REAL-CARE: Real-world Effectiveness of iptacopAn in itaLian Patients With Paroxysmal noCturnAl HemoglobinuRia: an Observational Study
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 50
- 试验地点
- 12
- 主要终点
- absolute change in Hb level post iptacopan initiation
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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))
排除标准
- •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).
研究组 & 干预措施
Iptacopan
Treatment with iptacopan
结局指标
主要结局
absolute change in Hb level post iptacopan initiation
时间窗: 12 months
12-month absolute change in hemoglobin (Hb) level post iptacopan initiation
Proportion of patients with no RBC transfusions
时间窗: 12 months
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.
次要结局
- Socio-demographic and clinical variables of Paroxysmal Nocturnal Hemoglobinuria population(date of iptacopan start, day 0)
- Absolute change in Hb level post iptacopan initiation(date of iptacopan start (day 0), 24 months)
- Proportion of patients who didn't need RBC transfusion(24 months)
- Proportion of patients with Hb levels ≥ 12 g/dL AND no RBC transfusion(12 month, 24 month)
- Proportion of patients with change in Hb levels ≥ 2 g/dL post iptacopan initiation AND no RBC transfusion(12 month, 24 month)
- Average number of RBC transfusion(12 months and 24 months)
- Percentage change in LDH level post iptacopan initiation(date of iptacopan start (day 0), month 12, month 24)
- Change in reticulocyte count post iptacopan initiation.(date of iptacopan start (day 0), month 12, month 24)
- Absolute change in bilirubin level post iptacopan initiation.(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(12 months and 24 months)
- Proportion of BTH occurrence(12 months and 24 months)
- Type and proportion of Infections occurrence over 12 and 24 months of follow-up(12 months and 24 months)
- Type and proportion of new comorbidities(12 months and 24 months)
- Type and proportion of major adverse vascular events (MAVEs) occurrence(12 months and 24 months)
- Type and proportion of concomitant medications newly prescribed(12 months and 24 months)
- Absolute change in Medication Adherence Report Scale - 5 item (MARS-5) scores(date of iptacopan start (day 0), month 12 and month 24)
- Absolute change from iptacopan initiation in PROMIS-29+2 score.(date of iptacopan start (day 0), month 12 and month 24)
- Number of hospitalizations, emergency room visits, and hospital-based outpatient visits attributable to the management of AEs related to the study treatment(Up to 24 months)
- Absolute change from iptacopan initiation in WPAI:SHP score.(date of iptacopan start (day 0), month 12 and month 24)