Ignite Creation Date:
2025-12-25 @ 2:09 AM
Ignite Modification Date:
2025-12-26 @ 12:35 AM
Study NCT ID:
NCT01374360
Status:
COMPLETED
Last Update Posted:
2025-02-24
First Post:
2011-04-15
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, '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': 'PROSPECTIVE', 'observationalModel': 'OTHER'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 6061}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2004-10-29', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-02', 'completionDateStruct': {'date': '2024-08-06', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-02-21', 'studyFirstSubmitDate': '2011-04-15', 'studyFirstSubmitQcDate': '2011-06-15', 'lastUpdatePostDateStruct': {'date': '2025-02-24', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2011-06-16', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2024-08-06', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Evaluate safety data specific to the use of Soliris and Ultomiris', 'timeFrame': 'Ongoing (up to 13 years)', 'description': 'Primary analyses will assess safety endpoints, including occurrence and time to first event for the following: meningococcal infections, infections with serious outcomes, formation of human anti-drug antibodies (ADA) to Soliris and Ultomiris, malignancy, thrombotic events, pulmonary hypertension, impaired renal function, impaired hepatic function, hemolysis, pregnancies, lactation, infusion reactions, bone marrow transplant, serious adverse events, and mortality.'}], 'secondaryOutcomes': [{'measure': 'Collect data to characterize the progression of PNH as well as clinical outcomes, mortality and morbidity in Soliris or Ultomiris and non-Soliris or non- Ultomiris treated patients', 'timeFrame': 'Ongoing (up to 13 years)', 'description': 'Secondary analyses will include descriptions of patient populations, PNH specific treatments, concomitant medications, progression of disease, PNH clone sites, clinical symptoms, and clinical outcomes.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['PNH', 'Paroxysmal Nocturnal hemoglobinuria', 'Soliris', 'Eculizumab', 'Ravulizumab', 'Ultomiris'], 'conditions': ['Paroxysmal Nocturnal Hemoglobinuria']}, 'referencesModule': {'references': [{'pmid': '37199789', 'type': 'DERIVED', 'citation': 'Dingli D, Maciejewski JP, Larratt L, Go RS, Hochsmann B, Zu K, Gustovic P, Kulagin AD. Relationship of paroxysmal nocturnal hemoglobinuria (PNH) granulocyte clone size to disease burden and risk of major vascular events in untreated patients: results from the International PNH Registry. Ann Hematol. 2023 Jul;102(7):1637-1644. doi: 10.1007/s00277-023-05269-4. Epub 2023 May 18.'}, {'pmid': '36108594', 'type': 'DERIVED', 'citation': 'Hill A, de Latour RP, Kulasekararaj AG, Griffin M, Brodsky RA, Maciejewski JP, Marantz JL, Gustovic P, Schrezenmeier H. Concomitant Immunosuppressive Therapy and Eculizumab Use in Patients with Paroxysmal Nocturnal Hemoglobinuria: An International PNH Registry Analysis. Acta Haematol. 2023;146(1):1-13. doi: 10.1159/000526979. Epub 2022 Sep 15.'}, {'pmid': '27766064', 'type': 'DERIVED', 'citation': 'Chou WC, Huang WH, Wang MC, Chang CS, Yeh SP, Chiou TJ, Chen YC, Lin TH, Shen MC; Taiwan PNH study group. Characteristics of Taiwanese patients of PNH in the international PNH registry. Thromb J. 2016 Oct 4;14(Suppl 1):39. doi: 10.1186/s12959-016-0094-0. eCollection 2016.'}, {'pmid': '26352814', 'type': 'DERIVED', 'citation': 'Kelly RJ, Hochsmann B, Szer J, Kulasekararaj A, de Guibert S, Roth A, Weitz IC, Armstrong E, Risitano AM, Patriquin CJ, Terriou L, Muus P, Hill A, Turner MP, Schrezenmeier H, Peffault de Latour R. Eculizumab in Pregnant Patients with Paroxysmal Nocturnal Hemoglobinuria. N Engl J Med. 2015 Sep 10;373(11):1032-9. doi: 10.1056/NEJMoa1502950.'}]}, 'descriptionModule': {'briefSummary': 'This study is a collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).', 'detailedDescription': 'Collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'samplingMethod': 'PROBABILITY_SAMPLE', 'studyPopulation': 'PNH Patients', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Patients of any age, including minors, with a diagnosis of PNH or a detected PNH clone, including patients previously treated with Soliris or Ultomiris and withdrawn from treatment. Patients who are minors must have parent/legal guardian consent and must be willing and able to give assent, if applicable as determined by the Ethics Committees/Institutional Review Boards. Upon attaining adulthood, these patients must be re-consented.\n* Ability to comprehend and sign consent to have data entered in the PNH Registry.\n\nExclusion Criteria:\n\n* Inability or unwillingness to sign informed consent.\n* Patients currently enrolled in an interventional clinical trial for treatment of PNH cannot be enrolled in the Registry at the same time.'}, 'identificationModule': {'nctId': 'NCT01374360', 'briefTitle': 'Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry', 'organization': {'class': 'INDUSTRY', 'fullName': 'Alexion Pharmaceuticals, Inc.'}, 'officialTitle': 'Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry', 'orgStudyIdInfo': {'id': 'M07-001'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Receiving Soliris or Ultomiris', 'description': 'PNH patients of any age, including minors, that are receiving Soliris or Ultomiris'}, {'label': 'Not receiving Soliris or Ultomiris', 'description': 'PNH patients of any age, including minors, that are not receiving Soliris or Ultomiris'}]}, 'contactsLocationsModule': {'locations': [{'zip': '02210', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Contact the PNH Registry at Alexion Pharmaceuticals, Inc. for worldwide locations.', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}], 'overallOfficials': [{'name': 'Phillipe Gustovic', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Alexion Pharmaceuticals, Inc.'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Alexion Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}