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Ignite Creation Date: 2025-12-25 @ 1:25 AM

Ignite Modification Date: 2025-12-27 @ 10:48 PM

Study NCT ID: NCT05176093

Status: COMPLETED

Last Update Posted: 2025-10-06

First Post: 2021-11-05

Is NOT Gene Therapy: True

Has Adverse Events: True

Brief Title: A 6-Month Extension Study to Assess the Long-Term Safety of Engensis in Amyotrophic Lateral Sclerosis

Sponsor:

Organization:

Overview Dates Organization Conditions Design Enrollment Locations Outcomes Modules Raw JSON

Raw JSON

{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2025-03-25', 'type': 'ACTUAL'}}}}, 'conditionBrowseModule': {'meshes': [{'id': 'D000690', 'term': 'Amyotrophic Lateral Sclerosis'}], 'ancestors': [{'id': 'D013118', 'term': 'Spinal Cord Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D016472', 'term': 'Motor Neuron Disease'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D057177', 'term': 'TDP-43 Proteinopathies'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D057165', 'term': 'Proteostasis Deficiencies'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'jinsub.lee@helixmith.com', 'phone': '+82-10-8256-0439', 'title': 'Jinsub Lee, PhD.', 'organization': 'Helixmith Co., Ltd.'}, 'certainAgreement': {'restrictionType': 'LTE60', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Day 0 to Day 365, or until participant withdraws informed consent for this extension study, which can be anytime between Day 180 and Day 365.', 'description': 'All study-related Adverse Events will be collected starting after completion of the Informed Consent process at Screening to Day 0/randomization of the VMALS-002-2 study. Treatment-Emergent Adverse Events and Treatment-Emergent Serious Adverse Events will be collected after first injections of VMALS-002-2 study until the time that the Participant withdraws their informed consent for this extension study.', 'eventGroups': [{'id': 'EG000', 'title': 'Engensis', 'description': 'Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart\n\nEngensis: Lyophilized biologic to be reconstituted containing Engensis', 'otherNumAtRisk': 4, 'deathsNumAtRisk': 4, 'otherNumAffected': 4, 'seriousNumAtRisk': 4, 'deathsNumAffected': 0, 'seriousNumAffected': 1}, {'id': 'EG001', 'title': 'Placebo', 'description': 'Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart\n\nPlacebo: Injectable Liquid', 'otherNumAtRisk': 4, 'deathsNumAtRisk': 4, 'otherNumAffected': 4, 'seriousNumAtRisk': 4, 'deathsNumAffected': 0, 'seriousNumAffected': 1}], 'otherEvents': [{'term': 'Injection site bruising', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 6, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 9, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Asthenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 4, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Injection site pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Injection site reaction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Productive cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Dysphonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Dyspnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Oropharyneal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Dysphagia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Haemorrhoids thrombosed', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Muscle spasms', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Myalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Muscle twitching', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'COVID-19', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Kidney infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Dysarthria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Muscle contractions involuntary', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Paraesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Sinus tachycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Multiple injuries', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Skin irritation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Peripheral arterial occlusive disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Tachycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Tinnitus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Bone density descreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Hypophagia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Urinary retention', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Dysmenorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}], 'seriousEvents': [{'term': 'Dyspnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}, {'term': 'Peripheral arterialocclusive disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MeDRA 23.0'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Treatment-Emergent Adverse Events in More Than 2 Subjects Overall by System Organ Class and Preferred Term', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Engensis', 'description': 'Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart\n\nEngensis: Lyophilized biologic to be reconstituted containing Engensis'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart\n\nPlacebo: Injectable Liquid'}], 'classes': [{'title': 'Injection site bruising', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}]}, {'title': 'Asthenia', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}]}, {'title': 'Pyrexia', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Injection site pain', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Cough', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Rhinorrhoea', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Dysphagia', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}]}, {'title': 'Arthralgia', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Myalgia', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'COVID-19', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}]}, {'title': 'Headache', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Day 0 to Day 365', 'description': 'To evaluate the long-term safety of intramuscular injections of Engensis in Participants with Amyotrophic Lateral Sclerosis in more than 2 Participants by System Organ Class and Preferred Term (Safety Analysis Population)', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Percentages were based on the number of subjects in the Safety Analysis Population by treatment group and overall.\n\nSubjects were counted once within for each unique Preferred Term.'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'To Evaluate Changes in Muscle Function Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Engensis', 'description': 'Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart\n\nEngensis: Lyophilized biologic to be reconstituted containing Engensis'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart\n\nPlacebo: Injectable Liquid'}], 'classes': [{'title': 'Baseline - actual value', 'categories': [{'measurements': [{'value': '39.0', 'spread': '1.41', 'groupId': 'OG000'}, {'value': '30.8', 'spread': '5.62', 'groupId': 'OG001'}]}]}, {'title': 'Day 365 / Early Termination - change from baseline', 'categories': [{'measurements': [{'value': '-10.5', 'spread': '8.23', 'groupId': 'OG000'}, {'value': '-8.7', 'spread': '9.61', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Day 0 and Day 365', 'description': 'Changes from Baseline (Study VMALS 002-2, Day 0) in Revised Amyotrophic Lateral Sclerosis Function Rating TOTAL scores at Day 365 for Engensis compared to Placebo\n\nThere are 12 separate assessments in this neuromuscular assessment covering Bulbar, Fine Motor, Gross Motor and Breathing assessments. Each individual assessment is scored from 0 (worst case) to 4 (best case).\n\nTotal score is calculated as the sum of all 12 assessment subscores. Note that only the Total Score will be reported in the clinical study report due to the small number of subjects.\n\nThe Total Score range is from a minimum of 0 (worst case) to a maximum of 48 (best case).', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Revised Amyotrophic Lateral Sclerosis Function Rating Total Score and Change from Baseline to Day 365 (Safety Analysis Population)'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'To Evaluate Muscle Strength Changes Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Engensis', 'description': 'Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart\n\nEngensis: Lyophilized biologic to be reconstituted containing Engensis'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart\n\nPlacebo: Injectable Liquid'}], 'classes': [{'title': 'Shoulder Flexion-Left - Baseline - Actual', 'categories': [{'measurements': [{'value': '24.10', 'spread': '12.572', 'groupId': 'OG000'}, {'value': '20.08', 'spread': '11.538', 'groupId': 'OG001'}]}]}, {'title': 'Shoulder Flexion-Left - Day 365 - Change from Baseline', 'categories': [{'measurements': [{'value': '-16.13', 'spread': '12.419', 'groupId': 'OG000'}, {'value': '-8.40', 'spread': '5.173', 'groupId': 'OG001'}]}]}, {'title': 'Shoulder Flexion- Right - Baseline - Actual', 'categories': [{'measurements': [{'value': '24.645', 'spread': '11.5448', 'groupId': 'OG000'}, {'value': '17.850', 'spread': '12.0423', 'groupId': 'OG001'}]}]}, {'title': 'Shoulder Flexion-Right - Day 365 - Change from Baseline', 'categories': [{'measurements': [{'value': '-16.395', 'spread': '12.8508', 'groupId': 'OG000'}, {'value': '-5.900', 'spread': '5.1798', 'groupId': 'OG001'}]}]}, {'title': 'Elbow Flexion- Left - Baseline - Actual', 'categories': [{'measurements': [{'value': '29.440', 'spread': '25.1779', 'groupId': 'OG000'}, {'value': '14.525', 'spread': '10.1923', 'groupId': 'OG001'}]}]}, {'title': 'Elbow Flexion-Left - Day 365 - Change from Baseline', 'categories': [{'measurements': [{'value': '-20.190', 'spread': '15.8662', 'groupId': 'OG000'}, {'value': '-5.433', 'spread': '3.6501', 'groupId': 'OG001'}]}]}, {'title': 'Elbow Flexion- Right - Baseline - Actual', 'categories': [{'measurements': [{'value': '30.53', 'spread': '26.522', 'groupId': 'OG000'}, {'value': '13.73', 'spread': '11.667', 'groupId': 'OG001'}]}]}, {'title': 'Elbow Flexion-Right - Day 365 - Change from Baseline', 'categories': [{'measurements': [{'value': '-20.45', 'spread': '16.083', 'groupId': 'OG000'}, {'value': '-4.1', 'spread': '1.082', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Day 0 and Day 365', 'description': 'As assessed bilaterally by Hand-Held Dynamometry in muscles in the upper and lower extremities. Muscle Strength as Measured by Handheld Dynamometry (lbs.) and Change from Baseline to Day 365 by Muscle Group (ITT Population)', 'unitOfMeasure': 'Pounds', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT Population'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'To Determine Whether Intramuscular Administration of Engensis Has Effects on Respiratory Capacity in Amyotrophic Lateral Sclerosis Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Engensis', 'description': 'Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart\n\nEngensis: Lyophilized biologic to be reconstituted containing Engensis'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart\n\nPlacebo: Injectable Liquid'}], 'classes': [{'title': 'Day 240 - Change from Baseline', 'categories': [{'measurements': [{'value': '0.0', 'spread': '1.41', 'groupId': 'OG000'}, {'value': '-16.5', 'spread': '7.78', 'groupId': 'OG001'}]}]}, {'title': 'Day 300 - Change from Baseline', 'categories': [{'measurements': [{'value': '-9.3', 'spread': '11.90', 'groupId': 'OG000'}, {'value': '-7.3', 'spread': '19.33', 'groupId': 'OG001'}]}]}, {'title': 'Day 365/Early Term - Change from Baseline', 'categories': [{'measurements': [{'value': '-17.3', 'spread': '19.39', 'groupId': 'OG000'}, {'value': '-15.0', 'spread': '17.52', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Day 0, Day 240, Day 300 and Day 365', 'description': 'Slow vital capacity is a pulmonary function test that quantifies the volume of air that can be slowly exhaled after slow maximum inhalation. It is measured in Liters of air and the percentage of change from baseline is the Percent Predicted Value and Change from Baseline.\n\nChange from Baseline (Study VMALS 002-2, Day 0) in Slow Vital Capacity on Day 240, Day 300 and Day 365 for Participants with intramuscular administration of Engensis compared to Placebo. Increasing negative Change from Baseline results indicates worsening of respiratory capacity.', 'unitOfMeasure': 'percentage of Change from Baseline', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Slow Vital Capacity Percent Change from Baseline by Visit (ITT Population)'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'To Determine if Intramuscular Engensis Has Effects on Respiratory Function in Amyotrophic Lateral Sclerosis Participants - Tracheostomy', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Engensis', 'description': 'Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart\n\nEngensis: Lyophilized biologic to be reconstituted containing Engensis'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart\n\nPlacebo: Injectable Liquid'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Day 0 to Day 365', 'description': 'Total participants requiring Tracheostomy procedures that received intramuscular administration of Engensis, compared to total Placebo participants requiring Tracheostomy procedures.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Total Tracheostomy procedures for the Safety Analysis Population - Number of Participants - Day 0 to Day 365'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'To Determine if Intramuscular Administration of Engensis Has Positive Effects on Survival in Amyotrophic Lateral Sclerosis Participants - All-Cause Mortality', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Engensis', 'description': 'Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart\n\nEngensis: Lyophilized biologic to be reconstituted containing Engensis'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart\n\nPlacebo: Injectable Liquid'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Day 0 to Day 365', 'description': 'Total all-cause Mortality for participants that received intramuscular injections of Engensis, compared to total all-cause Mortality for Placebo participants.\n\nTotal participant deaths that received intramuscular administration of Engensis, compared to total Placebo participant deaths.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Total all-cause Mortality for participants'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'To Evaluate Quality of Life Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Engensis', 'description': 'Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart\n\nEngensis: Lyophilized biologic to be reconstituted containing Engensis'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart\n\nPlacebo: Injectable Liquid'}], 'classes': [{'title': 'Day 240 - Physical mobility', 'categories': [{'measurements': [{'value': '12.5', 'spread': '2.500', 'groupId': 'OG000'}, {'value': '3.33', 'spread': '20.817', 'groupId': 'OG001'}]}]}, {'title': 'Day 365/Early Termination - Physical mobility', 'categories': [{'measurements': [{'value': '13.75', 'spread': '9.465', 'groupId': 'OG000'}, {'value': '21.67', 'spread': '24.664', 'groupId': 'OG001'}]}]}, {'title': 'Day 240 - Activities of Daily Living/Independence', 'categories': [{'measurements': [{'value': '14.17', 'spread': '11.815', 'groupId': 'OG000'}, {'value': '-.83', 'spread': '3.819', 'groupId': 'OG001'}]}]}, {'title': 'Day 365/Early Termination - Activities of Daily Living/Independence', 'categories': [{'measurements': [{'value': '21.88', 'spread': '21.250', 'groupId': 'OG000'}, {'value': '10.00', 'spread': '8.660', 'groupId': 'OG001'}]}]}, {'title': 'Day 240 - Eating and Drinking', 'categories': [{'measurements': [{'value': '0', 'spread': '0', 'groupId': 'OG000'}, {'value': '11.13', 'spread': '25.411', 'groupId': 'OG001'}]}]}, {'title': 'Day 365/Early Termination - Eating and Drinking', 'categories': [{'measurements': [{'value': '20.83', 'spread': '24.994', 'groupId': 'OG000'}, {'value': '33.33', 'spread': '28.868', 'groupId': 'OG001'}]}]}, {'title': 'Day 240 - Communication', 'categories': [{'measurements': [{'value': '16.67', 'spread': '32.036', 'groupId': 'OG000'}, {'value': '28.57', 'spread': '6.207', 'groupId': 'OG001'}]}]}, {'title': 'Day 365/Early Termination - Communication', 'categories': [{'measurements': [{'value': '26.80', 'spread': '33.350', 'groupId': 'OG000'}, {'value': '32.17', 'spread': '35.700', 'groupId': 'OG001'}]}]}, {'title': 'Day 240 - Emotional Functioning', 'categories': [{'measurements': [{'value': '3.33', 'spread': '13.769', 'groupId': 'OG000'}, {'value': '7.50', 'spread': '18.875', 'groupId': 'OG001'}]}]}, {'title': 'Day 365/Early Termination - Emotional Functioning', 'categories': [{'measurements': [{'value': '10.00', 'spread': '24.833', 'groupId': 'OG000'}, {'value': '12.50', 'spread': '4.330', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Day 0, Day 240 and Day 365', 'description': 'The Participant completes the 40 questions in the Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40) with 5 categories: Physical Mobility, Activities of Daily Living and Independence, Eating and Drinking, Communication, and Emotional Reactions. Each question has 5 responses to select from: 0-Never (Best Case), 1-Rarely, 2-Sometime, 3-Often, and 4-Always (Worst Case).\n\nNote for each question there is a Minimum of 0 (Best), to the Maximum 4 (Worst). Decreasing scores indicates improvement of symptoms. The Total Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40) scores are per Category as percentages of 100% maximum. Each Category percentage is summed for the Intent-to-treat Population.\n\nThe Change from Baseline was calculated and displayed in the Table. ALSAQ-40 was completed at the pre-dose baseline visit (Day 0), and at the Day 240 and Day 365 visits.', 'unitOfMeasure': 'percentage of Change from Baseline', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Percent Change from Baseline (ITT Population) for Engensis and Placebo.'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'To Evaluate Patient Reported Outcome Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Engensis', 'description': 'Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart\n\nEngensis: Lyophilized biologic to be reconstituted containing Engensis'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart\n\nPlacebo: Injectable Liquid'}], 'classes': [{'title': 'Day 240 - Impression change - or worse', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}]}, {'title': 'Day 240 - Impression change - Almost the same, hardly any change', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Day 365/Early termination - Impression change - or worse', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}]}, {'title': 'Day 365/Early Termination - Impression change - Almost the same, hardly any change', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Day 240 and Day 365', 'description': "The subject's impression of change after treatment was measured with the Patient Global Impression of Change questionnaire through use of the electronic Patient Reported Outcome . This questionnaire measures the subject's perception of how treatment has affected their level of activity, symptoms, emotions, and overall quality of life.\n\nEach descriptor is ranked on an increasing improvement scale; where 1 = No change (or condition has got worse), 2=Almost the same, hardly any change at all, 3=A little better, but no noticeable change, 4=Somewhat better, but the change has not made any real difference, 5=Moderately better, and a slight but noticeable change, 6=Better, and a definite improvement that has made a real and worthwhile difference, and 7 = A great deal better, and a considerable improvement that as made all the difference.\n\nThe test was self-administered on Days 240 and 365.", 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Patient Global Impression of Change - ITT Population.'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'To Evaluate Clinician Reported Outcome Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Engensis', 'description': 'Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart\n\nEngensis: Lyophilized biologic to be reconstituted containing Engensis'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart\n\nPlacebo: Injectable Liquid'}], 'classes': [{'title': 'Day 240 - No change', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Day 240 - Minimally worse', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Day 240 - Much worse', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Day 365 / Early Termination - Minimally improved', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Day 365 / Early Termination - No change', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Day 365 / Early Termination Minimally worse', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Day 365 / Early Termination Much worse', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Day 240 and Day 365', 'description': 'The Clinical Global Impression of Change is a validated instrument completed by observers as an assessment of Quality of Life. The Clinical Global Impression of Change is an 8-point scale with scores ranging rom Marked Improvement, Moderate Improvement, Minimal Improvement, Slight Improvement, and Unchanged (or Worse), along with an efficacy index with questions in a matrix for therapeutic effect and side effects.The test was completed on Days 240 and 365/ Early Termination.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Clinical Global Impression of Change - ITT Population'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Engensis', 'description': 'Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart\n\nEngensis: Lyophilized biologic to be reconstituted containing Engensis'}, {'id': 'FG001', 'title': 'Placebo', 'description': 'Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart\n\nPlacebo: Injectable Liquid'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '4'}, {'groupId': 'FG001', 'numSubjects': '4'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '4'}, {'groupId': 'FG001', 'numSubjects': '3'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '8', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Engensis', 'description': 'Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart\n\nEngensis: Lyophilized biologic to be reconstituted containing Engensis'}, {'id': 'BG001', 'title': 'Placebo', 'description': 'Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart\n\nPlacebo: Injectable Liquid'}, {'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': '4', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '8', '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': '39.0', 'spread': '16.99', 'groupId': 'BG000'}, {'value': '52.3', 'spread': '10.63', 'groupId': 'BG001'}, {'value': '45.6', 'spread': '14.91', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}]}]}], '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': 'BG002'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '8', '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': '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': '3', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '5', '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': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'White', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '3', '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': 'South Korea', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}]}]}, {'title': 'United States', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}], 'populationDescription': 'Demographic and Baseline Characteristics (Safety Analysis Population)'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2021-10-12', 'size': 19948040, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2025-02-01T21:17', 'hasProtocol': True}, {'date': '2023-01-30', 'size': 591598, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2025-02-01T21:18', 'hasProtocol': False}]}}, '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': 8}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2021-11-14', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-08', 'completionDateStruct': {'date': '2024-08-23', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-09-23', 'studyFirstSubmitDate': '2021-11-05', 'resultsFirstSubmitDate': '2025-02-18', 'studyFirstSubmitQcDate': '2021-12-15', 'lastUpdatePostDateStruct': {'date': '2025-10-06', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2025-05-12', 'studyFirstPostDateStruct': {'date': '2022-01-04', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2025-05-29', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-12-29', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'To Evaluate Changes in Muscle Function Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants', 'timeFrame': 'Day 0 and Day 365', 'description': 'Changes from Baseline (Study VMALS 002-2, Day 0) in Revised Amyotrophic Lateral Sclerosis Function Rating TOTAL scores at Day 365 for Engensis compared to Placebo\n\nThere are 12 separate assessments in this neuromuscular assessment covering Bulbar, Fine Motor, Gross Motor and Breathing assessments. Each individual assessment is scored from 0 (worst case) to 4 (best case).\n\nTotal score is calculated as the sum of all 12 assessment subscores. Note that only the Total Score will be reported in the clinical study report due to the small number of subjects.\n\nThe Total Score range is from a minimum of 0 (worst case) to a maximum of 48 (best case).'}, {'measure': 'To Evaluate Muscle Strength Changes Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants', 'timeFrame': 'Day 0 and Day 365', 'description': 'As assessed bilaterally by Hand-Held Dynamometry in muscles in the upper and lower extremities. Muscle Strength as Measured by Handheld Dynamometry (lbs.) and Change from Baseline to Day 365 by Muscle Group (ITT Population)'}, {'measure': 'To Determine Whether Intramuscular Administration of Engensis Has Effects on Respiratory Capacity in Amyotrophic Lateral Sclerosis Participants', 'timeFrame': 'Day 0, Day 240, Day 300 and Day 365', 'description': 'Slow vital capacity is a pulmonary function test that quantifies the volume of air that can be slowly exhaled after slow maximum inhalation. It is measured in Liters of air and the percentage of change from baseline is the Percent Predicted Value and Change from Baseline.\n\nChange from Baseline (Study VMALS 002-2, Day 0) in Slow Vital Capacity on Day 240, Day 300 and Day 365 for Participants with intramuscular administration of Engensis compared to Placebo. Increasing negative Change from Baseline results indicates worsening of respiratory capacity.'}, {'measure': 'To Determine if Intramuscular Engensis Has Effects on Respiratory Function in Amyotrophic Lateral Sclerosis Participants - Tracheostomy', 'timeFrame': 'Day 0 to Day 365', 'description': 'Total participants requiring Tracheostomy procedures that received intramuscular administration of Engensis, compared to total Placebo participants requiring Tracheostomy procedures.'}, {'measure': 'To Determine if Intramuscular Administration of Engensis Has Positive Effects on Survival in Amyotrophic Lateral Sclerosis Participants - All-Cause Mortality', 'timeFrame': 'Day 0 to Day 365', 'description': 'Total all-cause Mortality for participants that received intramuscular injections of Engensis, compared to total all-cause Mortality for Placebo participants.\n\nTotal participant deaths that received intramuscular administration of Engensis, compared to total Placebo participant deaths.'}, {'measure': 'To Evaluate Quality of Life Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo', 'timeFrame': 'Day 0, Day 240 and Day 365', 'description': 'The Participant completes the 40 questions in the Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40) with 5 categories: Physical Mobility, Activities of Daily Living and Independence, Eating and Drinking, Communication, and Emotional Reactions. Each question has 5 responses to select from: 0-Never (Best Case), 1-Rarely, 2-Sometime, 3-Often, and 4-Always (Worst Case).\n\nNote for each question there is a Minimum of 0 (Best), to the Maximum 4 (Worst). Decreasing scores indicates improvement of symptoms. The Total Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40) scores are per Category as percentages of 100% maximum. Each Category percentage is summed for the Intent-to-treat Population.\n\nThe Change from Baseline was calculated and displayed in the Table. ALSAQ-40 was completed at the pre-dose baseline visit (Day 0), and at the Day 240 and Day 365 visits.'}, {'measure': 'To Evaluate Patient Reported Outcome Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo', 'timeFrame': 'Day 240 and Day 365', 'description': "The subject's impression of change after treatment was measured with the Patient Global Impression of Change questionnaire through use of the electronic Patient Reported Outcome . This questionnaire measures the subject's perception of how treatment has affected their level of activity, symptoms, emotions, and overall quality of life.\n\nEach descriptor is ranked on an increasing improvement scale; where 1 = No change (or condition has got worse), 2=Almost the same, hardly any change at all, 3=A little better, but no noticeable change, 4=Somewhat better, but the change has not made any real difference, 5=Moderately better, and a slight but noticeable change, 6=Better, and a definite improvement that has made a real and worthwhile difference, and 7 = A great deal better, and a considerable improvement that as made all the difference.\n\nThe test was self-administered on Days 240 and 365."}, {'measure': 'To Evaluate Clinician Reported Outcome Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo', 'timeFrame': 'Day 240 and Day 365', 'description': 'The Clinical Global Impression of Change is a validated instrument completed by observers as an assessment of Quality of Life. The Clinical Global Impression of Change is an 8-point scale with scores ranging rom Marked Improvement, Moderate Improvement, Minimal Improvement, Slight Improvement, and Unchanged (or Worse), along with an efficacy index with questions in a matrix for therapeutic effect and side effects.The test was completed on Days 240 and 365/ Early Termination.'}], 'primaryOutcomes': [{'measure': 'Treatment-Emergent Adverse Events in More Than 2 Subjects Overall by System Organ Class and Preferred Term', 'timeFrame': 'Day 0 to Day 365', 'description': 'To evaluate the long-term safety of intramuscular injections of Engensis in Participants with Amyotrophic Lateral Sclerosis in more than 2 Participants by System Organ Class and Preferred Term (Safety Analysis Population)'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Amyotrophic Lateral Sclerosis']}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to evaluate the long-term safety of intramuscular administration of Engensis in Participants with Amyotrophic Lateral Sclerosis who were previously randomized, received treatment, and completed the Day 180 Visit of Study VMALS-002-2. Safety will be assessed by incidences of treatment-emergent adverse events, treatment emergent serious adverse events, adverse events of special interest, and the clinically significant laboratory values.', 'detailedDescription': 'VMALS-002-2b was a safety extension study following VMALS-002-2, which was a Phase 2a, double-blind, randomized, placebo-controlled, multicenter study designed to assess the safety of intramuscular administration of Engensis in subjects with Amyotrophic Lateral Sclerosis.Following completion of the Day 180 visit in VMALS-002-2 and signing of written informed consent, subjects were enrolled in this approximately 180-day extension study, with subjects completing combined participation with a Day 365 visit. Assessments to be conducted during this extension study were as follows: vital signs, weight and height, complete physical examination, 12-lead electrocardiogram, a record of all concomitant medications and procedures, clinical laboratory assessments, Revised Amyotrophic Lateral Sclerosis Functional Rating Scale, handheld dynamometry, slow vital capacity, Amyotrophic Lateral Sclerosis Assessment Questionnaire, 40 questions, Patient Global Impression of Change, and Clinical Global Impression of Change. Assessments of treatment-emergent adverse events, treatment-emergent serious adverse events, adverse events of special interest, and clinically significant laboratory values continued from the Day 180 visit of Study VMALS-002-2 through the VMALS-002-2b extension study to Day 365.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '80 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Participants who complete the Day 180 Visit in VMALS-002-2 are eligible to enroll in this extension study, VMALS-002-2b.\n\nExclusion Criteria:\n\n\\- None'}, 'identificationModule': {'nctId': 'NCT05176093', 'acronym': 'REViVALS-1B', 'briefTitle': 'A 6-Month Extension Study to Assess the Long-Term Safety of Engensis in Amyotrophic Lateral Sclerosis', 'organization': {'class': 'INDUSTRY', 'fullName': 'Helixmith Co., Ltd.'}, 'officialTitle': 'A 6-Month Extension Study Following Protocol VMALS-002-2 (A Phase 2a, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Assess the Safety of Engensis in Participants With Amyotrophic Lateral Sclerosis)', 'orgStudyIdInfo': {'id': 'VMALS-002-2b'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'ACTIVE_COMPARATOR', 'label': 'Engensis', 'description': 'Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart', 'interventionNames': ['Biological: Engensis']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart', 'interventionNames': ['Other: Placebo']}], 'interventions': [{'name': 'Engensis', 'type': 'BIOLOGICAL', 'description': 'Lyophilized biologic to be reconstituted containing Engensis', 'armGroupLabels': ['Engensis']}, {'name': 'Placebo', 'type': 'OTHER', 'description': 'Injectable Liquid', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '78759', 'city': 'Austin', 'state': 'Texas', 'country': 'United States', 'facility': 'Austin Neuromuscular Center', 'geoPoint': {'lat': 30.26715, 'lon': -97.74306}}, {'zip': '04763', 'city': 'Seoul', 'country': 'South Korea', 'facility': 'Hanyang University Medical Center', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Helixmith Co., Ltd.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}