Ignite Creation Date:
2025-12-24 @ 1:05 PM
Ignite Modification Date:
2025-12-31 @ 6:24 AM
Study NCT ID:
NCT01099761
Status:
TERMINATED
Last Update Posted:
2022-10-13
First Post:
2010-04-02
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Study of ACE-031 in Subjects With Duchenne 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'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'ClinicalTrialsDisclosure@merck.com', 'phone': '1-800-672-6372', 'title': 'Senior Vice President, Global Clinical Development', 'organization': 'Merck Sharp & Dohme LLC'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}}, 'adverseEventsModule': {'timeFrame': '24 weeks (12 weeks treatment period + 12 weeks follow up)', 'eventGroups': [{'id': 'EG000', 'title': 'ACE-031 0.5 mg/kg q4wk', 'description': 'ACE-031 0.5 mg/kg q4wk: ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.', 'otherNumAtRisk': 9, 'otherNumAffected': 3, 'seriousNumAtRisk': 9, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'ACE-031 1.0 mg/kg q2wk', 'description': 'ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.', 'otherNumAtRisk': 9, 'otherNumAffected': 6, 'seriousNumAtRisk': 9, 'seriousNumAffected': 0}, {'id': 'EG002', 'title': 'ACE-031 ACE-03 2.5 mg/kg q4wk', 'description': 'ACE-031 2.5 mg/kg q4wk: ACE-031 2.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.', 'otherNumAtRisk': 0, 'otherNumAffected': 0, 'seriousNumAtRisk': 0, 'seriousNumAffected': 0}, {'id': 'EG003', 'title': 'Placebo', 'description': 'Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.', 'otherNumAtRisk': 6, 'otherNumAffected': 3, 'seriousNumAtRisk': 6, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Injection site erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 9, 'numAffected': 6}, {'groupId': 'EG002', 'numAtRisk': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 6, 'numAffected': 3}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'epistaxis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numAffected': 5}, {'groupId': 'EG002', 'numAtRisk': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 6, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'telangiectasia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numAffected': 5}, {'groupId': 'EG002', 'numAtRisk': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 6, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 6, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Subjects With Adverse Reactions.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'ACE-031 0.5 mg/kg q4wk', 'description': 'ACE-031 0.5 mg/kg q4wk: ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.'}, {'id': 'OG001', 'title': 'ACE-031 1.0 mg/kg q2wk', 'description': 'ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'From treatment initiation to End-of-Study Visit, approximately 24 weeks later', 'description': 'Number of subjects in each cohort with a treatment-emergent adverse event considered at least possibly related to study drug', 'unitOfMeasure': 'Number of subjects', 'reportingStatus': 'POSTED'}, {'type': 'PRIMARY', 'title': 'Number of Subjects With Clinical Laboratory Adverse Reactions.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'ACE-031 0.5 mg/kg q4wk', 'description': 'ACE-031 0.5 mg/kg q4wk: ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.'}, {'id': 'OG001', 'title': 'ACE-031 1.0 mg/kg q2wk', 'description': 'ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline to End-of-Study Visit, approximately 24 weeks later.', 'description': 'Number of subjects in each cohort with treatment-emergent adverse laboratory values judged to be at least possibly related to study drug', 'unitOfMeasure': 'Number of subjects', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Percent Change in Total Lean Body Mass by DXA Scan.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'ACE-031 0.5 mg/kg q4wk', 'description': 'ACE-031 0.5 mg/kg q4wk: ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.'}, {'id': 'OG001', 'title': 'ACE-031 1.0 mg/kg q2wk', 'description': 'ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '3.6', 'spread': '1.3', 'groupId': 'OG000'}, {'value': '4.1', 'spread': '1.5', 'groupId': 'OG001'}, {'value': '2.6', 'spread': '1.8', 'groupId': 'OG002'}]}]}], 'analyses': [{'pValue': '0.023', 'groupIds': ['OG000'], 'ciNumSides': 'TWO_SIDED', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER_LEGACY'}, {'pValue': '0.012', 'groupIds': ['OG001'], 'ciNumSides': 'TWO_SIDED', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER_LEGACY'}, {'pValue': '0.435', 'groupIds': ['OG002'], 'ciNumSides': 'TWO_SIDED', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER_LEGACY'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to End-of-Study Visit, approximately 24 weeks later.', 'unitOfMeasure': 'percentage change in lean body mass', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Percent Change in Lumbar Spine Bone Mineral Density by DXA Scan.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'ACE-031 0.5 mg/kg q4wk', 'description': 'ACE-031 0.5 mg/kg q4wk: ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.'}, {'id': 'OG001', 'title': 'ACE-031 1.0 mg/kg q2wk', 'description': 'ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '1.6', 'spread': '1.8', 'groupId': 'OG000'}, {'value': '4.4', 'spread': '1.5', 'groupId': 'OG001'}, {'value': '0.3', 'spread': '4.4', 'groupId': 'OG002'}]}]}], 'analyses': [{'pValue': '0.039', 'groupIds': ['OG001'], 'ciNumSides': 'TWO_SIDED', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER_LEGACY'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to End-of-Study Visit, approximately 24 weeks later.', 'unitOfMeasure': 'percentage change from baseline', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Percent Change in Muscle Strength Score by Hand-held Myometry.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'ACE-031 0.5 mg/kg q4wk', 'description': 'ACE-031 0.5 mg/kg q4wk: ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.'}, {'id': 'OG001', 'title': 'ACE-031 1.0 mg/kg q2wk', 'description': 'ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.'}], 'classes': [{'title': 'Elbow extension (kg)', 'categories': [{'measurements': [{'value': '0.3', 'spread': '3.2', 'groupId': 'OG000'}, {'value': '0.1', 'spread': '2.3', 'groupId': 'OG001'}, {'value': '-0.1', 'spread': '2.1', 'groupId': 'OG002'}]}]}, {'title': 'Elbow flexion (kg)', 'categories': [{'measurements': [{'value': '-0.4', 'spread': '3.4', 'groupId': 'OG000'}, {'value': '-0.7', 'spread': '2.6', 'groupId': 'OG001'}, {'value': '0.3', 'spread': '3.1', 'groupId': 'OG002'}]}]}, {'title': 'Knee extension (kg)', 'categories': [{'measurements': [{'value': '-3.6', 'spread': '4.4', 'groupId': 'OG000'}, {'value': '-3.3', 'spread': '4.4', 'groupId': 'OG001'}, {'value': '-3.7', 'spread': '5.0', 'groupId': 'OG002'}]}]}, {'title': 'Knee flexion (kg)', 'categories': [{'measurements': [{'value': '-2.2', 'spread': '3.1', 'groupId': 'OG000'}, {'value': '-1.9', 'spread': '4.1', 'groupId': 'OG001'}, {'value': '-1.2', 'spread': '3.9', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to End-of-Study Visit, approximately 24 weeks later.', 'description': "Manual Muscle Testing (MMT) is a procedure to measure the function and strength of individual muscles and muscle groups. Hand-held myometry, using a device known as a dynamometer, is one method used for MMT. The dynamometer is held against the patient's limb by the examiner and the patient is asked to resist the force applied by the examiner. The dynamometer measures the force applied by the patient, providing a quantitative and objective assessment of strength of the particular muscle or muscle group. The effectiveness of a therapeutic intervention on muscle strength, as measured by hand-held myometry, can be assessed by comparing post-treatment to pre-treatment (baseline) measurements.", 'unitOfMeasure': 'percentage change from baseline', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change in Distance Traveled in 6 Minutes (Standardized 6-Minute-Walk Test).', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'ACE-031 0.5 mg/kg q4wk', 'description': 'ACE-031 0.5 mg/kg q4wk: ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.'}, {'id': 'OG001', 'title': 'ACE-031 1.0 mg/kg q2wk', 'description': 'ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.'}], 'classes': [{'title': '<10 years of age at baseline', 'categories': [{'measurements': [{'value': '43.8', 'spread': '61.5', 'groupId': 'OG000'}, {'value': '2.5', 'spread': '35.6', 'groupId': 'OG001'}, {'value': '5.2', 'spread': '23.6', 'groupId': 'OG002'}]}]}, {'title': '>=10 years of age at baseline', 'categories': [{'measurements': [{'value': '4.5', 'spread': '37.6', 'groupId': 'OG000'}, {'value': '-3.2', 'spread': '35.5', 'groupId': 'OG001'}, {'value': '-47.6', 'spread': '34.5', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to End-of-Study Visit, approximately 24 weeks later.', 'description': 'Change in distance traveled in 6 minutes (standardized 6-Minute-Walk Test); stratified by baseline age (\\<10 years vs. \\>=10 years)', 'unitOfMeasure': 'change (m) in 6MWT from baseline', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Time to Travel 10 Meters (Standardized 10-Meter-Walk/Run Test).', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'ACE-031 0.5 mg/kg q4wk', 'description': 'ACE-031 0.5 mg/kg q4wk: ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.'}, {'id': 'OG001', 'title': 'ACE-031 1.0 mg/kg q2wk', 'description': 'ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.7', 'spread': '0.9', 'groupId': 'OG000'}, {'value': '0.7', 'spread': '0.9', 'groupId': 'OG001'}, {'value': '1.0', 'spread': '0.8', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to End-of-Study Visit, approximately 24 weeks later.', 'unitOfMeasure': 'Change (sec) in 10MWT from baseline', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change in Pulmonary Function Tests (FVC)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'ACE-031 0.5 mg/kg q4wk', 'description': 'ACE-031 0.5 mg/kg q4wk: ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.'}, {'id': 'OG001', 'title': 'ACE-031 1.0 mg/kg q2wk', 'description': 'ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.1', 'spread': '0.2', 'groupId': 'OG000'}, {'value': '0.1', 'spread': '0.1', 'groupId': 'OG001'}, {'value': '0.1', 'spread': '0.2', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to End-of-Study Visit, approximately 24 weeks later.', 'description': 'Forced Vital Capacity (FVC); 1 of 3 separate tests employed to assess pulmonary function in this study', 'unitOfMeasure': 'Liters', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change in Pulmonary Function Test (MIP)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'ACE-031 0.5 mg/kg q4wk', 'description': 'ACE-031 0.5 mg/kg q4wk: ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.'}, {'id': 'OG001', 'title': 'ACE-031 1.0 mg/kg q2wk', 'description': 'ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '17.2', 'spread': '17.2', 'groupId': 'OG000'}, {'value': '8.7', 'spread': '7.6', 'groupId': 'OG001'}, {'value': '8.8', 'spread': '22.1', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to End-of-Study Visit. approximately 24 weeks', 'description': 'Maximum Inspiratory Pressure (MIP); 2 of 3 separate tests employed to assess pulmonary function in this study', 'unitOfMeasure': 'cm H2O', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change in Pulmonary Function Test (MEP)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'ACE-031 0.5 mg/kg q4wk', 'description': 'ACE-031 0.5 mg/kg q4wk: ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.'}, {'id': 'OG001', 'title': 'ACE-031 1.0 mg/kg q2wk', 'description': 'ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '11.9', 'spread': '12.5', 'groupId': 'OG000'}, {'value': '0.8', 'spread': '3.9', 'groupId': 'OG001'}, {'value': '5.8', 'spread': '12.1', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to End-of-Stuidy Visit, approximately 24 weeks', 'description': 'Maximum Expiratory Pressure (MEP); 3 of 3 separate tests employed to assess pulmonary function in this study', 'unitOfMeasure': 'cm H2O', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'ACE-031 0.5 mg/kg q4wk', 'description': 'ACE-031 0.5 mg/kg q4wk: ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.'}, {'id': 'FG001', 'title': 'ACE-031 1.0 mg/kg q2wk', 'description': 'ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.'}, {'id': 'FG002', 'title': 'ACE-031 ACE-03 2.5 mg/kg q4wk', 'description': 'ACE-031 2.5 mg/kg q4wk: ACE-031 2.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.'}, {'id': 'FG003', 'title': 'Placebo', 'description': 'Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '9'}, {'groupId': 'FG001', 'numSubjects': '9'}, {'comment': 'Study terminated prior to enrolling cohort 3', 'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '6'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '9'}, {'groupId': 'FG001', 'numSubjects': '9'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '6'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '6', 'groupId': 'BG003'}, {'value': '24', 'groupId': 'BG004'}]}], 'groups': [{'id': 'BG000', 'title': 'ACE-031 0.5 mg/kg q4wk', 'description': 'ACE-031 0.5 mg/kg q4wk: ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.'}, {'id': 'BG001', 'title': 'ACE-031 1.0 mg/kg q2wk', 'description': 'ACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.'}, {'id': 'BG002', 'title': 'ACE-031 ACE-03 2.5 mg/kg q4wk', 'description': 'ACE-031 2.5 mg/kg q4wk: ACE-031 2.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.'}, {'id': 'BG003', 'title': 'Placebo', 'description': 'Placebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.'}, {'id': 'BG004', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '9', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG003'}, {'value': '24', 'groupId': 'BG004'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'Male', 'measurements': [{'value': '9', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG003'}, {'value': '24', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '9', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG003'}, {'value': '24', 'groupId': 'BG004'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'Canada', 'categories': [{'measurements': [{'value': '9', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG003'}, {'value': '24', 'groupId': 'BG004'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Able to ambulate 10 meters in < 12 seconds', 'classes': [{'categories': [{'measurements': [{'value': '9', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG003'}, {'value': '24', 'groupId': 'BG004'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 24}}, 'statusModule': {'whyStopped': 'Based on preliminary safety data.', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2010-04'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-09', 'dispFirstSubmitDate': '2013-01-24', 'completionDateStruct': {'date': '2011-06', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2022-09-14', 'studyFirstSubmitDate': '2010-04-02', 'dispFirstSubmitQcDate': '2013-01-31', 'resultsFirstSubmitDate': '2016-05-10', 'studyFirstSubmitQcDate': '2010-04-06', 'dispFirstPostDateStruct': {'date': '2013-02-04', 'type': 'ESTIMATED'}, 'lastUpdatePostDateStruct': {'date': '2022-10-13', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2016-08-22', 'studyFirstPostDateStruct': {'date': '2010-04-08', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2016-10-14', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2011-06', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Subjects With Adverse Reactions.', 'timeFrame': 'From treatment initiation to End-of-Study Visit, approximately 24 weeks later', 'description': 'Number of subjects in each cohort with a treatment-emergent adverse event considered at least possibly related to study drug'}, {'measure': 'Number of Subjects With Clinical Laboratory Adverse Reactions.', 'timeFrame': 'Baseline to End-of-Study Visit, approximately 24 weeks later.', 'description': 'Number of subjects in each cohort with treatment-emergent adverse laboratory values judged to be at least possibly related to study drug'}], 'secondaryOutcomes': [{'measure': 'Percent Change in Total Lean Body Mass by DXA Scan.', 'timeFrame': 'Baseline to End-of-Study Visit, approximately 24 weeks later.'}, {'measure': 'Percent Change in Lumbar Spine Bone Mineral Density by DXA Scan.', 'timeFrame': 'Baseline to End-of-Study Visit, approximately 24 weeks later.'}, {'measure': 'Percent Change in Muscle Strength Score by Hand-held Myometry.', 'timeFrame': 'Baseline to End-of-Study Visit, approximately 24 weeks later.', 'description': "Manual Muscle Testing (MMT) is a procedure to measure the function and strength of individual muscles and muscle groups. Hand-held myometry, using a device known as a dynamometer, is one method used for MMT. The dynamometer is held against the patient's limb by the examiner and the patient is asked to resist the force applied by the examiner. The dynamometer measures the force applied by the patient, providing a quantitative and objective assessment of strength of the particular muscle or muscle group. The effectiveness of a therapeutic intervention on muscle strength, as measured by hand-held myometry, can be assessed by comparing post-treatment to pre-treatment (baseline) measurements."}, {'measure': 'Change in Distance Traveled in 6 Minutes (Standardized 6-Minute-Walk Test).', 'timeFrame': 'Baseline to End-of-Study Visit, approximately 24 weeks later.', 'description': 'Change in distance traveled in 6 minutes (standardized 6-Minute-Walk Test); stratified by baseline age (\\<10 years vs. \\>=10 years)'}, {'measure': 'Change From Baseline in Time to Travel 10 Meters (Standardized 10-Meter-Walk/Run Test).', 'timeFrame': 'Baseline to End-of-Study Visit, approximately 24 weeks later.'}, {'measure': 'Change in Pulmonary Function Tests (FVC)', 'timeFrame': 'Baseline to End-of-Study Visit, approximately 24 weeks later.', 'description': 'Forced Vital Capacity (FVC); 1 of 3 separate tests employed to assess pulmonary function in this study'}, {'measure': 'Change in Pulmonary Function Test (MIP)', 'timeFrame': 'Baseline to End-of-Study Visit. approximately 24 weeks', 'description': 'Maximum Inspiratory Pressure (MIP); 2 of 3 separate tests employed to assess pulmonary function in this study'}, {'measure': 'Change in Pulmonary Function Test (MEP)', 'timeFrame': 'Baseline to End-of-Stuidy Visit, approximately 24 weeks', 'description': 'Maximum Expiratory Pressure (MEP); 3 of 3 separate tests employed to assess pulmonary function in this study'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'conditions': ['Duchenne Muscular Dystrophy']}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to determine if ACE-031 is safe and well-tolerated in boys with Duchenne Muscular Dystrophy (DMD) and to select the optimal doses of ACE-031 in terms of safety and pharmacodynamic (PD) activity for designing future studies. \\[Note: This study was terminated based on safety data\\]', 'detailedDescription': 'ACE-031, a soluble form of the human activin receptor type IIB, was administered once every 2 to 4 weeks by subcutaneous (SC) injection to boys with DMD. Dose levels and regimens for this multiple-dose study were based on data from the initial clinical studies in healthy subjects in which doses of 0.02 to 3 mg/kg SC were evaluated. A total of 24 subjects were enrolled into the study; 18 received ACE-031 and 6 placebo. All subjects were treated for a period of 12 weeks.The pharmacodynamic effects of ACE-031 treatment were assessed by a battery of motor function test that included the 6-Minute Walk Test, the 10-Minute Walk/Run Test, the 4-Stair Climb Test and the Gower Maneuver (GW). Muscle strength was assessed by hand-held myometry and fixed system testing. Body composition (i.e., spine BMD, lean mass, and fat mass) was assessed by whole body and lumbar spine DXA scans. Pulmonary function was assessed by forced vital capacity (FVC), maximal inspiratory pressure (MIP) and maximal expiratory pressure (MEP). ACE-031 safety was evaluated through observation of the incidence and severity of adverse events.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '4 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Diagnosis of DMD confirmed\n* Ambulant\n* Corticosteroid therapy for at least one year prior to study day 1 and on a stable dose and schedule for at least 6 months prior to study day 1\n* Evidence of muscle weakness by clinical assessment\n\nExclusion Criteria:\n\n* Any previous treatment with another investigational product within 6 months prior to study day 1\n* Any clinically significant cardiac, endocrine, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, renal, and/or other major disease that is not related to DMD\n* Inability to perform a whole body dual x-ray absorptiometry (DXA) scan'}, 'identificationModule': {'nctId': 'NCT01099761', 'briefTitle': 'Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy', 'organization': {'class': 'INDUSTRY', 'fullName': 'Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA'}, 'officialTitle': 'A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular Dystrophy', 'orgStudyIdInfo': {'id': 'A031-03'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'ACE-031 0.5 mg/kg q4wk', 'interventionNames': ['Biological: ACE-031 0.5 mg/kg q4wk']}, {'type': 'EXPERIMENTAL', 'label': 'ACE-031 1.0 mg/kg q2wk', 'interventionNames': ['Biological: ACE-031 1.0 mg/kg q2wk']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'interventionNames': ['Other: Placebo']}], 'interventions': [{'name': 'ACE-031 0.5 mg/kg q4wk', 'type': 'BIOLOGICAL', 'description': 'ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.', 'armGroupLabels': ['ACE-031 0.5 mg/kg q4wk']}, {'name': 'ACE-031 1.0 mg/kg q2wk', 'type': 'BIOLOGICAL', 'description': 'ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.', 'armGroupLabels': ['ACE-031 1.0 mg/kg q2wk']}, {'name': 'Placebo', 'type': 'OTHER', 'description': 'Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Calgary', 'state': 'Alberta', 'country': 'Canada', 'facility': 'Acceleron Investigative Site', 'geoPoint': {'lat': 51.05011, 'lon': -114.08529}}, {'city': 'Vancouver', 'state': 'British Columbia', 'country': 'Canada', 'facility': 'Acceleron Investigative Site', 'geoPoint': {'lat': 49.24966, 'lon': -123.11934}}, {'city': 'Hamilton', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Acceleron Investigative Site', 'geoPoint': {'lat': 43.25011, 'lon': -79.84963}}, {'city': 'London', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Acceleron Investigative Site', 'geoPoint': {'lat': 42.98339, 'lon': -81.23304}}, {'city': 'Ottawa', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Acceleron Investigative Site', 'geoPoint': {'lat': 45.41117, 'lon': -75.69812}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO', 'description': 'Abstract summarizing trial data has been published online in Muscle \\& Nerve 23-Dec-2016 https://www.ncbi.nlm.nih.gov/pubmed/27462804 The Sponor currently has no plans to make IPD available for a number of reasons; (i) the study was terminated by the Sponsor prematurely based upon concerns for potential adverse drug reactions following long-term use, which were identified in chronic toxicity studies in animals, (ii) the study population was one with a rare disease, in a groups of subjects with a relatively narrow age range, across a small number of study sites. The Sponsor believes that these factors, taken together, make patient anonymity a significant challenge.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}