Ignite Creation Date:
2025-12-25 @ 3:37 AM
Ignite Modification Date:
2026-01-07 @ 6:29 AM
Study NCT ID:
NCT03053102
Status:
COMPLETED
Last Update Posted:
2022-06-23
First Post:
2017-02-01
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006457', 'term': 'Hemoglobinuria, Paroxysmal'}, {'id': 'D006456', 'term': 'Hemoglobinuria'}], 'ancestors': [{'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D009190', 'term': 'Myelodysplastic Syndromes'}, {'id': 'D001855', 'term': 'Bone Marrow Diseases'}, {'id': 'D011507', 'term': 'Proteinuria'}, {'id': 'D014555', 'term': 'Urination Disorders'}, {'id': 'D014570', 'term': 'Urologic Diseases'}, {'id': 'D052776', 'term': 'Female Urogenital Diseases'}, {'id': 'D005261', 'term': 'Female Urogenital Diseases and Pregnancy Complications'}, {'id': 'D000091642', 'term': 'Urogenital Diseases'}, {'id': 'D052801', 'term': 'Male Urogenital Diseases'}, {'id': 'D020924', 'term': 'Urological Manifestations'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000718467', 'term': 'danicopan'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'clinicaltrials@alexion.com', 'phone': '855-752-2356', 'title': 'Alexion Pharmaceuticals Inc.', 'organization': 'Alexion Pharmaceuticals Inc.'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'After the first dose of study medication (Day 1) through 14 days after the last dose of study drug (up to Day 104).', 'description': '\\[Not specified\\]', 'eventGroups': [{'id': 'EG000', 'title': 'Danicopan', 'description': 'Participants received danicopan 100 to 200 mg TID.', 'otherNumAtRisk': 10, 'deathsNumAtRisk': 10, 'otherNumAffected': 9, 'seriousNumAtRisk': 10, 'deathsNumAffected': 0, 'seriousNumAffected': 1}], 'otherEvents': [{'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Non-cardiac chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 4}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Viral upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Myalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Irritability', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Iron deficiency', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Rash papular', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Mouth ulceration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Oedema peripheral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Vaccination site erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Paroxysmal nocturnal haemoglobinuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Dysmenorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Haemoglobinuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 2}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Haemolysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 2}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Pharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}], 'seriousEvents': [{'term': 'Haemolysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Aspartate aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change From Baseline In Serum LDH Levels At Day 28', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants received danicopan 100 to 200 mg TID.'}], 'classes': [{'title': 'Baseline', 'categories': [{'measurements': [{'value': '1416', 'spread': '540.25', 'groupId': 'OG000'}]}]}, {'title': 'Day 28 (Part 1)', 'categories': [{'measurements': [{'value': '444.3', 'spread': '255.78', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline', 'categories': [{'measurements': [{'value': '-971.7', 'spread': '549.76', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 28', 'description': 'Change from Baseline = Serum LDH levels on Day 28 - Baseline Serum LDH levels.', 'unitOfMeasure': 'international units per liter (IU/L)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of study drug and had analyzable data at the timepoints specified.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline In Hemoglobin (Hgb) At Days 28 And 84', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants received danicopan 100 to 200 mg TID.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '9.76', 'spread': '1.758', 'groupId': 'OG000'}]}]}, {'title': 'Part 1: Day 28', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '10.94', 'spread': '1.651', 'groupId': 'OG000'}]}]}, {'title': 'Part 1: Change from Baseline at Day 28', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.18', 'spread': '0.877', 'groupId': 'OG000'}]}]}, {'title': 'Part 2: Day 84', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '11.45', 'spread': '1.414', 'groupId': 'OG000'}]}]}, {'title': 'Part 2: Change from Baseline at Day 84', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.80', 'spread': '1.166', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Days 28 and 84', 'description': 'Change from Baseline = Hgb levels on Days 28 or 84 - Baseline Hgb levels.', 'unitOfMeasure': 'grams/deciliter (g/dL)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of study drug and had analyzable data at the timepoints specified.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline In Serum LDH Levels At Day 84', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants received danicopan 100 to 200 mg TID.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1416', 'spread': '540.25', 'groupId': 'OG000'}]}]}, {'title': 'Day 84 (Part 2)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '537.3', 'spread': '260.42', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-865.9', 'spread': '447.86', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 84', 'description': 'Change from Baseline = Serum LDH levels on Day 84 - Baseline Serum LDH levels.', 'unitOfMeasure': 'IU/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of study drug and had analyzable data at the timepoints specified.'}, {'type': 'SECONDARY', 'title': 'Paroxysmal Nocturnal Hemoglobinuria (PNH) Type III Red Blood Cell (RBC) Clone Size', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants received danicopan 100 to 200 mg TID.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '31.5363', 'spread': '24.62004', 'groupId': 'OG000'}]}]}, {'title': 'Day 28', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '43.6279', 'spread': '15.58021', 'groupId': 'OG000'}]}]}, {'title': 'Day 84', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '56.2953', 'spread': '19.85086', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 28, and Day 84', 'description': 'PNH RBC, summed type III, clone size levels were assessed from Baseline to Day 28 and Day 84. The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population.', 'unitOfMeasure': 'percentage of the total cell population', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of study drug and had analyzable data at the timepoints specified.'}, {'type': 'SECONDARY', 'title': 'Serious Adverse Events (SAEs), Grade 3 And Grade 4 Treatment-emergent Adverse Events (TEAEs), And Adverse Events (AEs) Leading To Discontinuation', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants received danicopan 100 to 200 mg TID.'}], 'classes': [{'title': 'SAE', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'TEAE Grade 3', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'TEAE Grade 4', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'AE leading to discontinuation', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'After the first dose of study medication (Day 1) through 14 days after the last dose of study drug (up to Day 104)', 'description': 'An AE was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. The intensity of an AE was graded according to the Common Terminology Criteria for Adverse Events (CTCAE) Adverse Event Severity Grading Table. A summary of SAEs and other non-serious AEs regardless of causality is located in the Reported Adverse Events module.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of study drug.'}, {'type': 'SECONDARY', 'title': 'Grade 3 And Grade 4 Laboratory Abnormalities', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants received danicopan 100 to 200 mg TID.'}], 'classes': [{'title': 'Alanine aminotransferase increased', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Aspartate aminotransferase increased', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Low hemoglobin', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Low neutrophil count', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'High triglycerides', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'After the first dose of study medication (Day 1) through 14 days after the last dose of study drug (up to Day 104).', 'description': 'Laboratory abnormalities were determined from laboratory measurements analyzed at the central or local laboratories, and were graded using CTCAE.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of study drug.'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetics (PK): Area Under The Plasma Concentration-time Curve From Time Of Administration To 8 Hours Post-dose (AUC0-8)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants received danicopan 100 to 200 mg TID.'}], 'classes': [{'title': 'Day 6: 100 mg TID', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1432.611', 'spread': '19.41', 'groupId': 'OG000'}]}]}, {'title': 'Day 20: 150 mg TID', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2370.421', 'spread': '14.92', 'groupId': 'OG000'}]}]}, {'title': 'Day 20: 175 mg TID', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2278.038', 'spread': '37.30', 'groupId': 'OG000'}]}]}], 'paramType': 'GEOMETRIC_MEAN', 'timeFrame': 'Days 6 and 20', 'description': 'Serial blood samples were collected predose and up to 8 hours postdose.', 'unitOfMeasure': 'hour (hr)*nanograms/milliliter (ng/mL)', 'dispersionType': 'Geometric Coefficient of Variation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of study drug and had analyzable data at the timepoints specified. PK analysis was not conducted on the 200 mg dose.'}, {'type': 'SECONDARY', 'title': 'PK: Maximum Plasma Concentration (Cmax)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants received danicopan 100 to 200 mg TID.'}], 'classes': [{'title': 'Day 6: 100 mg TID', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '347.707', 'spread': '9.57', 'groupId': 'OG000'}]}]}, {'title': 'Day 20: 150 mg TID', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '583.626', 'spread': '27.29', 'groupId': 'OG000'}]}]}, {'title': 'Day 20: 175 mg TID', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '516.8788', 'spread': '33.39', 'groupId': 'OG000'}]}]}], 'paramType': 'GEOMETRIC_MEAN', 'timeFrame': 'Days 6 and 20', 'description': 'Serial blood samples were collected predose and up to 12 hours postdose.', 'unitOfMeasure': 'ng/mL', 'dispersionType': 'Geometric Coefficient of Variation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of study drug and had analyzable data at the timepoints specified. PK analysis was not conducted on the 200 mg dose.'}, {'type': 'SECONDARY', 'title': 'PK: Time To Maximum Concentration (Tmax)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants who received danicopan 100 to 200 mg TID.'}], 'classes': [{'title': 'Day 6: 100 mg TID', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.17', 'groupId': 'OG000', 'lowerLimit': '3.67', 'upperLimit': '4.67'}]}]}, {'title': 'Day 20: 150 mg TID', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.67', 'groupId': 'OG000', 'lowerLimit': '1.05', 'upperLimit': '4.67'}]}]}, {'title': 'Day 20: 175 mg TID', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.11', 'groupId': 'OG000', 'lowerLimit': '1.67', 'upperLimit': '4.65'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Days 6 and 20', 'description': 'Serial blood samples were collected predose and up to 12 hours postdose.', 'unitOfMeasure': 'hr', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of study drug and had analyzable data at the timepoints specified. PK analysis was not conducted on the 200 mg dose.'}, {'type': 'SECONDARY', 'title': 'Complement Alternative Pathway (AP) Functional Activity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants received danicopan 100 to 200 mg TID.'}], 'classes': [{'title': 'Baseline', 'categories': [{'measurements': [{'value': '65.216', 'spread': '13.7143', 'groupId': 'OG000'}]}]}, {'title': 'Day 28', 'categories': [{'measurements': [{'value': '12.730', 'spread': '14.37670', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Day 28', 'description': 'Serum AP functional activity was measured by the Wieslab functional immunoassay method.', 'unitOfMeasure': 'percentage of activity', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of study drug and had analyzable data at the timepoints specified.'}, {'type': 'SECONDARY', 'title': 'Complement Bb', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants received danicopan 100 to 200 mg TID.'}], 'classes': [{'title': 'Baseline', 'categories': [{'measurements': [{'value': '2.24160', 'spread': '0.773962', 'groupId': 'OG000'}]}]}, {'title': 'Day 28', 'categories': [{'measurements': [{'value': '0.83913', 'spread': '0.838219', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Day 28', 'description': 'Plasma Bb was measured by enzyme-linked immunosorbent assay (ELISA).', 'unitOfMeasure': 'microgram (ug)/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of study drug and had analyzable data at the timepoints specified.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Danicopan', 'description': 'Participants received danicopan 100 to 200 mg TID.'}], 'periods': [{'title': 'Study Part 1: 28-Day Treatment', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '10'}]}, {'type': 'Received at Least 1 Dose of Study Drug', 'achievements': [{'groupId': 'FG000', 'numSubjects': '10'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '10'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}, {'title': 'Study Part 2: 84-Day Treatment', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '10'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}], 'preAssignmentDetails': 'The study was conducted in 2 parts. In Part 1, participants received danicopan for 28 days. Starting doses were 100 or 150 milligrams (mg) three times daily (TID). A further dose increase up to 175 mg TID (N=8 participants) and 200 mg TID (N=4 participants) was conducted. Participants with reductions in lactate dehydrogenase (LDH) meeting specified criteria were offered continued dosing beyond Day 28, for up to 8 additional weeks (Part 2).'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Danicopan', 'description': 'Participants received danicopan 100 to 200 mg TID.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '35.94', 'spread': '13.575', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '10', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'White', 'categories': [{'measurements': [{'value': '7', 'groupId': 'BG000'}]}]}, {'title': 'Asian', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}, {'title': 'Other', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'New Zealand', 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}]}]}, {'title': 'South Korea', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}, {'title': 'United Kingdom', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}, {'title': 'Italy', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}], 'populationDescription': 'All participants who were enrolled in the study and received at least 1 dose of study drug.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2018-03-13', 'size': 1180025, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2021-05-06T22:29', 'hasProtocol': True}, {'date': '2018-12-06', 'size': 915852, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2021-05-07T08:54', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 10}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2017-03-31', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-05', 'dispFirstSubmitDate': '2019-08-19', 'completionDateStruct': {'date': '2018-11-14', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2022-05-31', 'studyFirstSubmitDate': '2017-02-01', 'dispFirstSubmitQcDate': '2019-08-20', 'resultsFirstSubmitDate': '2021-05-07', 'studyFirstSubmitQcDate': '2017-02-10', 'dispFirstPostDateStruct': {'date': '2019-08-28', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2022-06-23', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2021-05-07', 'studyFirstPostDateStruct': {'date': '2017-02-14', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2021-06-02', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2018-11-14', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change From Baseline In Serum LDH Levels At Day 28', 'timeFrame': 'Baseline, Day 28', 'description': 'Change from Baseline = Serum LDH levels on Day 28 - Baseline Serum LDH levels.'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline In Hemoglobin (Hgb) At Days 28 And 84', 'timeFrame': 'Baseline, Days 28 and 84', 'description': 'Change from Baseline = Hgb levels on Days 28 or 84 - Baseline Hgb levels.'}, {'measure': 'Change From Baseline In Serum LDH Levels At Day 84', 'timeFrame': 'Baseline, Day 84', 'description': 'Change from Baseline = Serum LDH levels on Day 84 - Baseline Serum LDH levels.'}, {'measure': 'Paroxysmal Nocturnal Hemoglobinuria (PNH) Type III Red Blood Cell (RBC) Clone Size', 'timeFrame': 'Baseline, Day 28, and Day 84', 'description': 'PNH RBC, summed type III, clone size levels were assessed from Baseline to Day 28 and Day 84. The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population.'}, {'measure': 'Serious Adverse Events (SAEs), Grade 3 And Grade 4 Treatment-emergent Adverse Events (TEAEs), And Adverse Events (AEs) Leading To Discontinuation', 'timeFrame': 'After the first dose of study medication (Day 1) through 14 days after the last dose of study drug (up to Day 104)', 'description': 'An AE was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. The intensity of an AE was graded according to the Common Terminology Criteria for Adverse Events (CTCAE) Adverse Event Severity Grading Table. A summary of SAEs and other non-serious AEs regardless of causality is located in the Reported Adverse Events module.'}, {'measure': 'Grade 3 And Grade 4 Laboratory Abnormalities', 'timeFrame': 'After the first dose of study medication (Day 1) through 14 days after the last dose of study drug (up to Day 104).', 'description': 'Laboratory abnormalities were determined from laboratory measurements analyzed at the central or local laboratories, and were graded using CTCAE.'}, {'measure': 'Pharmacokinetics (PK): Area Under The Plasma Concentration-time Curve From Time Of Administration To 8 Hours Post-dose (AUC0-8)', 'timeFrame': 'Days 6 and 20', 'description': 'Serial blood samples were collected predose and up to 8 hours postdose.'}, {'measure': 'PK: Maximum Plasma Concentration (Cmax)', 'timeFrame': 'Days 6 and 20', 'description': 'Serial blood samples were collected predose and up to 12 hours postdose.'}, {'measure': 'PK: Time To Maximum Concentration (Tmax)', 'timeFrame': 'Days 6 and 20', 'description': 'Serial blood samples were collected predose and up to 12 hours postdose.'}, {'measure': 'Complement Alternative Pathway (AP) Functional Activity', 'timeFrame': 'Baseline and Day 28', 'description': 'Serum AP functional activity was measured by the Wieslab functional immunoassay method.'}, {'measure': 'Complement Bb', 'timeFrame': 'Baseline and Day 28', 'description': 'Plasma Bb was measured by enzyme-linked immunosorbent assay (ELISA).'}]}, 'oversightModule': {'isUsExport': True, 'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['PNH', 'Paroxysmal', 'Hemoglobinuria'], 'conditions': ['Paroxysmal Nocturnal Hemoglobinuria (PNH)']}, 'referencesModule': {'references': [{'pmid': '33121236', 'type': 'RESULT', 'citation': 'Risitano AM, Kulasekararaj AG, Lee JW, Maciejewski JP, Notaro R, Brodsky R, Huang M, Geffner M, Browett P. Danicopan: an oral complement factor D inhibitor for paroxysmal nocturnal hemoglobinuria. Haematologica. 2021 Dec 1;106(12):3188-3197. doi: 10.3324/haematol.2020.261826.'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study was to determine the safety and efficacy of ACH-0144471 (also known as danicopan and ALXN2040) in currently untreated participants with PNH.', 'detailedDescription': 'After 12 weeks of treatment, participants deriving clinical benefit were offered enrollment in a separate long-term extension study (ACH471-103, NCT03181633).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Currently untreated PNH participants with PNH Type III erythrocyte and/or granulocyte clone size ≥10% and anemia (hemoglobin \\<12 grams/deciliter) with adequate reticulocytosis (as determined by the Investigator).\n* LDH ≥1.5 x the upper limit of normal.\n* Platelets ≥50,000/microliter without the need for platelet transfusions.\n* Documentation of vaccination for Neisseria meningitidis, Haemophilus influenza, and Streptococcus pneumoniae, or willingness to receive vaccinations during the screening period.\n* Negative pregnancy test for females prior to dosing and throughout the study.\n\nExclusion Criteria:\n\n* History of a major organ transplant (for example, heart, lung, kidney, liver) or hematopoietic stem cell/marrow transplant.\n* Participants who had received another investigational agent within 30 days or 5 half-lives of the investigational agent prior to study entry, whichever is greater.\n* Participants who had received eculizumab at any dose or interval within the past 75 days before study entry.\n* Participants with known or suspected complement deficiency.\n* Participants with active bacterial infection or clinically significant active viral infection, a body temperature \\>38°Celsius, or other evidence of infection on Day 1, or with a history of febrile illness within 14 days prior to first study drug administration.\n* History of meningococcal infection, or a first-degree relative or household contact with a history of meningococcal infection.\n* Females who were pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration or participants with a female partner who was pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration.'}, 'identificationModule': {'nctId': 'NCT03053102', 'briefTitle': 'Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Alexion Pharmaceuticals, Inc.'}, 'officialTitle': 'A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of ACH-0144471 in Untreated Patients With Paroxysmal Nocturnal Hemoglobinuria', 'orgStudyIdInfo': {'id': 'ACH471-100'}, 'secondaryIdInfos': [{'id': '2016-002652-25', 'type': 'EUDRACT_NUMBER'}, {'id': 'U1111-1190-3490', 'type': 'OTHER', 'domain': 'UTN'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Danicopan', 'description': 'Starting doses of danicopan ranged from 100 to 150 milligrams (mg) three times daily (TID), with subsequent dose escalation up to 200 mg TID based on response (clinical and biochemical) for 28 days (Part 1). Participants with reductions in lactate dehydrogenase (LDH) meeting specified criteria were offered continued dosing beyond Day 28, for up to 8 additional weeks (Part 2).', 'interventionNames': ['Drug: Danicopan']}], 'interventions': [{'name': 'Danicopan', 'type': 'DRUG', 'otherNames': ['ACH-0144471', 'ACH-4471', 'ACH4471', '4471', 'ALXN2040'], 'description': 'Danicopan was administered as multiple oral doses over a period of at least 28 days.', 'armGroupLabels': ['Danicopan']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Florence', 'country': 'Italy', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 43.77925, 'lon': 11.24626}}, {'city': 'Naples', 'country': 'Italy', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 40.85216, 'lon': 14.26811}}, {'city': 'Auckland', 'country': 'New Zealand', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': -36.84853, 'lon': 174.76349}}, {'city': 'Seoul', 'country': 'South Korea', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}, {'city': 'London', 'country': 'United Kingdom', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Alexion Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Achillion, a wholly owned subsidiary of Alexion', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}