REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria

Novartis

Status: Not yet enrolling

Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH)

Study type: Observational

Funder types: Industry

Identifiers

NCT07229235

CLNP023C1IT01

Details and patient eligibility

About

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.

Full 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.

Enrollment: 50 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. 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
2. Male and female,
3. ≥ 18 years of age,
4. documented diagnosis of PNH,
5. followed in Italian clinical sites,
6. 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).

Trial design

50 participants in 1 patient group

Iptacopan

Description:

Treatment with iptacopan