REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
Ориентир для пациента и семьи
Простыми словами
Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.
- Что изучают
- Это наблюдательное исследование: исследуемое лечение участникам по протоколу не назначают.
- Кому может быть актуально
- Состояния в реестре: Paroxysmal Nocturnal Hemoglobinuria (PNH). Базовые параметры: от 18 лет · Все.
- Что важно проверить
- Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
- Где проводится
- Италия
- Следующий шаг
- Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →
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Официальное название
REAL-CARE: Real-world Effectiveness of iptacopAn in itaLian Patients With Paroxysmal noCturnAl HemoglobinuRia: an Observational Study
Обзор
This study evaluates iptacopan effectiveness and safety in routine clinical practice, with a focus on hematological response, transfusion avoidance, and patient-reported outcomes. The 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.
Подробное описание
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.
No 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.
Первичные конечные точки
- absolute change in Hb level post iptacopan initiation [Срок оценки: 12 months]
- Proportion of patients with no RBC transfusions [Срок оценки: 12 months]
Вторичные конечные точки (12)
- Socio-demographic and clinical variables of Paroxysmal Nocturnal Hemoglobinuria population [Срок оценки: date of iptacopan start, day 0]
- Absolute change in Hb level post iptacopan initiation [Срок оценки: date of iptacopan start (day 0), 24 months]
- Proportion of patients who didn't need RBC transfusion [Срок оценки: 24 months]
- Proportion of patients with Hb levels ≥ 12 g/dL AND no RBC transfusion [Срок оценки: 12 month, 24 month]
- Proportion of patients with change in Hb levels ≥ 2 g/dL post iptacopan initiation AND no RBC transfusion [Срок оценки: 12 month, 24 month]
- Average number of RBC transfusion [Срок оценки: 12 months and 24 months]
- Percentage change in LDH level post iptacopan initiation [Срок оценки: date of iptacopan start (day 0), month 12, month 24]
- Change in reticulocyte count post iptacopan initiation. [Срок оценки: date of iptacopan start (day 0), month 12, month 24]
- Absolute change in bilirubin level post iptacopan initiation. [Срок оценки: date of iptacopan start (day 0), month 12, month 24]
- Frequency of adverse events (AE) and serious adverse events (SAE) over 12 and 24 months of follow-up [Срок оценки: 12 months and 24 months]
- Proportion of BTH occurrence [Срок оценки: 12 months and 24 months]
- Type and proportion of Infections occurrence over 12 and 24 months of follow-up [Срок оценки: 12 months and 24 months]
Критерии участия
Критерии включения
- 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
- Male and female,
- ≥ 18 years of age,
- documented diagnosis of PNH,
- followed in Italian clinical sites,
- 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))
Критерии исключения
1.Concomitant participation in an interventional clinical study related to PNH or its treatment.
No other inclusion/exclusion criteria apply other than the requirements stated in the Summary of Product Characteristics (SmPC).
Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.
Здоровые добровольцы: Нет
Дизайн исследования
- Модель наблюдения
- Когортное
Центры проведения
Италия · 12 центров
- Novartis Investigative Site — Alessandria
- Novartis Investigative Site — Avellino
- Novartis Investigative Site — Brescia
- Novartis Investigative Site — Florence
- Novartis Investigative Site — Genova
- Novartis Investigative Site — Milan
- Novartis Investigative Site — Milan
- Novartis Investigative Site — Palermo
- … и ещё 4 центра
Идентификаторы
NCT: NCT07229235 · CLNP023C1IT01