Ignite Creation Date:
2025-12-24 @ 5:37 PM
Ignite Modification Date:
2025-12-29 @ 12:22 PM
Study NCT ID:
NCT04868968
Status:
COMPLETED
Last Update Posted:
2024-10-09
First Post:
2021-04-22
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Study of Safety, Tolerability and Efficacy of DFV890 in Participants With Familial Cold Auto-inflammatory Syndrome (FCAS)
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Italy', 'United Kingdom'], 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2024-04-09', 'type': 'ACTUAL'}}}}, 'conditionBrowseModule': {'meshes': [{'id': 'D056587', 'term': 'Cryopyrin-Associated Periodic Syndromes'}], 'ancestors': [{'id': 'D056660', 'term': 'Hereditary Autoinflammatory Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D012873', 'term': 'Skin Diseases, Genetic'}, {'id': 'D012871', 'term': 'Skin Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D000094482', 'term': 'Chronic Inducible Urticaria'}, {'id': 'D000080223', 'term': 'Chronic Urticaria'}, {'id': 'D014581', 'term': 'Urticaria'}, {'id': 'D017445', 'term': 'Skin Diseases, Vascular'}, {'id': 'D000096703', 'term': 'Cold Urticaria'}, {'id': 'D006969', 'term': 'Hypersensitivity, Immediate'}, {'id': 'D006967', 'term': 'Hypersensitivity'}, {'id': 'D007154', 'term': 'Immune System Diseases'}, {'id': 'D002908', 'term': 'Chronic Disease'}, {'id': 'D020969', 'term': 'Disease Attributes'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'novartis.email@novartis.com', 'phone': '+1 862-778-8300', 'title': 'Study Director', 'organization': 'Novartis Pharmaceuticals'}, 'certainAgreement': {'otherDetails': "The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.", 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatment, up to a maximum duration of approximately 34 days', 'eventGroups': [{'id': 'EG000', 'title': 'DFV890 100 mg - Treatment', 'description': 'DFV890 100mg oral dose, twice daily for 3 days and one last dose in the morning of Day 4 of the treatment period', 'otherNumAtRisk': 4, 'deathsNumAtRisk': 4, 'otherNumAffected': 4, 'seriousNumAtRisk': 4, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Asthenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Chills', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Conjunctivitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 3}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Myalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Cold urticaria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Dermatitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Urticaria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Ratio of Fold Change From Pre-challenge to the Highest Post-challenge Value of White Cell Count (WCC) Between Treatment and Screening Period', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'DFV890 100mg', 'description': 'DFV890 100mg oral dose, twice daily for 3 days and one last dose in the morning of Day 4 of the treatment period'}], 'classes': [{'categories': [{'measurements': [{'value': '0.82', 'groupId': 'OG000', 'lowerLimit': '0.56', 'upperLimit': '1.21'}]}]}], 'paramType': 'GEOMETRIC_LEAST_SQUARES_MEAN', 'timeFrame': 'Screening period and treatment period (Day 4): pre cold challenge and up to 8 hours post cold challenge. The duration of the cold challenge was 45 minutes.', 'description': 'A cold challenge was performed during the screening period and on Day 4 of the treatment period. Fold change from pre-challenge to highest post-challenge value of WCC was defined as the ratio of the highest post-challenge WCC value to the pre-challenge WCC value. The ratio of fold change was defined as treatment fold change divided by the screen fold change. A value of less than 1 for the ratio of fold change indicates a lower relative increase of WCC in the treatment than in the screening period, which is a favorable outcome. The log-transformed fold change from pre-challenge to the highest post challenge WCC was analyzed using a log-linear mixed effect model. The analysis was carried out considering the data from -2 to 8 hrs post challenge. The unforeseen screen failure rate and recruitment challenges resulted in early closure of the study. Only 4 out of planned 6 participants were enrolled in the study; thus, the results should be interpreted cautiously.', 'unitOfMeasure': 'ratio of fold change', 'dispersionType': '90% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'The Pharmacodynamic (PD) analysis set included all participants with no protocol deviations with relevant impact on PD data.'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'DFV890 100mg - Treatment', 'description': 'DFV890 100mg oral dose, twice daily for 3 days and one last dose in the morning of Day 4 of the treatment period'}], 'classes': [{'title': 'At least one AE', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'At least one SAE', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'AE leading to discontinuation', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'SAE leading to discontinuation', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatment, up to a maximum duration of approximately 34 days', 'description': 'Number of participants with treatment emergent AEs (any AE regardless of seriousness), AEs led to study treatment discontinuation, SAEs and SAEs led to study treatment discontinuation.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The safety analysis set included all participants that received any study drug.'}, {'type': 'SECONDARY', 'title': 'Physician Global Assessment of Autoinflammatory Disease Activity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Screening', 'description': 'Screening period'}, {'id': 'OG001', 'title': 'DFV890 100mg - Treatment', 'description': 'DFV890 100mg oral dose, twice daily for 3 days and one last dose in the morning of Day 4 of the treatment period'}], 'classes': [{'title': '1 hour pre', 'categories': [{'title': 'Absent', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': '2 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': '3 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': '5 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': '9 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': '24 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Screening and Treatment (Day 4): 1 hour pre and 2, 3, 5, 9 and 24 hours post. Scheduled time refers to the time post-meal (screening) and to the time post-dose (treatment). The start of the cold challenge is at 1 hour post and the duration is 45 minutes.', 'description': 'The Physician global assessment of autoinflammatory disease activity is a questionnaire completed by the Investigator. It uses a 5-point scale. Lower scores represent better outcomes.\n\n0 = Absent\n\n1. = Minimal\n2. = Mild\n3. = Moderate\n4. = Severe', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The Pharmacodynamic (PD) analysis set included all participants with no protocol deviations with relevant impact on PD data.'}, {'type': 'SECONDARY', 'title': "Physician's Severity Assessment of Autoinflammatory Disease Signs and Symptoms", 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Screening', 'description': 'Screening period'}, {'id': 'OG001', 'title': 'DFV890 100mg - Treatment', 'description': 'DFV890 100mg oral dose, twice daily for 3 days and one last dose in the morning of Day 4 of the treatment period'}], 'classes': [{'title': 'Arthralgia 1 hour pre', 'categories': [{'title': 'Absent', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Arthralgia 2 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Arthralgia 3 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Arthralgia 5 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Arthralgia 9 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Arthralgia 24 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '4', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Conjunctivitis 1 hour pre', 'categories': [{'title': 'Absent', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Conjunctivitis 2 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Conjunctivitis 3 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Conjunctivitis 5 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Conjunctivitis 9 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Conjunctivitis 24 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Fatigue/Malaise 1 hour pre', 'categories': [{'title': 'Absent', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Fatigue/Malaise 2 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Fatigue/Malaise 3 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Fatigue/Malaise 5 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Fatigue/Malaise 9 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Fatigue/Malaise 24 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Headache/Migraine 1 hour pre', 'categories': [{'title': 'Absent', 'measurements': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Headache/Migraine 2 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Headache/Migraine 3 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Headache/Migraine 5 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Headache/Migraine 9 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Headache/Migraine 24 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Myalgia 1 hour pre', 'categories': [{'title': 'Absent', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Myalgia 2 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '4', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Myalgia 3 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Myalgia 5 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Myalgia 9 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Myalgia 24 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Skin disease 1 hour pre', 'categories': [{'title': 'Absent', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Skin disease 2 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Skin disease 3 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Skin disease 5 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Skin disease 9 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Skin disease 24 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Screening and Treatment (Day 4): 1 hour pre and 2, 3, 5, 9 and 24 hours post. Scheduled time refers to the time post-meal (screening) and to the time post-dose (treatment). The start of the cold challenge is at 1 hour post and the duration is 45 minutes.', 'description': "The Physician's severity assessment of autoinflammatory disease signs and symptoms is a questionnaire completed by the Investigator. It uses a 5-point scale. Lower scores represent better outcomes.\n\n0 = Absent\n\n1. = Minimal\n2. = Mild\n3. = Moderate\n4. = Severe\n\nThe following items were assessed:\n\n* Assessment of skin disease (urticarial skin rash)\n* Assessment of arthralgia\n* Assessment of myalgia\n* Assessment of headache/migraine\n* Assessment of conjunctivitis\n* Assessment of fatigue/malaise", 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The Pharmacodynamic (PD) analysis set included all participants with no protocol deviations with relevant impact on PD data.'}, {'type': 'SECONDARY', 'title': "Patient's Global Assessment of Disease Activity", 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Screening', 'description': 'Screening period'}, {'id': 'OG001', 'title': 'DFV890 100mg - Treatment', 'description': 'DFV890 100mg oral dose, twice daily for 3 days and one last dose in the morning of Day 4 of the treatment period'}], 'classes': [{'title': '1 hour pre', 'categories': [{'title': 'Absent', 'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': '2 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '4', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': '3 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': '5 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': '9 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': '24 hours post', 'categories': [{'title': 'Absent', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}, {'title': 'Minimal', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}, {'title': 'Mild', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Moderate', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}, {'title': 'Severe', 'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Screening and Treatment (Day 4): 1 hour pre and 2, 3, 5, 9 and 24 hours post. Scheduled time refers to the time post-meal (screening) and to the time post-dose (treatment). The start of the cold challenge is at 1 hour post and the duration is 45 minutes.', 'description': "Patient's global assessment of disease activity is a questionnaire completed by the patient. It uses a 5-point scale. The patient selected a rating based on the patient's current disease activity at the time of the assessment. Lower scores represent better outcomes.\n\n0 = Absent\n\n1. = Minimal\n2. = Mild\n3. = Moderate\n4. = Severe", 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The Pharmacodynamic (PD) analysis set included all participants with no protocol deviations with relevant impact on PD data.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'DFV890 100mg - Treatment', 'description': 'DFV890 100mg oral dose, twice daily for 3 days and one last dose in the morning of Day 4 of the treatment period'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'comment': 'All participants enrolled in the trial', 'achievements': [{'groupId': 'FG000', 'numSubjects': '4'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'Participants took part in 3 investigative sites in 3 countries.', 'preAssignmentDetails': "During the screening period the participant's eligibility was assessed at a screening visit and a screening cold challenge was performed."}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'DFV890 100mg - Treatment', 'description': 'DFV890 100mg oral dose, twice daily for 3 days and one last dose in the morning of Day 4 of the treatment period'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '41.3', 'spread': '15.48', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'other', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}, {'title': 'white', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2022-05-19', 'size': 1031922, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2024-03-11T11:13', 'hasProtocol': True}, {'date': '2023-07-17', 'size': 599126, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2024-03-11T11:13', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 4}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2021-09-20', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-10', 'dispFirstSubmitDate': '2023-05-18', 'completionDateStruct': {'date': '2023-05-05', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-10-07', 'studyFirstSubmitDate': '2021-04-22', 'resultsFirstSubmitDate': '2024-03-11', 'studyFirstSubmitQcDate': '2021-04-28', 'dispFirstPostDateStruct': {'date': '2024-05-03', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2024-10-09', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2024-05-02', 'studyFirstPostDateStruct': {'date': '2021-05-03', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2024-05-03', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-12-13', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Ratio of Fold Change From Pre-challenge to the Highest Post-challenge Value of White Cell Count (WCC) Between Treatment and Screening Period', 'timeFrame': 'Screening period and treatment period (Day 4): pre cold challenge and up to 8 hours post cold challenge. The duration of the cold challenge was 45 minutes.', 'description': 'A cold challenge was performed during the screening period and on Day 4 of the treatment period. Fold change from pre-challenge to highest post-challenge value of WCC was defined as the ratio of the highest post-challenge WCC value to the pre-challenge WCC value. The ratio of fold change was defined as treatment fold change divided by the screen fold change. A value of less than 1 for the ratio of fold change indicates a lower relative increase of WCC in the treatment than in the screening period, which is a favorable outcome. The log-transformed fold change from pre-challenge to the highest post challenge WCC was analyzed using a log-linear mixed effect model. The analysis was carried out considering the data from -2 to 8 hrs post challenge. The unforeseen screen failure rate and recruitment challenges resulted in early closure of the study. Only 4 out of planned 6 participants were enrolled in the study; thus, the results should be interpreted cautiously.'}], 'secondaryOutcomes': [{'measure': 'Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)', 'timeFrame': 'Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatment, up to a maximum duration of approximately 34 days', 'description': 'Number of participants with treatment emergent AEs (any AE regardless of seriousness), AEs led to study treatment discontinuation, SAEs and SAEs led to study treatment discontinuation.'}, {'measure': 'Physician Global Assessment of Autoinflammatory Disease Activity', 'timeFrame': 'Screening and Treatment (Day 4): 1 hour pre and 2, 3, 5, 9 and 24 hours post. Scheduled time refers to the time post-meal (screening) and to the time post-dose (treatment). The start of the cold challenge is at 1 hour post and the duration is 45 minutes.', 'description': 'The Physician global assessment of autoinflammatory disease activity is a questionnaire completed by the Investigator. It uses a 5-point scale. Lower scores represent better outcomes.\n\n0 = Absent\n\n1. = Minimal\n2. = Mild\n3. = Moderate\n4. = Severe'}, {'measure': "Physician's Severity Assessment of Autoinflammatory Disease Signs and Symptoms", 'timeFrame': 'Screening and Treatment (Day 4): 1 hour pre and 2, 3, 5, 9 and 24 hours post. Scheduled time refers to the time post-meal (screening) and to the time post-dose (treatment). The start of the cold challenge is at 1 hour post and the duration is 45 minutes.', 'description': "The Physician's severity assessment of autoinflammatory disease signs and symptoms is a questionnaire completed by the Investigator. It uses a 5-point scale. Lower scores represent better outcomes.\n\n0 = Absent\n\n1. = Minimal\n2. = Mild\n3. = Moderate\n4. = Severe\n\nThe following items were assessed:\n\n* Assessment of skin disease (urticarial skin rash)\n* Assessment of arthralgia\n* Assessment of myalgia\n* Assessment of headache/migraine\n* Assessment of conjunctivitis\n* Assessment of fatigue/malaise"}, {'measure': "Patient's Global Assessment of Disease Activity", 'timeFrame': 'Screening and Treatment (Day 4): 1 hour pre and 2, 3, 5, 9 and 24 hours post. Scheduled time refers to the time post-meal (screening) and to the time post-dose (treatment). The start of the cold challenge is at 1 hour post and the duration is 45 minutes.', 'description': "Patient's global assessment of disease activity is a questionnaire completed by the patient. It uses a 5-point scale. The patient selected a rating based on the patient's current disease activity at the time of the assessment. Lower scores represent better outcomes.\n\n0 = Absent\n\n1. = Minimal\n2. = Mild\n3. = Moderate\n4. = Severe"}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Familial Cold Auto-inflammatory Syndrome', 'FCAS', 'DFV890', 'NLRP3 inhibitor, inflammasome inhibition', 'Cryopyrin-associated periodic syndromes', 'CAPS'], 'conditions': ['Familial Cold Autoinflammatory Syndrome']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'https://www.novctrd.com/ctrdweb/patientsummary/patientsummaries?patientSummaryId=1980', 'label': 'A Plain Language Trial Summary is available on www.novctrd.com'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this phase II study was to assess the safety, tolerability and efficacy of DFV890 in participants with FCAS.', 'detailedDescription': 'This was an open-label, single-arm, multiple dose, phase II study to assess safety, tolerability and clinical efficacy of DFV890 in participants with FCAS who showed evidence of inflammatory activity after the cold challenge performed during screening.\n\nThe study included a screening period, a treatment period and a follow-up period.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '80 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Written informed consent must be obtained before any study-specific assessment is performed\n* Body mass index within the range of 18-35 kg/m2\n* Patients with a genetic diagnosis of FCAS\n* Patients with a clinical history and investigations consistent with FCAS\n\nExclusion Criteria:\n\n* Anti-rejection and/or immunomodulatory drugs must be discontinued (please, see protocol for further details)\n* Clinically significant, suspected active or chronic bacterial (including Mycobacterium tuberculosis), viral or fungal infection within 30 days prior to Day 1.\n* Patients with innate (e.g. TLR immunodeficiencies, defects in IFN-γ signaling) or acquired immune deficiencies (e.g. AIDS).\n* Presence of human immunodeficiency virus (HIV) infection, hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (anti-HBc), or hepatitis C antibodies at screening.\n* Live vaccines within 4 weeks of Day 1\n* Pregnant or nursing (lactating) women.\n* Women of child-bearing potential unless they are using highly effective methods of contraception.\n\nOther protocol-defined inclusion/exclusion criteria may apply.'}, 'identificationModule': {'nctId': 'NCT04868968', 'acronym': 'DFV890-FCAS', 'briefTitle': 'Study of Safety, Tolerability and Efficacy of DFV890 in Participants With Familial Cold Auto-inflammatory Syndrome (FCAS)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Novartis'}, 'officialTitle': 'An Open-label, Single Arm Phase II Study of DFV890 to Assess the Safety, Tolerability and Efficacy in Participants With Familial Cold Auto-inflammatory Syndrome (FCAS)', 'orgStudyIdInfo': {'id': 'CDFV890A12201'}, 'secondaryIdInfos': [{'id': '2020-005948-33', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'DFV890', 'description': 'DFV890', 'interventionNames': ['Drug: DFV890']}], 'interventions': [{'name': 'DFV890', 'type': 'DRUG', 'description': '100 mg of DFV890 film coated tablets twice daily (b.i.d.). for 3 days starting in the morning of Day 1 and 100 mg of DFV890 in the morning on the fourth day.', 'armGroupLabels': ['DFV890']}]}, 'contactsLocationsModule': {'locations': [{'zip': '92093', 'city': 'La Jolla', 'state': 'California', 'country': 'United States', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 32.84727, 'lon': -117.2742}}, {'zip': '75970', 'city': 'Paris', 'country': 'France', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '72076', 'city': 'Tübingen', 'country': 'Germany', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 48.52266, 'lon': 9.05222}}], 'overallOfficials': [{'name': 'Novartis Pharmaceuticals', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Novartis Pharmaceuticals'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'YES', 'description': 'Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.\n\nThis trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Novartis Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}