Ignite Creation Date:
2025-12-25 @ 4:42 AM
Ignite Modification Date:
2025-12-26 @ 3:43 AM
Study NCT ID:
NCT07036718
Status:
RECRUITING
Last Update Posted:
2025-12-01
First Post:
2025-06-17
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
NIS PDC the Efficacy and Safety of Iptacopan in Adults in Routine Clinical Practice
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': 'CASE_ONLY'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 100}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2025-07-28', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-11', 'completionDateStruct': {'date': '2028-01-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-11-27', 'studyFirstSubmitDate': '2025-06-17', 'studyFirstSubmitQcDate': '2025-06-17', 'lastUpdatePostDateStruct': {'date': '2025-12-01', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2025-06-25', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2028-01-31', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of patients with an increase in Hb level ≥ 20 g/l', 'timeFrame': '6 months', 'description': 'Number of patients who achieved an increase in Hb level ≥ 20 g/l over 6 months after iptacopan treatment initiation and were Red Blood Cells (RBC) transfusion independent.'}], 'secondaryOutcomes': [{'measure': 'Number of patients with an increase in Hb level ≥ 120 g/L', 'timeFrame': '12 months', 'description': 'Number of patients who achieved an increase in Hb level ≥ 120 g/L at 12 months after initiation of iptacopan treatment and were RBC transfusion independent'}, {'measure': 'Number of patients with an increase in Hb level ≥ 20 g/L', 'timeFrame': '12 months', 'description': 'Number of patients who achieved an increase in Hb level ≥ 20 g/L at 12 months after initiation of iptacopan treatment and was RBC transfusion independent'}, {'measure': 'Change from baseline in reticulocyte count (10⁹/L)', 'timeFrame': '12 months', 'description': 'Change from baseline in reticulocyte count (10⁹/L) over 12 months after initiation of iptacopan treatment'}, {'measure': 'Change from baseline in LDH levels (U/L)', 'timeFrame': '12 months', 'description': 'Change from baseline in LDH levels (U/L) over 12 months after initiation of iptacopan treatment'}, {'measure': 'Number of patients with an absence of administration of packed-RBC transfusions', 'timeFrame': '12 months', 'description': 'Number of patients with an absence of administration of packed-RBC transfusions at 12 months after initiation of iptacopan treatment'}, {'measure': 'Change from baseline in the Hb level (g/L)', 'timeFrame': 'Baseline, month 3, month 6 and month 12', 'description': 'Change from baseline in the Hb level (g/L) at 3, 6 and 12 months after initiation of iptacopan treatment'}, {'measure': 'Number of breakthrough hemolysis (BTH)', 'timeFrame': '12 months', 'description': 'Number of breakthrough hemolysis (BTH) cases 12 months following the initiation of iptacopan therapy'}, {'measure': 'Percentage of patients by BMI', 'timeFrame': '12 months', 'description': 'Percentage of patients by Body mass index (BMI) (\\<25, 25.1-29.9, \\>30)'}]}, 'oversightModule': {'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['PNH', 'Paroxysmal nocturnal hemoglobinuria', 'Iptacopan'], 'conditions': ['Paroxysmal Nocturnal Hemoglobinuria']}, 'descriptionModule': {'briefSummary': 'This is a prospective, non-interventional, multicenter study to evaluate the efficacy and safety of iptacopan in patients with PNH in real-world settings in Russia.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Enrollment of female and male patients with PNH', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Age ≥18 years at the start of iptacopan therapy.\n2. The patient is undergoing treatment with iptacopan.\n3. The treating physician decided to prescribe iptacopan based on the Summary of Product Characteristics during routine clinical practice, regardless of study participation.\n4. Provision of written informed consent.\n\nExclusion Criteria:\n\n1\\. Any situations where iptacopan is contraindicated in accordance with the Summary of Product Characteristics.'}, 'identificationModule': {'nctId': 'NCT07036718', 'acronym': 'PRIORITY- PNH', 'briefTitle': 'NIS PDC the Efficacy and Safety of Iptacopan in Adults in Routine Clinical Practice', 'organization': {'class': 'INDUSTRY', 'fullName': 'Novartis'}, 'officialTitle': 'A Prospective, Non-interventional Study to Evaluate the Efficacy and Safety of Iptacopan in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) in Routine Clinical Practice in the Russian Federation', 'orgStudyIdInfo': {'id': 'CLNP023C1RU03'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'iptacopan', 'description': 'Adults with PNH who started the iptacopan therapy'}]}, 'contactsLocationsModule': {'locations': [{'zip': '167904', 'city': 'Syktyvkar', 'state': 'Komi', 'status': 'RECRUITING', 'country': 'Russia', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 61.66393, 'lon': 50.8163}}, {'zip': '364047', 'city': 'Grozny', 'status': 'RECRUITING', 'country': 'Russia', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 43.31195, 'lon': 45.68895}}, {'zip': '350040', 'city': 'Krasnodar', 'status': 'RECRUITING', 'country': 'Russia', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 45.04534, 'lon': 38.98178}}, {'zip': '129110', 'city': 'Moscow', 'status': 'RECRUITING', 'country': 'Russia', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 55.75204, 'lon': 37.61781}}, {'zip': '183047', 'city': 'Murmansk', 'status': 'RECRUITING', 'country': 'Russia', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 68.96778, 'lon': 33.09922}}, {'zip': '194291', 'city': 'Saint Petersburg', 'status': 'RECRUITING', 'country': 'Russia', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 59.93863, 'lon': 30.31413}}, {'zip': '198205', 'city': 'Saint Petersburg', 'status': 'RECRUITING', 'country': 'Russia', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 59.93863, 'lon': 30.31413}}, {'zip': '693000', 'city': 'Yuzhno-Sakhalinsk', 'status': 'RECRUITING', 'country': 'Russia', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 46.9543, 'lon': 142.73559}}], 'centralContacts': [{'name': 'Novartis Pharmaceuticals', 'role': 'CONTACT', 'email': 'novartis.email@novartis.com', 'phone': '+41613241111'}, {'name': 'Novartis Pharmaceuticals', 'role': 'CONTACT'}], 'overallOfficials': [{'name': 'Novartis Pharmaceuticals', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Novartis Pharmaceuticals'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Novartis Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}