Ignite Creation Date:
2025-12-24 @ 2:22 PM
Ignite Modification Date:
2026-01-31 @ 9:53 AM
Study NCT ID:
NCT05643495
Status:
TERMINATED
Last Update Posted:
2025-09-08
First Post:
2022-11-29
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D019896', 'term': 'alpha 1-Antitrypsin Deficiency'}], 'ancestors': [{'id': 'D008107', 'term': 'Liver Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D008171', 'term': 'Lung Diseases'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D013352', 'term': 'Subcutaneous Emphysema'}, {'id': 'D004646', 'term': 'Emphysema'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'medicalinfo@vrtx.com', 'phone': '617-341-6777', 'title': 'Medical Monitor', 'organization': 'Vertex Pharmaceuticals Incorporated'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Day 1 up to Week 52', 'description': 'Safety set included all participants who had received at least 1 dose of study drug in this study.', 'eventGroups': [{'id': 'EG000', 'title': 'Group A VX-864 500 mg', 'description': 'Participants received VX-864 q12h for 48 weeks or until study drug dosing was terminated.', 'otherNumAtRisk': 10, 'deathsNumAtRisk': 10, 'otherNumAffected': 10, 'seriousNumAtRisk': 10, 'deathsNumAffected': 0, 'seriousNumAffected': 1}, {'id': 'EG001', 'title': 'Group B VX-864 500 mg', 'description': 'Participants undergo a liver biopsy before receiving VX-864 q12h for 48 weeks or until study drug dosing was terminated and undergo a second liver biopsy at either Week 24 or Week 48.', 'otherNumAtRisk': 4, 'deathsNumAtRisk': 4, 'otherNumAffected': 4, 'seriousNumAtRisk': 4, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Porphyria non-acute', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Congenital, familial and genetic disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Abdominal distension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Mouth ulceration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Paraesthesia oral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Hypertransaminasaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Diverticulitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Blood creatine phosphokinase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Hypercholesterolaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Joint swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Dysgeusia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Hyperaesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Irritability', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Benign prostatic hyperplasia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Blister', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Dermatitis contact', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Drug eruption', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Dyshidrotic eczema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Photosensitivity reaction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Post inflammatory pigmentation change', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Pruritus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Pseudoporphyria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Urticaria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}], 'seriousEvents': [{'term': 'Diverticulitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change in Blood Functional Alpha-1 Antitrypsin (AAT) Levels', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Group A VX-864 500 mg', 'description': 'Participants received VX-864 q12h for 48 weeks or until study drug dosing was terminated.'}, {'id': 'OG001', 'title': 'Group B VX-864 500 mg', 'description': 'Participants undergo a liver biopsy before receiving VX-864 q12h for 48 weeks or until study drug dosing was terminated and undergo a second liver biopsy at either Week 24 or Week 48.'}], 'timeFrame': 'From Baseline at Week 48', 'reportingStatus': 'POSTED', 'populationDescription': 'Data was not collected for this Outcome Measure as the study drug dosing was terminated prior to any participant reaching Week 48.'}, {'type': 'SECONDARY', 'title': 'Change in Blood Functional AAT Levels', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Group A VX-864 500 mg', 'description': 'Participants received VX-864 q12h for 48 weeks or until study drug dosing was terminated.'}, {'id': 'OG001', 'title': 'Group B VX-864 500 mg', 'description': 'Participants undergo a liver biopsy before receiving VX-864 q12h for 48 weeks or until study drug dosing was terminated and undergo a second liver biopsy at either Week 24 or Week 48.'}], 'timeFrame': 'From Baseline up to Week 48', 'reportingStatus': 'POSTED', 'populationDescription': 'Data was not collected for this Outcome Measure as the study drug dosing was terminated prior to any participant reaching Week 48.'}, {'type': 'SECONDARY', 'title': 'Change in Blood Antigenic AAT Levels', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Group A VX-864 500 mg', 'description': 'Participants received VX-864 q12h for 48 weeks or until study drug dosing was terminated.'}, {'id': 'OG001', 'title': 'Group B VX-864 500 mg', 'description': 'Participants undergo a liver biopsy before receiving VX-864 q12h for 48 weeks or until study drug dosing was terminated and undergo a second liver biopsy at either Week 24 or Week 48.'}], 'timeFrame': 'From Baseline up to Week 48', 'reportingStatus': 'POSTED', 'populationDescription': 'Data was not collected for this Outcome Measure as the study drug dosing was terminated prior to any participant reaching Week 48.'}, {'type': 'SECONDARY', 'title': 'Change in Blood Z-polymer Levels', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Group A VX-864 500 mg', 'description': 'Participants received VX-864 q12h for 48 weeks or until study drug dosing was terminated.'}, {'id': 'OG001', 'title': 'Group B VX-864 500 mg', 'description': 'Participants undergo a liver biopsy before receiving VX-864 q12h for 48 weeks or until study drug dosing was terminated and undergo a second liver biopsy at either Week 24 or Week 48.'}], 'timeFrame': 'From Baseline up to Week 48', 'reportingStatus': 'POSTED', 'populationDescription': 'Data was not collected for this Outcome Measure as the study drug dosing was terminated prior to any participant reaching Week 48.'}, {'type': 'SECONDARY', 'title': 'Group B: Change in Z-polymer Accumulation in the Liver', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Group A VX-864 500 mg', 'description': 'Participants received VX-864 q12h for 48 weeks or until study drug dosing was terminated.'}, {'id': 'OG001', 'title': 'Group B VX-864 500 mg', 'description': 'Participants undergo a liver biopsy before receiving VX-864 q12h for 48 weeks or until study drug dosing was terminated and undergo a second liver biopsy at either Week 24 or Week 48.'}], 'timeFrame': 'From Baseline up to Week 48', 'reportingStatus': 'POSTED', 'populationDescription': 'No participants in Group B underwent a second liver biopsy at week 24 or week 48 as the study drug dosing was terminated prior to any participant reaching week 24 or week 48. Therefore, data was not collected for this Outcome Measure.'}, {'type': 'SECONDARY', 'title': 'Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Group A VX-864 500 mg', 'description': 'Participants received VX-864 q12h for 48 weeks or until study drug dosing was terminated.'}, {'id': 'OG001', 'title': 'Group B VX-864 500 mg', 'description': 'Participants undergo a liver biopsy before receiving VX-864 q12h for 48 weeks or until study drug dosing was terminated and undergo a second liver biopsy at either Week 24 or Week 48.'}], 'classes': [{'title': 'Participants with TEAEs', 'categories': [{'measurements': [{'value': '10', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}]}, {'title': 'Participants with SAEs', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Day 1 up to Week 52', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety set included all participants who had received at least 1 dose of study drug in this study.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Group A VX-864 500 mg', 'description': 'Participants received VX-864 every 12 hours (q12h) for 48 weeks or until study drug dosing was terminated.'}, {'id': 'FG001', 'title': 'Group B VX-864 500 mg', 'description': 'Participants undergo a liver biopsy before receiving VX-864 q12h for 48 weeks or until study drug dosing was terminated and undergo a second liver biopsy at either Week 24 or Week 48.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '10'}, {'groupId': 'FG001', 'numSubjects': '4'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '9'}, {'groupId': 'FG001', 'numSubjects': '2'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '2'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Withdrawal of consent (not due to AE)', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'This study had 2 Groups: Group A participants did not have a liver biopsy and Group B participants have 2 liver biopsies performed over the course of the study.', 'preAssignmentDetails': 'A total of 14 participants were enrolled from 23 February 2023 to 03 November 2023 in this study. Study drug dosing and efficacy assessments were terminated early due to Sponsor decision, therefore evaluation of efficacy outcome measure was not completed.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '14', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Group A VX-864 500 mg', 'description': 'Participants received VX-864 q12h for 48 weeks or until study drug dosing was terminated.'}, {'id': 'BG001', 'title': 'Group B VX-864 500 mg', 'description': 'Participants undergo a liver biopsy before receiving VX-864 q12h for 48 weeks or until study drug dosing was terminated and undergo a second liver biopsy at either Week 24 or Week 48.'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '52.8', 'spread': '14.2', 'groupId': 'BG000'}, {'value': '50.8', 'spread': '10.4', 'groupId': 'BG001'}, {'value': '52.2', 'spread': '12.9', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '8', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '11', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'calculatePct': False, 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'White', 'categories': [{'measurements': [{'value': '10', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '14', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'calculatePct': False, 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'Not Hispanic or Latino', 'categories': [{'measurements': [{'value': '10', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '14', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'The Full Analysis Set (FAS) is defined as all enrolled participants who have received at least 1 dose of study drug.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2023-11-02', 'size': 2352647, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2025-08-11T08:17', 'hasProtocol': True}, {'date': '2023-11-30', 'size': 646270, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2025-08-11T08:18', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 14}}, 'statusModule': {'whyStopped': "Study drug dosing was permanently discontinued at the Sponsor's discretion based on frequency and nature of skin and subcutaneous tissue disorders.", 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2023-02-23', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-08', 'completionDateStruct': {'date': '2024-08-19', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-08-19', 'studyFirstSubmitDate': '2022-11-29', 'resultsFirstSubmitDate': '2025-08-19', 'studyFirstSubmitQcDate': '2022-11-29', 'lastUpdatePostDateStruct': {'date': '2025-09-08', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2025-08-19', 'studyFirstPostDateStruct': {'date': '2022-12-08', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2025-09-08', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2024-08-19', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change in Blood Functional Alpha-1 Antitrypsin (AAT) Levels', 'timeFrame': 'From Baseline at Week 48'}], 'secondaryOutcomes': [{'measure': 'Change in Blood Functional AAT Levels', 'timeFrame': 'From Baseline up to Week 48'}, {'measure': 'Change in Blood Antigenic AAT Levels', 'timeFrame': 'From Baseline up to Week 48'}, {'measure': 'Change in Blood Z-polymer Levels', 'timeFrame': 'From Baseline up to Week 48'}, {'measure': 'Group B: Change in Z-polymer Accumulation in the Liver', 'timeFrame': 'From Baseline up to Week 48'}, {'measure': 'Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)', 'timeFrame': 'Day 1 up to Week 52'}]}, 'oversightModule': {'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Alpha-1 Antitrypsin Deficiency']}, 'descriptionModule': {'briefSummary': 'The purpose of this study evaluates the efficacy and safety of VX-864 in participants with the PiZZ genotype over 48 weeks.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '80 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n* Participants must have a PiZZ genotype confirmed at screening\n* Plasma AAT levels indicating severe deficiency at screening\n\nKey Exclusion Criteria:\n\n* History of a medical condition that could negatively impact the ability to complete the study\n* Solid organ, or hematological transplantation or is currently on a transplant list\n* History of use of gene therapy or Ribonucleic acid interference (RNAi) therapy at any time previously\n* Participants for whom discontinuation of augmentation therapy is not considered to be in their best interest, based on the clinical judgement of the treating physician\n\nOther protocol defined Inclusion/Exclusion criteria may apply.'}, 'identificationModule': {'nctId': 'NCT05643495', 'briefTitle': 'A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype', 'organization': {'class': 'INDUSTRY', 'fullName': 'Vertex Pharmaceuticals Incorporated'}, 'officialTitle': 'A Phase 2, Open-label Study Evaluating Efficacy and Safety of VX-864 in Subjects With Alpha-1 Antitrypsin Deficiency Who Have the PiZZ Genotype, Over 48 Weeks', 'orgStudyIdInfo': {'id': 'VX22-864-108'}, 'secondaryIdInfos': [{'id': '2022-002746-40', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Group A', 'description': 'Participants received VX-864 every 12 hours (q12h) for 48 weeks or until study drug dosing was terminated.', 'interventionNames': ['Drug: VX-864']}, {'type': 'EXPERIMENTAL', 'label': 'Group B', 'description': 'Participants undergo a liver biopsy before receiving VX-864 q12h for 48 weeks or until study drug dosing was terminated, and undergo a second liver biopsy at either Week 24 or Week 48.', 'interventionNames': ['Drug: VX-864']}], 'interventions': [{'name': 'VX-864', 'type': 'DRUG', 'description': 'Tablets for oral administration.', 'armGroupLabels': ['Group A', 'Group B']}]}, 'contactsLocationsModule': {'locations': [{'zip': '32610', 'city': 'Gainesville', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Florida', 'geoPoint': {'lat': 29.65163, 'lon': -82.32483}}, {'zip': '32803', 'city': 'Orlando', 'state': 'Florida', 'country': 'United States', 'facility': 'Central Florida Pulmonary Group, P.A.', 'geoPoint': {'lat': 28.53834, 'lon': -81.37924}}, {'zip': '52242', 'city': 'Iowa City', 'state': 'Iowa', 'country': 'United States', 'facility': 'The University of Iowa Hospitals and Clinics: Adult Pulmonary Clinic', 'geoPoint': {'lat': 41.66113, 'lon': -91.53017}}, {'zip': '66160', 'city': 'Kansas City', 'state': 'Kansas', 'country': 'United States', 'facility': 'University of Kansas Medical Center', 'geoPoint': {'lat': 39.11417, 'lon': -94.62746}}, {'zip': '63401', 'city': 'Hannibal', 'state': 'Missouri', 'country': 'United States', 'facility': 'Hannibal Regional Healthcare System', 'geoPoint': {'lat': 39.70838, 'lon': -91.35848}}, {'zip': '10032', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Columbia University Irving Medical Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '27517', 'city': 'Chapel Hill', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Marsico Clinical Research Center at UNC Pulmonary Clinic', 'geoPoint': {'lat': 35.9132, 'lon': -79.05584}}, {'zip': '77380', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'Renovatio Clinical', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '84108', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': 'University of Utah Health', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '22042', 'city': 'Falls Church', 'state': 'Virginia', 'country': 'United States', 'facility': 'Inova Fairfax Medical Campus', 'geoPoint': {'lat': 38.88233, 'lon': -77.17109}}, {'city': 'Aachen', 'country': 'Germany', 'facility': 'University Hospital RWTH Aachen', 'geoPoint': {'lat': 50.77664, 'lon': 6.08342}}, {'city': 'Beaumont', 'country': 'Ireland', 'facility': 'Royal College of Surgeons in Ireland Clinical Research Centre, Beaumont Hospital', 'geoPoint': {'lat': 53.38721, 'lon': -6.22713}}, {'city': 'London', 'country': 'United Kingdom', 'facility': "King's College Hospital", 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO', 'description': 'Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Vertex Pharmaceuticals Incorporated', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}