Ignite Creation Date:
2026-03-26 @ 3:15 PM
Ignite Modification Date:
2026-03-30 @ 2:18 AM
Study NCT ID:
NCT07413250
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2026-02-17
First Post:
2026-02-10
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2026-03-25'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006457', 'term': 'Hemoglobinuria, Paroxysmal'}], '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'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000718467', 'term': 'danicopan'}, {'id': 'C481642', 'term': 'eculizumab'}, {'id': 'C000629409', 'term': 'ravulizumab'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'OTHER', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 50}, 'targetDuration': '6 Years', 'patientRegistry': True}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2026-01-14', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2026-01', 'completionDateStruct': {'date': '2030-01-15', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2026-02-10', 'studyFirstSubmitDate': '2026-02-10', 'studyFirstSubmitQcDate': '2026-02-10', 'lastUpdatePostDateStruct': {'date': '2026-02-17', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2026-02-17', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2030-01-15', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)', 'timeFrame': 'From initiation of danicopan until discontinuation + 7 days, or end of follow-up, up to 5 years'}, {'measure': 'Event Rate of Meningococcal Infections', 'timeFrame': 'From initiation of danicopan until discontinuation + 7 days, or end of follow-up, up to 5 years'}, {'measure': 'Event Rate of Serious Infections', 'timeFrame': 'Up to approximately 5 years'}, {'measure': 'Event Rate of Malignancies and Hematologic Abnormalities', 'timeFrame': 'Up to approximately 5 years'}], 'secondaryOutcomes': [{'measure': 'Number of Participants with AEs and SAEs in Pregnant Participants, Pregnant Partners of Participants and Partners Who are Breastfeeding', 'timeFrame': 'Up to approximately 5 years'}, {'measure': 'Number of Infant Health Abnormalities in Pregnant Participants, Pregnant Partners of Participants and Partners Who are Breastfeeding', 'timeFrame': 'Up to 12 months'}, {'measure': 'Number of Participants with PNH symptoms at Diagnosis', 'timeFrame': 'Baseline (Day 1)'}, {'measure': 'Number of Participants with a History Bone Marrow Transplant', 'timeFrame': 'Baseline (Day 1)'}, {'measure': 'Number of Participants with a history of Major Adverse Vascular Events, including Thrombosis', 'timeFrame': 'Baseline (Day 1)'}, {'measure': 'Number of Participants with Ongoing Severe Hepatic Impairment as Defined by Child-Pugh Class C at Enrollment', 'timeFrame': 'Baseline (Day 1)'}, {'measure': 'Number of Participants Who Discontinue Danicopan Treatment', 'timeFrame': 'Up to approximately 5 years'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Paroxysmal Nocturnal Hemoglobinuria', 'PNH', 'Danicopan', 'Ravulizumab', 'Eculizumab'], 'conditions': ['Paroxysmal Nocturnal Hemoglobinuria', 'PNH']}, 'descriptionModule': {'briefSummary': 'This is a noninterventional registry-based cohort study utilizing data collected on danicopan-treated patients with paroxysmal nocturnal hemoglobinuria (PNH) through the International PNH Interest Group (IPIG) PNH registry. The primary objectives seek to characterize the long-term safety profile of danicopan as add-on therapy to ravulizumab/eculizumab in participants with PNH.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'The study population will consist of 2 treatment cohorts:\n\n* Participants ever-treated with danicopan as add-on therapy to ravulizumab/eculizumab on or after IPIG PNH Registry enrollment.\n* Participants initiating treatment with Ultomiris or Soliris on or after IPIG or Alexion International PNH Registry enrollment and without any danicopan treatment experience during follow-up.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Adult participants aged ≥ 18 years at treatment initiation.\n* Initiated treatment with Ultomiris, Soliris, and/or danicopan on or after IPIG or Alexion International PNH Registry enrollment.\n\nExclusion Criteria:\n\n* Participants without known year of birth, sex, informed consent date, or treatment status of danicopan and Ultomiris and/or Soliris.'}, 'identificationModule': {'nctId': 'NCT07413250', 'briefTitle': 'Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data', 'organization': {'class': 'INDUSTRY', 'fullName': 'Alexion Pharmaceuticals, Inc.'}, 'officialTitle': 'A Study to Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of International PNH Interest Group (IPIG)-Registry Data', 'orgStudyIdInfo': {'id': 'D7332R00003'}, 'secondaryIdInfos': [{'id': 'ALX-PNH-502', 'type': 'REGISTRY', 'domain': 'Alexion'}]}, 'armsInterventionsModule': {'armGroups': [{'label': 'Cohort 1', 'description': 'Participants ever-treated with danicopan as add-on therapy to ravulizumab/eculizumab on or after IPIG PNH Registry enrollment.', 'interventionNames': ['Drug: Danicopan']}, {'label': 'Cohort 2', 'description': 'Participants initiating treatment with Ultomiris or Soliris on or after IPIG or Alexion International PNH Registry enrollment and without any danicopan treatment experience during follow-up.', 'interventionNames': ['Drug: Soliris/Ultomiris']}], 'interventions': [{'name': 'Danicopan', 'type': 'DRUG', 'description': 'Participants treated with danicopan as an add-on therapy.', 'armGroupLabels': ['Cohort 1']}, {'name': 'Soliris/Ultomiris', 'type': 'DRUG', 'description': 'Participants treated with Soliris/Ultomiris monotherapy.', 'armGroupLabels': ['Cohort 2']}]}, 'contactsLocationsModule': {'locations': [{'zip': '02210', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Clinical Research Site', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Alexion Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}