Real-world use of iptacopan for PNH in Italy

REAL-CARE: Real-world Effectiveness of iptacopAn in itaLian Patients With Paroxysmal noCturnAl HemoglobinuRia: an Observational Study

This project will see if iptacopan works and is safe for adults with paroxysmal nocturnal hemoglobinuria (PNH) who are treated in Italian clinics.

Quick facts

• Study type: Observational
• Enrollment: 50 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Novartis (Industry-sponsored)
• Locations: 7 sites (Alessandria, AL and 6 other locations)
• Trial ID: NCT07229235 on ClinicalTrials.gov

What this trial studies

This is a multicenter observational program enrolling adults with PNH who are starting iptacopan or who were treated under the Managed Access Program, using retrospective, retro-prospective, and prospective cohorts. Primary data will be collected prospectively while secondary data will be retrieved from medical records for patients previously treated under the MAP. Patients are indexed at the date of iptacopan initiation and followed for up to 24 months, with the primary outcome being change in hemoglobin at 12 months and the proportion free from red blood cell transfusion from Day 14 through Month 12. No extra visits or procedures are mandated beyond routine care, and patient questionnaires are collected when possible but are not required.

Who should consider this trial

Good fit: Ideal candidates are adults (≥18 years) with a documented diagnosis of PNH who receive iptacopan as part of routine care at participating Italian sites, including those transitioning from the Managed Access Program.

Not a fit: Patients who are not on iptacopan, who are enrolled in interventional PNH trials, who are under 18, or who are treated outside participating Italian centers are unlikely to gain benefit from participation.

Why it matters

Potential benefit: If successful, the study could show that iptacopan raises hemoglobin and reduces the need for red blood cell transfusions in real-world practice.

How similar studies have performed: Randomized clinical trials of iptacopan have shown promising efficacy and safety in PNH, but real-world observational evidence remains limited.

Eligibility criteria

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).

Where this trial is running

Alessandria, AL and 6 other locations

• Novartis Investigative Site — Alessandria, Al, Italy (Recruiting)
• Novartis Investigative Site — Avellino, Av, Italy (Recruiting)
• Novartis Investigative Site — Brescia, Bs, Italy (Recruiting)
• Novartis Investigative Site — Florence, Fi, Italy (Recruiting)
• Novartis Investigative Site — Palermo, PA, Italy (Recruiting)
• Novartis Investigative Site — Roma, Rm, Italy (Recruiting)
• Novartis Investigative Site — Bassano del Grappa, Vi, Italy (Recruiting)

Study contacts

• Study coordinator: Novartis Pharmaceuticals

Conditions Paroxysmal Nocturnal Hemoglobinuria; REAL-CARE; ptacopan; paroxysmal nocturnal hemoglobinuria; PNH; NIS