Ignite Creation Date:
2025-12-25 @ 3:03 AM
Ignite Modification Date:
2026-02-26 @ 12:42 AM
Study NCT ID:
NCT03181633
Status:
COMPLETED
Last Update Posted:
2023-03-14
First Post:
2017-06-05
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2023-01-23', 'type': 'ACTUAL'}}}}, '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': 'Baseline up to 4.5 years', 'description': 'Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.', 'eventGroups': [{'id': 'EG000', 'title': 'Danicopan', 'description': 'Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \\[mg\\] 3 times daily \\[TID\\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.', 'otherNumAtRisk': 8, 'deathsNumAtRisk': 8, 'otherNumAffected': 8, 'seriousNumAtRisk': 8, 'deathsNumAffected': 0, 'seriousNumAffected': 3}], 'otherEvents': [{'term': 'Dysphagia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Gastrooesophageal reflux disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Non-cardiac chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Chest discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Influenza like illness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Cellulitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Herpes simplex', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 3}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Lethargy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Paraesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Haemoglobinuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 2}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Chromaturia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Haematuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Paroxysmal nocturnal haemoglobinuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Accidental overdose', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Intentional overdose', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Ligament sprain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Skin laceration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Haemolysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 2}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Myalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Limb discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Muscle spasms', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Dry throat', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Hypothyroidism', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Endocrine disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Conjunctival hyperaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Uterine leiomyoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Hand dermatitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}], 'seriousEvents': [{'term': 'Cystitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Infectious mononucleosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Haemolysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change From Baseline in LDH Level at Week 25', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \\[mg\\] 3 times daily \\[TID\\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.'}], 'classes': [{'categories': [{'measurements': [{'value': '-683.29', 'spread': '845.175', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 25', 'description': 'Change from Baseline = Serum LDH levels at Week 25 - Baseline Serum LDH levels. Baseline was the baseline value from the primary Study ACH471-100.', 'unitOfMeasure': 'U/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the post baseline assessments for LDH level."}, {'type': 'PRIMARY', 'title': 'Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Week 25', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \\[mg\\] 3 times daily \\[TID\\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.'}], 'classes': [{'categories': [{'measurements': [{'value': '24.67', 'spread': '18.052', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 25', 'description': 'Change from Baseline = Hgb levels at Week 25 - Baseline Hgb levels. Baseline was the baseline value from the primary Study ACH471-100.', 'unitOfMeasure': 'g/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for Hgb level."}, {'type': 'PRIMARY', 'title': 'Change From Baseline in Reticulocyte Counts at Week 25', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \\[mg\\] 3 times daily \\[TID\\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.07', 'spread': '0.063', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 25', 'description': 'Change from Baseline = reticulocyte count at Week 25 - Baseline reticulocyte count. Baseline was the baseline value from the primary Study ACH471-100.', 'unitOfMeasure': '10^12 cells/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for reticulocyte count."}, {'type': 'PRIMARY', 'title': 'Number of RBC Units Transfused', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \\[mg\\] 3 times daily \\[TID\\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.'}], 'classes': [{'categories': [{'measurements': [{'value': '6.5', 'spread': '16.83', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline up to Week 169', 'unitOfMeasure': 'RBC units', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full analysis set included all enrolled and treated participants.'}, {'type': 'PRIMARY', 'title': 'Number of RBC Transfusion Instances', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \\[mg\\] 3 times daily \\[TID\\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.'}], 'classes': [{'categories': [{'measurements': [{'value': '3.4', 'spread': '8.00', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline up to Week 169', 'unitOfMeasure': 'RBC transfusion instances', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full analysis set included all enrolled and treated participants.'}, {'type': 'PRIMARY', 'title': 'Change From Baseline in PNH Clone Size at Week 25', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \\[mg\\] 3 times daily \\[TID\\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.'}], 'classes': [{'categories': [{'measurements': [{'value': '22.00', 'spread': '5.831', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 25', 'description': 'The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Change from Baseline = PNH clone size at Week 25 - Baseline PNH clone size. Baseline was the baseline value from the primary Study ACH471-100.', 'unitOfMeasure': 'percentage of the total cell population', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for PNH clone size."}, {'type': 'PRIMARY', 'title': 'Change From Baseline in AP Complement Functional Activity at Week 25', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \\[mg\\] 3 times daily \\[TID\\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.'}], 'classes': [{'categories': [{'measurements': [{'value': '-46.36', 'spread': '25.316', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 25', 'description': 'Serum AP functional activity was measured by the Wieslab functional immunoassay method. Change from Baseline = Serum AP functional activity at Week 25 - Baseline Serum AP functional activity. Baseline was the baseline value from the primary Study ACH471-100.', 'unitOfMeasure': 'percentage of activity', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for Serum AP functional activity."}, {'type': 'PRIMARY', 'title': 'Change From Baseline in Free Hgb at Week 25', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \\[mg\\] 3 times daily \\[TID\\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.'}], 'classes': [{'categories': [{'measurements': [{'value': '59.83', 'spread': '66.414', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 25', 'description': 'Change from Baseline = free Hgb at Week 25 - Baseline free Hgb. Baseline was the baseline value from the primary Study ACH471-100.', 'unitOfMeasure': 'mg/dL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for free Hgb."}, {'type': 'PRIMARY', 'title': 'Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 and Grade 4 Adverse Events (AEs), And AEs Leading To Discontinuation', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \\[mg\\] 3 times daily \\[TID\\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.'}], 'classes': [{'title': 'Any TEAEs', 'categories': [{'measurements': [{'value': '8', 'groupId': 'OG000'}]}]}, {'title': 'SAEs', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'TEAE Grade 3', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'TEAE Grade 4', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'AE leading to discontinuation', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline up to 4.5 years', '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 all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.", 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in LDH Level at Weeks 49 and 169', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \\[mg\\] 3 times daily \\[TID\\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.'}], 'classes': [{'title': 'Change at Week 49', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-862.50', 'spread': '568.302', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 169', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-1388.00', 'spread': '562.857', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Weeks 49 and 169', 'description': 'Change from Baseline = Serum LDH levels at specified postbaseline visit - Baseline Serum LDH levels. Baseline was the baseline value from the primary Study ACH471-100.', 'unitOfMeasure': 'U/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for LDH level. 'Number analyzed' = participants evaluable at specified timepoint."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Weeks 49 and 169', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \\[mg\\] 3 times daily \\[TID\\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.'}], 'classes': [{'title': 'Change at Week 49', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '21.40', 'spread': '18.995', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 169', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '54.50', 'spread': '14.849', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Weeks 49 and 169', 'description': 'Change from Baseline = Hgb levels at specified postbaseline visit - Baseline Hgb levels. Baseline was the baseline value from the primary Study ACH471-100.', 'unitOfMeasure': 'g/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for Hgb level. 'Number analyzed' = participants evaluable at specified timepoint."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Reticulocyte Counts at Weeks 49 and 169', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \\[mg\\] 3 times daily \\[TID\\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.'}], 'classes': [{'title': 'Change at Week 49', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.05', 'spread': '0.065', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 169', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.12', 'spread': '0.049', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Weeks 49 and 169', 'description': 'Change from Baseline = reticulocyte count at specified postbaseline visit - Baseline reticulocyte count. Baseline was the baseline value from the primary Study ACH471-100.', 'unitOfMeasure': '10^12 cells/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for reticulocyte count. 'Number analyzed' = participants evaluable at specified timepoint."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in PNH Clone Size at Weeks 49 and 73', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \\[mg\\] 3 times daily \\[TID\\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.'}], 'classes': [{'title': 'Change at Week 49', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '30.83', 'spread': '11.531', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 73', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '32.00', 'spread': '19.170', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Weeks 49 and 73', 'description': 'The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Change from Baseline = PNH clone size at specified postbaseline visit - Baseline PNH clone size. Baseline was the baseline value from the primary Study ACH471-100.', 'unitOfMeasure': 'percentage of the total cell population', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for PNH clone size. 'Number analyzed' = participants evaluable at specified timepoint."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in AP Complement Functional Activity at Weeks 49 and 145', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \\[mg\\] 3 times daily \\[TID\\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.'}], 'classes': [{'title': 'Change at Week 49', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-59.80', 'spread': '14.217', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 145', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-52.43', 'spread': '2.779', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Weeks 49 and 145', 'description': 'Serum AP functional activity was measured by the Wieslab functional immunoassay method. Change from Baseline = Serum AP functional activity at specified postbaseline visit - Baseline Serum AP functional activity. Baseline was the baseline value from the primary Study ACH471-100.', 'unitOfMeasure': 'percentage of activity', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for Serum AP functional activity. 'Number analyzed' = participants evaluable at specified timepoint."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Free Hgb at Weeks 49 and 169', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \\[mg\\] 3 times daily \\[TID\\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.'}], 'classes': [{'title': 'Change at Week 49', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '105.92', 'spread': '92.308', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 169', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-30.75', 'spread': '54.942', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Weeks 49 and 169', 'description': 'Change from Baseline = free Hgb at specified postbaseline visit - Baseline free Hgb. Baseline was the baseline value from the primary Study ACH471-100.', 'unitOfMeasure': 'mg/dL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for free Hgb. 'Number analyzed' = participants evaluable at specified timepoint."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at Weeks 21, 41, and 153', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \\[mg\\] 3 times daily \\[TID\\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.'}], 'classes': [{'title': 'Change at Week 21', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8.4', 'spread': '11.75', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 41', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '9.1', 'spread': '13.37', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 153', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.0', 'spread': '0.00', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Weeks 21, 41, and 153', 'description': 'The FACIT-Fatigue scale is a collection of quality of life questionnaires pertaining to the management of fatigue symptoms due to a chronic illness. The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Participants score each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores range from 0 to 52, with higher score indicating better quality of life.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for FACIT-Fatigue scale score. 'Number analyzed' = participants evaluable at specified timepoint."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in European Organisation for Research and Treatment of Cancer, Quality of Life Questionnaire-Core 30 Scale (EORTC-QLQ-C30): Global Health Status/Qol Score at Weeks 21, 41, and 153', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Danicopan', 'description': 'Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \\[mg\\] 3 times daily \\[TID\\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.'}], 'classes': [{'title': 'Change at Week 21', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '16.67', 'spread': '25.973', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 41', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '11.90', 'spread': '29.603', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 153', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.17', 'spread': '17.678', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Weeks 21, 41, and 153', 'description': "EORTC-QLQ-C30 is comprised of 30 questions. First 28 questions used 4-point scale (1=not at all,2=a little,3=quite a bit,4=very much) for evaluating 5 functional scales (physical, role, emotional, cognitive, social), 3 symptom scales (fatigue, nausea/vomiting, pain) \\& other single items. For each item, high score = high level of symptomatology/problem. Last 2 questions represented participant's assessment of overall health (global health status) and quality of life, coded on 7-point scale (1=very poor to 7=excellent). Answers were converted into grading scale, with total score between 0 and 100. A high score represented a favourable outcome with a best quality of life for participant.", 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for EORTC-QLQ-C30 (Global Health Status/Qol) score. 'Number analyzed' = participants evaluable at specified timepoint."}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Danicopan', 'description': 'Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \\[mg\\] 3 times daily \\[TID\\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}]}, {'type': 'Received at Least 1 Dose of Study Drug', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '6'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Missing follow-up visit', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}], 'preAssignmentDetails': 'Participants who demonstrated a clinical benefit from danicopan in the primary Study ACH471-100 (NCT03053102), were eligible for long-term treatment with danicopan in this extension study ACH471-103.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Danicopan', 'description': 'Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \\[mg\\] 3 times daily \\[TID\\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '37.51', 'spread': '14.752', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '4', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '4', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '8', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'Race', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'BG000'}]}], 'categories': [{'title': 'White', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Other', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Lactate Dehydrogenase (LDH) Level', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '1403.13', 'spread': '602.824', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'units (U)/liter (L)', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Hemoglobin (Hgb) Level in the Absence of Red Blood Cell (RBC) Transfusion', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '96.50', 'spread': '18.055', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'grams (g)/liter (L)', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Reticulocyte Counts', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '0.15', 'spread': '0.078', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': '10^12 cells/L', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Paroxysmal Nocturnal Hemoglobinuria (PNH) Clone Size', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '39.67', 'spread': '29.696', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'percentage of the total cell population', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': "Here, 'number analyzed' signifies participants evaluable for this baseline measure."}, {'title': 'Free Hgb', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '36.08', 'spread': '42.417', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'mg/deciliter (dL)', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Alternative Pathway (AP) Complement Functional Activity', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '66.70', 'spread': '12.732', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'Serum AP functional activity was measured by the Wieslab functional immunoassay method.', 'unitOfMeasure': 'percentage of activity', 'dispersionType': 'STANDARD_DEVIATION'}], 'populationDescription': 'Full analysis set included all enrolled and treated participants.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2021-06-04', 'size': 1612328, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2022-12-21T12:56', 'hasProtocol': True}, {'date': '2021-02-23', 'size': 2733719, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2022-12-21T12:56', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 8}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2017-06-22', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-02', 'completionDateStruct': {'date': '2022-01-04', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-02-14', 'studyFirstSubmitDate': '2017-06-05', 'resultsFirstSubmitDate': '2022-12-21', 'studyFirstSubmitQcDate': '2017-06-07', 'lastUpdatePostDateStruct': {'date': '2023-03-14', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2023-02-14', 'studyFirstPostDateStruct': {'date': '2017-06-09', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2023-03-14', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-01-04', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change From Baseline in LDH Level at Week 25', 'timeFrame': 'Baseline, Week 25', 'description': 'Change from Baseline = Serum LDH levels at Week 25 - Baseline Serum LDH levels. Baseline was the baseline value from the primary Study ACH471-100.'}, {'measure': 'Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Week 25', 'timeFrame': 'Baseline, Week 25', 'description': 'Change from Baseline = Hgb levels at Week 25 - Baseline Hgb levels. Baseline was the baseline value from the primary Study ACH471-100.'}, {'measure': 'Change From Baseline in Reticulocyte Counts at Week 25', 'timeFrame': 'Baseline, Week 25', 'description': 'Change from Baseline = reticulocyte count at Week 25 - Baseline reticulocyte count. Baseline was the baseline value from the primary Study ACH471-100.'}, {'measure': 'Number of RBC Units Transfused', 'timeFrame': 'Baseline up to Week 169'}, {'measure': 'Number of RBC Transfusion Instances', 'timeFrame': 'Baseline up to Week 169'}, {'measure': 'Change From Baseline in PNH Clone Size at Week 25', 'timeFrame': 'Baseline, Week 25', 'description': 'The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Change from Baseline = PNH clone size at Week 25 - Baseline PNH clone size. Baseline was the baseline value from the primary Study ACH471-100.'}, {'measure': 'Change From Baseline in AP Complement Functional Activity at Week 25', 'timeFrame': 'Baseline, Week 25', 'description': 'Serum AP functional activity was measured by the Wieslab functional immunoassay method. Change from Baseline = Serum AP functional activity at Week 25 - Baseline Serum AP functional activity. Baseline was the baseline value from the primary Study ACH471-100.'}, {'measure': 'Change From Baseline in Free Hgb at Week 25', 'timeFrame': 'Baseline, Week 25', 'description': 'Change from Baseline = free Hgb at Week 25 - Baseline free Hgb. Baseline was the baseline value from the primary Study ACH471-100.'}, {'measure': 'Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 and Grade 4 Adverse Events (AEs), And AEs Leading To Discontinuation', 'timeFrame': 'Baseline up to 4.5 years', '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 all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section."}], 'secondaryOutcomes': [{'measure': 'Change From Baseline in LDH Level at Weeks 49 and 169', 'timeFrame': 'Baseline, Weeks 49 and 169', 'description': 'Change from Baseline = Serum LDH levels at specified postbaseline visit - Baseline Serum LDH levels. Baseline was the baseline value from the primary Study ACH471-100.'}, {'measure': 'Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Weeks 49 and 169', 'timeFrame': 'Baseline, Weeks 49 and 169', 'description': 'Change from Baseline = Hgb levels at specified postbaseline visit - Baseline Hgb levels. Baseline was the baseline value from the primary Study ACH471-100.'}, {'measure': 'Change From Baseline in Reticulocyte Counts at Weeks 49 and 169', 'timeFrame': 'Baseline, Weeks 49 and 169', 'description': 'Change from Baseline = reticulocyte count at specified postbaseline visit - Baseline reticulocyte count. Baseline was the baseline value from the primary Study ACH471-100.'}, {'measure': 'Change From Baseline in PNH Clone Size at Weeks 49 and 73', 'timeFrame': 'Baseline, Weeks 49 and 73', 'description': 'The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Change from Baseline = PNH clone size at specified postbaseline visit - Baseline PNH clone size. Baseline was the baseline value from the primary Study ACH471-100.'}, {'measure': 'Change From Baseline in AP Complement Functional Activity at Weeks 49 and 145', 'timeFrame': 'Baseline, Weeks 49 and 145', 'description': 'Serum AP functional activity was measured by the Wieslab functional immunoassay method. Change from Baseline = Serum AP functional activity at specified postbaseline visit - Baseline Serum AP functional activity. Baseline was the baseline value from the primary Study ACH471-100.'}, {'measure': 'Change From Baseline in Free Hgb at Weeks 49 and 169', 'timeFrame': 'Baseline, Weeks 49 and 169', 'description': 'Change from Baseline = free Hgb at specified postbaseline visit - Baseline free Hgb. Baseline was the baseline value from the primary Study ACH471-100.'}, {'measure': 'Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at Weeks 21, 41, and 153', 'timeFrame': 'Baseline, Weeks 21, 41, and 153', 'description': 'The FACIT-Fatigue scale is a collection of quality of life questionnaires pertaining to the management of fatigue symptoms due to a chronic illness. The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Participants score each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores range from 0 to 52, with higher score indicating better quality of life.'}, {'measure': 'Change From Baseline in European Organisation for Research and Treatment of Cancer, Quality of Life Questionnaire-Core 30 Scale (EORTC-QLQ-C30): Global Health Status/Qol Score at Weeks 21, 41, and 153', 'timeFrame': 'Baseline, Weeks 21, 41, and 153', 'description': "EORTC-QLQ-C30 is comprised of 30 questions. First 28 questions used 4-point scale (1=not at all,2=a little,3=quite a bit,4=very much) for evaluating 5 functional scales (physical, role, emotional, cognitive, social), 3 symptom scales (fatigue, nausea/vomiting, pain) \\& other single items. For each item, high score = high level of symptomatology/problem. Last 2 questions represented participant's assessment of overall health (global health status) and quality of life, coded on 7-point scale (1=very poor to 7=excellent). Answers were converted into grading scale, with total score between 0 and 100. A high score represented a favourable outcome with a best quality of life for participant."}]}, 'oversightModule': {'isUsExport': True, 'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['PNH', 'Paroxysmal', 'Hemoglobinuria', 'ACH-0144471', 'ALXN2040', 'Danicopan'], 'conditions': ['Paroxysmal Nocturnal Hemoglobinuria']}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to evaluate the long-term safety and efficacy of ACH-0144471 in participants with paroxysmal nocturnal hemoglobinuria (PNH) who have demonstrated clinical benefit from ACH-0144471 in Study ACH471-100. This study is designed to include up to 12 participants.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Study designed to include up to 12 participants who completed treatment in Study ACH471-100 and demonstrated clinical benefit from ACH-0144471 with no significant safety or tolerability concerns.\n* Negative pregnancy test for females prior to dosing and throughout the study.\n\nExclusion Criteria:\n\n* Have developed any clinically relevant co-morbidities while participating in Study ACH471-100 that would make the participant inappropriate for the continuation of treatment with ACH-0144471, in the opinion of the Investigator.\n* Have developed any clinically significant laboratory abnormalities while participating in Study ACH471-100 that, in the opinion of the Investigator, would make the participant inappropriate for the study or put the participant at undue risk.\n* Females who are 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 is pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration.'}, 'identificationModule': {'nctId': 'NCT03181633', 'briefTitle': 'A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Alexion Pharmaceuticals, Inc.'}, 'officialTitle': 'An Open-Label Study to Evaluate Efficacy and Safety of Long-Term Treatment With ACH-0144471 in Participants Who Completed Clinical Study ACH471-100', 'orgStudyIdInfo': {'id': 'ACH471-103'}, 'secondaryIdInfos': [{'id': '2017-000665-79', 'type': 'EUDRACT_NUMBER'}, {'id': 'U1111-1196-0653', 'type': 'OTHER', 'domain': 'UTN'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'ACH-0144471', 'description': 'All participants will receive ACH-0144471 during the treatment period.', 'interventionNames': ['Drug: ACH-0144471']}], 'interventions': [{'name': 'ACH-0144471', 'type': 'DRUG', 'description': 'ACH-0144471 will be administered to all participants enrolled in the study.', 'armGroupLabels': ['ACH-0144471']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Avellino', 'country': 'Italy', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 40.91494, 'lon': 14.79103}}, {'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}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Alexion Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Achillion, a wholly owned subsidiary of Alexion', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}