Ignite Creation Date:
2026-03-26 @ 3:18 PM
Ignite Modification Date:
2026-03-31 @ 5:44 AM
Study NCT ID:
NCT07457151
Status:
NOT_YET_RECRUITING
Last Update Posted:
2026-03-09
First Post:
2026-02-13
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Danicopan PMS in Korea
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'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'OTHER', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 8}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2026-03-31', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2026-03', 'completionDateStruct': {'date': '2028-06-27', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2026-03-03', 'studyFirstSubmitDate': '2026-02-13', 'studyFirstSubmitQcDate': '2026-03-03', 'lastUpdatePostDateStruct': {'date': '2026-03-09', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2026-03-09', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2028-06-27', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Safety (adverse events (AEs), serious AEs (SAEs), adverse drug reactions (ADRs), serious ADRs (SADRs), unexpected AEs/ADRs), AESI', 'timeFrame': 'Patient data will be gathered for up to 12 weeks from the first dose of the study drug.', 'description': 'To assess the safety of Danicopan as add-on therapy to a C5 inhibitor (Eculizumab or Ravulizumab)in patients with PNH in Korea.'}], 'secondaryOutcomes': [{'measure': 'Change from baseline in hemoglobin (Hgb) at Week 12', 'timeFrame': 'Effectiveness variables will be assessed at Week 12 or end of treatment (if treatment is discontinued before Week 12).', 'description': 'Hemoglobin (Hgb) will be summarized with descriptive statistics (mean, standard deviation, median, minimum, and maximum).'}, {'measure': 'Change from baseline in absolute reticulocyte count (ARC) at Week 12', 'timeFrame': 'Effectiveness variables will be assessed at Week 12 orend of treatment (if treatment is discontinued before Week 12)', 'description': 'Absolute reticulocyte count (ARC) will be summarized with descriptive statistics (mean, standard deviation, median, minimum, and maximum).'}, {'measure': 'Change from baseline in FACIT Fatigue scores at Week 12', 'timeFrame': 'Effectiveness variables will be assessed at Week 12 orend of treatment (if treatment is discontinued before Week 12)', 'description': 'FACIT Fatigue scores will be summarized with descriptive statistics (mean, standard deviation, median, minimum, and maximum).'}, {'measure': 'Proportion of patients with transfusion avoidance* at Week 12(* Transfusion Avoidance: Defined as remaining free from red blood cell transfusions)', 'timeFrame': 'Effectiveness variables will be assessed at Week 12 orend of treatment (if treatment is discontinued before Week 12)', 'description': 'The proportion of patients with transfusion avoidance will be summarized. Patients with transfusion avoidance will be considered to show effectiveness and the number and percentage of subjects corresponding to this classification will be presented, along with the 95% confidence interval (CI) for the percentage calculated using the Clopper-Pearson method.'}]}, 'conditionsModule': {'keywords': ['PNH'], 'conditions': ['Paroxysmal Nocturnal Hemoglobinuria']}, 'descriptionModule': {'briefSummary': 'As part of a post-approval commitment, the Korean health authority requests a study to characterize safety and effectiveness in patients who are treated with Danicopan as an add-on to ravulizumab or eculizumab in normal clinical practice settings. This study is designed to assess the known safety profile or identify previously unsuspected adverse reactions and to evaluate the effectiveness of Danicopan under conditions of routine daily medical practice in Korea.', 'detailedDescription': 'The objectives of this study are to assess the safety and effectiveness of Danicopan in a real world setting in patients who are prescribed with the study drug under the approved indication in Korea.\n\nPrimary objective(s) To assess the safety of Danicopan as add-on therapy to a C5 inhibitor (Eculizumab or Ravulizumab)in patients with PNH in Korea.\n\nSecondary objective(s) To assess effectiveness of Danicopan as add- on therapy to a C5 inhibitor (Eculizumab or Ravulizumab) in patients with PNH at 12 weeks.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Patients currently treated with or initiating the study drug under approved local label and conditions of routine daily medical practice in Korea will be included in the study. This study will be conducted using a total surveillance method to investigate the safety and effectiveness of all patients being treated with the study drug.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Patients eligible for and treated with Danicpan as add-on therapy to a C5 inhibitor (Eculizumab or Ravulizumab) in patient with PNH in Korea\n2. Provision of a signed and dated written informed consent by the patient or their legally acceptable representative\n\nExclusion Criteria:\n\n1. Participation in any concurrent interventional trials during the period of study drug treatment\n2. Other off-label indications according to the approved label in Korea'}, 'identificationModule': {'nctId': 'NCT07457151', 'briefTitle': 'Danicopan PMS in Korea', 'organization': {'class': 'INDUSTRY', 'fullName': 'AstraZeneca'}, 'officialTitle': 'Danicopan Regulatory Post-Marketing Study in Korea', 'orgStudyIdInfo': {'id': 'D7332R00002'}}, 'contactsLocationsModule': {'centralContacts': [{'name': 'AstraZeneca Clinical Study Information Center', 'role': 'CONTACT', 'email': 'information.center@astrazeneca.com', 'phone': '1-877-240-9479'}]}, 'ipdSharingStatementModule': {'url': 'https://vivli.org/', 'timeFrame': 'AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA PhRMA Data Sharing Principles. For details of our timelines, please rerefer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.', 'ipdSharing': 'YES', 'description': 'Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment:\n\nhttps://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.', 'accessCriteria': 'When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org.\n\nSigned Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'AstraZeneca', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}