third of the forearm, proximal to the radial styloid process.'}, {'measure': 'Mean Changes From Baseline to Visit 11 in Quality of Life (QoL, Assessed by the 36-item Short Form Survey [SF36])', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'A summary of QoL (SF-36, Single items Short Form survey) in the ITT set is shown.\n\nQoL (SF-36) is assessed considering:\n\n* Physical Functioning (PF)\n* Role-Physical (RP)\n* Bodily Pain (BP)\n* General Health (GH)\n* Vitality (VT)\n* Social Functioning (SF)\n* Role-Emotional (RE)\n* Mental Health (MH)\n* Physical Component Summary (PCS)\n* Mental Component Summary (MCS) The SF-36 consists of eight scaled scores, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. The lower the score, the greater the disability. The SF-36 is a set of generic, coherent, and easily administered quality-of-life measures. These measures rely upon patient self-reporting and are widely utilized by managed care organizations for routine monitoring and assessment of care outcomes in adult patients.'}, {'measure': 'Number of Patients Experiencing Any Kind of Severity of TEAEs, Serious and Non Serious) From Baseline Through End of Study (EOS).', 'timeFrame': 'Throughout the study, till week 52 +/-7 days', 'description': 'Treatment-emergent adverse events (TEAEs) are defined as those events with an onset date after study treatment initiation. An AE is an untoward medical occurrence after exposure to a medicine, which is not necessarily caused by that medicine. A serious AE is an adverse reaction that results in death, is life-threatening, requires hospitalisation or prolongation of existing hospitalisation, results in persistent or significant disability or incapacity, or is a birth defect.'}, {'measure': 'Mean Change From Baseline to Visit 11 in the Percentage of Fat Fraction of Lower Limb Muscles (Log)', 'timeFrame': 'after 12 months of treatment (at Visit 11)', 'description': 'Evaluation was performed comparing Dixon Magnetic Resonance Imaging (MRI) before and after 12 months of treatment with Givinostat versus placebo.\n\nThe lower limb muscles assessed were: whole thigh, quadriceps, medial thigh, hamstrings, triceps surae and pelvic girdle. Please note that statistics data are expressed as: Least Square Means (95% CI) for whole thigh, quadriceps, hamstrings, triceps surae and pelvis girdle; while as Log Least Square Means (95% CI) for medial thigh.'}, {'measure': 'Mean Change From Baseline to Visit 11 in Contractile Area of Lower Limb Muscles (MRI) (Log)', 'timeFrame': 'after 12 months of treatment', 'description': 'A summary of mean change in the contractile area of lower limb muscles comparing MRI findings before and after 12 months of treatment in the ITT set is reported. The lower limb muscles considered are the same as the outcome 3. Please note that statistic data are expressed as Least Square Means (95% CI) except for Hamstrings which is expressed as Log Least Square Means.'}, {'measure': 'Mean Change From Baseline to Visit 11 in Biopsy Histology Parameters: CSA by Type (I or II Fibers), Total CSA (Log)', 'timeFrame': 'After 12 months of treatment (Visit 11)', 'description': 'A summary of the mean change in cross-sectional area (CSA) by type after 12 months of treatment in the ITT set is reported. Please note that descriptive statistic data are expressed as Log Least Square Means (95% CI) for CSA type I fibers, and as Least Square Means (95% CI) for CSA type II fibers and Total CSA.'}, {'measure': 'Mean Change From Baseline to Visit 11 in Percentage for the Following Histology Parameters: Fibers With Nuclear Centralizations (%), Total Number of Fibers (%), Regenerative Fibers (%). (Log)', 'timeFrame': 'after 12 months of treatment (Visit 11)', 'description': 'Evaluation of histologic parameters such as Fiber with nuclear centralizations (%), Total number of fibers (%), and Regenerative fibers (%) were performed comparing muscle biopsies after 12 months of treatment with Givinostat. Please note that descriptive statistic data are expressed as log least square mean for Regenerative fibers (%), and total number of fibers (Slides I), while are expressed as least square mean for Fibers with nuclear centralizations (%) and total number of fibers (Slides II and III).'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Givinostat', 'Becker Dystrophy'], 'conditions': ['Becker Muscular Dystrophy']}, 'descriptionModule': {'briefSummary': 'Objectives:\n\nPrimary objective: to establish the histological effects of Givinostat versus placebo administered over 12 months.\n\nSecondary Objectives:\n\n* To establish the macroscopic muscle effects of Givinostat versus placebo administered over 12 months assessed by Magnetic Resonance Imaging (MRI)/Magnetic Resonance Spectroscopy (MRS).\n* To determine the other histological effects of Givinostat versus placebo administered over 12 months.\n* To establish the efficacy of Givinostat versus placebo administered chronically over 12 months in slowing disease progression.\n* To assess the safety and tolerability of Givinostat versus placebo administered chronically.\n* To evaluate the pharmacokinetic (PK) profile of Givinostat administered chronically in the target population.\n* To evaluate the impact of Givinostat versus placebo administered chronically on quality of life and activities of daily living.', 'detailedDescription': 'This was a phase 2, randomised, double-blind, placebo-controlled study. Eligible patients were randomized in a 2:1 ratio to receive Givinostat or placebo for 12 months. Randomization was stratified by concomitant steroid use at baseline (yes or no). The study comprised twelve (12) visits: screening (V1, V2), randomization (V3), treatment (V4-V10), end of study (V11) and follow-up (V12). Visits during treatment took place every 12 weeks, except for the first 2 months, when they occurred every 2 weeks to allow closer monitoring of safety.\n\nGivinostat (ITF2357) oral suspension (10 mg/mL) was initially administered as 2 daily doses of 40-70 mg according to body weight after a meal (high dose). With amendment 2 of the protocol, a lower starting dose was implemented to address cases of thrombocytopenia reported following the treatment of the first 21 patients and corresponded to the reduced dose of the original protocol (i.e., 26.7-46.7 mg b.i.d according to body weight, i.e., low dose).\n\n51 patients were to be enrolled to provide a sample size of 48 patients with evaluable baseline biopsies. Seventy patients provided written informed consent, 51 (72.86%) completed screening successfully and were randomized; 34 patients (66.67%) to the Givinostat group and 17 (33.33%) to the placebo group.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n1. Ambulant patients with BMD diagnosis confirmed by genetic testing.\n2. Able and willing to give informed consent in writing.\n3. Able to perform 6MWT at screening with a minimum distance of 200 m and maximum distance of 450 m.\n4. If in treatment with systemic corticosteroids and/or angiotensin-converting-enzyme (ACE) inhibitor , and/or β or α adrenergic receptor blocker, no significant change in dosage or dosing regimen (excluding changes related to body weight) was to be presented for a minimum of 6 months prior to start of study treatment.\n5. Patients had to be willing to use adequate contraception from randomization until 3 months after the last dose of study treatment, and included the following:\n\n * True abstinence when in line with the preferred and usual lifestyle of the patient. Periodic abstinence (e.g., calendar ovulation, symptothermal, post ovulation methods) and withdrawal were not acceptable methods of contraception.\n * Condom with spermicide, with the female partner using an acceptable method of contraception, such as an oral, transdermal, injectable or implanted steroid-based contraceptive, or a diaphragm or a barrier method of contraception in conjunction with spermicidal jelly such as a cervical cap with spermicide jelly.\n\nExclusion Criteria:\n\n1. Exposure to another investigational drug within 3 months prior to the start of study treatment.\n2. Use of any pharmacological treatment, other than corticosteroids, that could have affected muscle strength or function within 3 months prior to the start of study treatment (e.g., growth hormone). vitamin D, calcium, and other supplements were allowed.\n3. Surgery that could have affected muscle strength or function within 3 months before study entry or planned surgery at any time during the study.\n4. Presence of other clinically significant disease that in the Investigator's opinion could have adversely affected the safety of the patient or could have impaired the assessment of study results.\n5. A diagnosis of other uncontrolled neurological diseases or presence of relevant somatic disorders not related to BMD that could have interfered with the ability to perform the muscle function tests and/or to comply with the study protocol procedures.\n6. Platelet count, white blood cell (WBC) count and hemoglobin at screening less than the lower limit of normal (LLN). If laboratory screening results were \\< LLN, platelet count, WBC count and hemoglobin were to be repeated once, and if again \\< LLN became exclusionary.\n7. Symptomatic cardiomyopathy or heart failure (New York Heart Association Class III or IV) or left ventricular ejection fraction \\< 50% at screening or with heart transplant.\n8. Current liver disease or impairment, including but not limited to elevated total bilirubin (\\>. 1.5 x upper limit of normal \\[ULN\\]), unless secondary to Gilbert's disease or pattern consistent with Gilbert's disease.\n9. Inadequate renal function defined by serum cystatin C \\> 2 x ULN. If the value was \\> 2 x ULN, serum Cystatin C was to be repeated once, and if again \\> 2 x ULN became exclusionary.\n10. Positive test for hepatitis B surface antigen, hepatitis C antibody, or human immunodeficiency virus at screening.\n11. Baseline corrected QT interval using Fridericia's correction (QTcF) \\> 450 msec (as the mean of 3 consecutive readings 5 minutes apart) or history of additional risk factors for torsades de pointes (e.g., heart failure, hypokalemia, or family history of long QT syndrome).\n12. Current psychiatric illness/social situations rendering the patient unable to understand or comply with the muscle function tests and/or with the study protocol procedures.\n13. Hypersensitivity to the components of the study medication.\n14. Sorbitol intolerance or sorbitol malabsorption, or the hereditary form of fructose intolerance.\n15. Contraindications for muscle biopsy.\n16. Contraindications forMRI/MRS (e.g., claustrophobia, metal implants or seizure disorders).\n17. Hypertriglyceridemia (˃ 1.5 x ULN). At screening, patients with hypertriglyceridemia could be enrolled if on stable treatment and with controlled levels of triglycerides (i.e., within normal range) for at least six months."}, 'identificationModule': {'nctId': 'NCT03238235', 'briefTitle': 'Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular Dystrophy', 'organization': {'class': 'INDUSTRY', 'fullName': 'Italfarmaco'}, 'officialTitle': 'A Randomised, Double Blind, Placebo Controlled Study to Evaluate the Micro-macroscopic Effects on Muscles, the Safety and Tolerability, and the Efficacy of Givinostat in Patients With Becker Muscular Dystrophy (BMD)', 'orgStudyIdInfo': {'id': 'DSC/15/2357/53'}, 'secondaryIdInfos': [{'id': '2017-001629-41', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Givinostat', 'description': 'Givinostat oral suspension (10 mg/mL) twice daily in a fed state', 'interventionNames': ['Drug: Givinostat']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Placebo oral suspension (10 mg/mL) twice daily in a fed state', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'Givinostat', 'type': 'DRUG', 'otherNames': ['ITF2357'], 'description': 'suspension of Givinostat (10 mg/mL)', 'armGroupLabels': ['Givinostat']}, {'name': 'Placebo', 'type': 'DRUG', 'otherNames': ['Placebo Comparator: placebo'], 'description': 'suspension manufactured to mimic Givinostat', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '20122', 'city': 'Milan', 'country': 'Italy', 'facility': "Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano, UOS", 'geoPoint': {'lat': 45.46427, 'lon': 9.18951}}, {'zip': 'ZH 2300 RC', 'city': 'Leiden', 'country': 'Netherlands', 'facility': 'Leiden University Medical Center LUMC', 'geoPoint': {'lat': 52.15833, 'lon': 4.49306}}], 'overallOfficials': [{'name': 'Giacomo Comi, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano"}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Italfarmaco', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}