REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH). Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Italy
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
REAL-CARE: Real-world Effectiveness of iptacopAn in itaLian Patients With Paroxysmal noCturnAl HemoglobinuRia: an Observational Study
Overview
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.
Detailed description
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.
Primary outcome measures
- absolute change in Hb level post iptacopan initiation [Time frame: 12 months]
- Proportion of patients with no RBC transfusions [Time frame: 12 months]
Secondary outcome measures (12)
- Socio-demographic and clinical variables of Paroxysmal Nocturnal Hemoglobinuria population [Time frame: date of iptacopan start, day 0]
- Absolute change in Hb level post iptacopan initiation [Time frame: date of iptacopan start (day 0), 24 months]
- Proportion of patients who didn't need RBC transfusion [Time frame: 24 months]
- Proportion of patients with Hb levels ≥ 12 g/dL AND no RBC transfusion [Time frame: 12 month, 24 month]
- Proportion of patients with change in Hb levels ≥ 2 g/dL post iptacopan initiation AND no RBC transfusion [Time frame: 12 month, 24 month]
- Average number of RBC transfusion [Time frame: 12 months and 24 months]
- Percentage change in LDH level post iptacopan initiation [Time frame: date of iptacopan start (day 0), month 12, month 24]
- Change in reticulocyte count post iptacopan initiation. [Time frame: date of iptacopan start (day 0), month 12, month 24]
- Absolute change in bilirubin level post iptacopan initiation. [Time frame: 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 [Time frame: 12 months and 24 months]
- Proportion of BTH occurrence [Time frame: 12 months and 24 months]
- Type and proportion of Infections occurrence over 12 and 24 months of follow-up [Time frame: 12 months and 24 months]
Eligibility criteria
Inclusion criteria
- 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))
Exclusion criteria
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).
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Italy · 12 centers
- 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
- … and 4 more centers
Identifiers
NCT: NCT07229235 · CLNP023C1IT01