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

Recruiting now

Conditions studied: Paroxysmal Nocturnal Hemoglobinuria (PNH)

In brief

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.

Key facts

Study ID

NCT07229235

Run by

Novartis Pharmaceuticals

People needed

50

Starts

2026-01-13

Expected to finish

2028-08-31

Last updated by the study team

2026-06-25

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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))

You may not qualify if…

• 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 it is running

• Novartis Investigative Site — Alessandria, AL, Italy (enrolling)
• Novartis Investigative Site — Avellino, AV, Italy (enrolling)
• Novartis Investigative Site — Brescia, BS, Italy (enrolling)
• Novartis Investigative Site — Florence, FI, Italy (enrolling)
• Novartis Investigative Site — Genova, GE, Italy (enrolling)
• Novartis Investigative Site — Milan, MI, Italy (enrolling)
• Novartis Investigative Site — Milan, MI, Italy (enrolling)
• Novartis Investigative Site — Palermo, PA, Italy (enrolling)
• Novartis Investigative Site — Roma, RM, Italy (enrolling)
• Novartis Investigative Site — Roma, RM, Italy (enrolling)
• Novartis Investigative Site — Torino, TO, Italy (enrolling)
• Novartis Investigative Site — Bassano del Grappa, VI, Italy (enrolling)

Full record on ClinicalTrials.gov

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