Ignite Creation Date:
2025-12-24 @ 10:34 PM
Ignite Modification Date:
2025-12-25 @ 8:06 PM
Study NCT ID:
NCT07229235
Status:
NOT_YET_RECRUITING
Last Update Posted:
2025-11-14
First Post:
2025-10-30
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
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': 'OTHER', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 50}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2025-11-21', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-11', 'completionDateStruct': {'date': '2028-08-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-11-13', 'studyFirstSubmitDate': '2025-10-30', 'studyFirstSubmitQcDate': '2025-11-13', 'lastUpdatePostDateStruct': {'date': '2025-11-14', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2025-11-14', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2028-08-31', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'absolute change in Hb level post iptacopan initiation', 'timeFrame': '12 months', 'description': '12-month absolute change in hemoglobin (Hb) level post iptacopan initiation'}, {'measure': 'Proportion of patients with no RBC transfusions', 'timeFrame': '12 months', 'description': "12-month proportion of patients with no Red Blood Cells (RBC) transfusions, prescribed as per local requirement and based on Investigator's judgment, after Day 14."}], 'secondaryOutcomes': [{'measure': 'Socio-demographic and clinical variables of Paroxysmal Nocturnal Hemoglobinuria population', 'timeFrame': 'date of iptacopan start, day 0', 'description': 'Descriptive measures of the socio-demographic variables, proportion of PNH cells, clinical symptoms, laboratory parameters, concomitant diseases and treatments and outcomes of PNH patients at iptacopan initiation.'}, {'measure': 'Absolute change in Hb level post iptacopan initiation', 'timeFrame': 'date of iptacopan start (day 0), 24 months', 'description': '24-month absolute change in Hb level post iptacopan initiation'}, {'measure': "Proportion of patients who didn't need RBC transfusion", 'timeFrame': '24 months', 'description': "24-month proportion of patients who didn't need RBC transfusion, prescribed as per local requirement and based on Investigator's judgment, post iptacopan initiation"}, {'measure': 'Proportion of patients with Hb levels ≥ 12 g/dL AND no RBC transfusion', 'timeFrame': '12 month, 24 month', 'description': "12- and 24-month proportion of patients with Hb levels ≥ 12 g/dL AND no RBC transfusion, prescribed as per local requirement and based on Investigator's judgment, between Day 14 and month 12 or month 24"}, {'measure': 'Proportion of patients with change in Hb levels ≥ 2 g/dL post iptacopan initiation AND no RBC transfusion', 'timeFrame': '12 month, 24 month', 'description': "12- and 24-month proportion of patients with change in Hb levels ≥ 2 g/dL post iptacopan initiation AND no RBC transfusion, prescribed as per local requirement and based on Investigator's judgment, between Day 14 and month 12 or month 24"}, {'measure': 'Average number of RBC transfusion', 'timeFrame': '12 months and 24 months', 'description': "Average number of RBC transfusion, prescribed as per local requirement and based on Investigator's judgment, per patient during 12- and 24-months post iptacopan initiation"}, {'measure': 'Percentage change in LDH level post iptacopan initiation', 'timeFrame': 'date of iptacopan start (day 0), month 12, month 24', 'description': '12- and 24-month percentage change in LDH level post iptacopan initiation.'}, {'measure': 'Change in reticulocyte count post iptacopan initiation.', 'timeFrame': 'date of iptacopan start (day 0), month 12, month 24', 'description': '12- and 24-month change in reticulocyte count post iptacopan initiation.'}, {'measure': 'Absolute change in bilirubin level post iptacopan initiation.', 'timeFrame': 'date of iptacopan start (day 0), month 12, month 24', 'description': '12- and 24-month absolute change in bilirubin level post iptacopan initiation.'}, {'measure': 'Frequency of adverse events (AE) and serious adverse events (SAE) over 12 and 24 months of follow-up', 'timeFrame': '12 months and 24 months', 'description': 'Frequency of adverse events (AE) and serious adverse events (SAE) over 12 and 24 months of follow-up post iptacopan initiation'}, {'measure': 'Proportion of BTH occurrence', 'timeFrame': '12 months and 24 months', 'description': 'Proportion of Breakthrough Hemolysis (BTH) occurrence over 12 and 24 months of follow-up post iptacopan initiation'}, {'measure': 'Type and proportion of Infections occurrence over 12 and 24 months of follow-up', 'timeFrame': '12 months and 24 months', 'description': 'Type and proportion (count and percentage) of Infections occurrence over 12 and 24 months of follow-up post iptacopan initiation'}, {'measure': 'Type and proportion of major adverse vascular events (MAVEs) occurrence', 'timeFrame': '12 months and 24 months', 'description': 'Type and proportion of major adverse vascular events (MAVEs) occurrence over 12 and 24 months of follow-up post iptacopan initiation'}, {'measure': 'Type and proportion of new comorbidities', 'timeFrame': '12 months and 24 months', 'description': 'Type and proportion of new comorbidities occurred within 12 and 24 months of follow-up post iptacopan initiation'}, {'measure': 'Type and proportion of concomitant medications newly prescribed', 'timeFrame': '12 months and 24 months', 'description': 'Type and proportion of concomitant medications newly prescribed during 12 and 24 months of follow-up post iptacopan initiation'}, {'measure': 'Absolute change in Medication Adherence Report Scale - 5 item (MARS-5) scores', 'timeFrame': 'date of iptacopan start (day 0), month 12 and month 24', 'description': 'The MARS-5 questionnaire is a validated self-report questionnaire developed to assess patient adherence to prescribed medication, focusing on both intentional and unintentional non-adherence.\n\nIt consists of five items, each rated on a 5-point Likert Scale, from 0 ("Always") to 5 ("Never").\n\nItems evaluate behaviors such as forgetting to take medications, altering the dose or stopping the treatment. The total score ranges from 5 to 25, with higher scores indicating better adherence to treatment.'}, {'measure': 'Absolute change from iptacopan initiation in PROMIS-29+2 score.', 'timeFrame': 'date of iptacopan start (day 0), month 12 and month 24', 'description': 'The Patient-Reported Outcomes Measurement Information System (PROMIS) Profile-29+2 is a validated standardized patient reported outcome measure to assess generic health-related quality of life. The questionnaire covers the following nine health domains: Physical function, Anxiety, Depression, Fatigue, Sleep disturbance, Ability to participate in social roles and activities, Pain interference, Cognitive function and Pain intensity.\n\nThe recall period for all items is the past 7 days. Each of the first eight domains is assessed through 4 items using a 5-point Likert scale. Pain intensity is measured with a single item on an 11-point numeric rating scale from 0 ("no pain") to 10 ("worst imaginable pain").\n\nEach domain is scored separately, with T-scores used to represent the severity or level of functioning in each area. Higher scores can indicate either better health (e.g., physical function) or greater severity of symptoms (e.g., anxiety, depression).'}, {'measure': 'Absolute change from iptacopan initiation in WPAI:SHP score.', 'timeFrame': 'date of iptacopan start (day 0), month 12 and month 24', 'description': 'Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI:SHP) is a validated patient-reported outcome measure designed to assess the impact of a specific health problem on work and daily activities. In this study, the questionnaire has been customized for PNH. It includes six questions grouped into four key domains: Absenteeism (work time missed due to PNH), Presenteeism (impairment while working due to PNH), Overall work productivity loss (combined absenteeism and presenteeism), Activity impairment (impact of PNH on regular daily activities outside of work).\n\nThe recall period is the past 7 days. Responses are used to calculate the percentage of work time missed and the level of impairment experienced by the patient. Higher scores represent greater impairment and reduced productivity.'}, {'measure': 'Number of hospitalizations, emergency room visits, and hospital-based outpatient visits attributable to the management of AEs related to the study treatment', 'timeFrame': 'Up to 24 months', 'description': 'Healthcare resources utilization:\n\nThe number of hospitalizations, emergency room visits, and hospital-based outpatient visits attributable to the management of adverse events (AEs) related to the study treatment will be documented.'}]}, 'conditionsModule': {'keywords': ['REAL-CARE', 'ptacopan', 'paroxysmal nocturnal hemoglobinuria', 'PNH', 'NIS'], 'conditions': ['Paroxysmal Nocturnal Hemoglobinuria (PNH)']}, 'descriptionModule': {'briefSummary': "This study evaluates iptacopan effectiveness and safety in routine clinical practice, with a focus on hematological response, transfusion avoidance, and patient-reported outcomes.\n\nThe primary objective of the REAL-CARE study is to is to assess the long-term hematological response following iptacopan initiation. This will be assessed through the absolute change in hemoglobin (Hb) levels at 12 months post-initiation, and the proportion of patients who remain free from red blood cell (RBC) transfusions, prescribed as per local requirement and based on Investigator's judgment, from Day 14 through Month 12 after starting iptacopan.", 'detailedDescription': 'This is a local, multicenter, observational study with retrospective, retro-prospective and prospective cohorts. Adult patients with PNH either naïve to iptacopan or transitioning from the Managed Access Program (MAP) starting from January 2023 will be enrolled. Primary data will be collected prospectively; secondary data will be retrieved retrospectively from patients previously treated under MAP. A mix of primary and secondary data will be collected in retro-prospective patients. Patients will be indexed on the date of iptacopan initiation and will be followed up for 24 months or until iptacopan discontinuation (due to occurrence of an adverse event, lack of efficacy etc), clinical decision, death, administrative issues, consent withdrawal or loss to follow-up, whichever comes first. The enrollment period is planned to last 15 months, with potential adjustments based on enrollment progress. No treatment decision will be influenced by study participation.\n\nNo extra visits, examinations, or procedures are imposed. Questionnaires should be collected at the indicated timepoints whenever possible, but, since the administration of the questionnaires represents an additional procedure not included in routine clinical care, these timepoints are considered indicative only, and patients will not be recalled specifically to complete questionnaires if a routine clinical visit is not scheduled.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Italian population of adult patients with PNH either naïve to iptacopan or transitioning from the Managed Access Program (MAP) starting from January 2023', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion criteria:\n\n1. signed informed consent: patient must provide written informed before any study assessment is collected; in case of deceased patients, informed consent is not required as per Art. 110, comma 1 of Legislative Decree n° 196, dated 30 June 2003 as amended by D.lgs. 101/2018 and the "Autorizzazione generale al trattamento dei dati personali effettuato per scopi di ricerca scientifica" n° 9/2016\n2. Male and female,\n3. ≥ 18 years of age,\n4. documented diagnosis of PNH,\n5. followed in Italian clinical sites,\n6. on treatment with iptacopan prescribed as per routine medical care (i.e. patients naïve to treatment for whom the decision to start this treatment has already been made on the basis of clinical practice and according to SmPC and AIFA criteria and regardless of inclusion in this study or patients already treated under the Managed Access Program (MAP))\n\nExclusion criteria:\n\n1.Concomitant participation in an interventional clinical study related to PNH or its treatment.\n\nNo other inclusion/exclusion criteria apply other than the requirements stated in the Summary of Product Characteristics (SmPC).'}, 'identificationModule': {'nctId': 'NCT07229235', 'acronym': 'REAL-CARE', 'briefTitle': 'REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria', 'organization': {'class': 'INDUSTRY', 'fullName': 'Novartis'}, 'officialTitle': 'REAL-CARE: Real-world Effectiveness of iptacopAn in itaLian Patients With Paroxysmal noCturnAl HemoglobinuRia: an Observational Study', 'orgStudyIdInfo': {'id': 'CLNP023C1IT01'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Iptacopan', 'description': 'Treatment with iptacopan'}]}, 'contactsLocationsModule': {'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'}}}}