Actively Recruiting

Age: 18Years +
All Genders
NCT07229235

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

Led by Novartis Pharmaceuticals · Updated on 2026-04-16

50

Participants Needed

7

Research Sites

137 weeks

Total Duration

On this page

AI-Summary

What this Trial Is 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.

CONDITIONS

Official Title

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

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Signed informed consent before any study assessments
  • Male or female
  • 18 years of age or older
  • Documented diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH)
  • Followed at clinical sites in Italy
  • Receiving iptacopan treatment as part of routine medical care or transitioned from Managed Access Program (MAP)
Not Eligible

You will not qualify if you...

  • Currently participating in an interventional clinical study related to PNH or its treatment
  • Any other criteria outside those stated in the Summary of Product Characteristics (SmPC)

AI-Screening

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Trial Site Locations

Total: 7 locations

1

Novartis Investigative Site

Alessandria, AL, Italy, 15121

Actively Recruiting

2

Novartis Investigative Site

Avellino, AV, Italy, 83100

Actively Recruiting

3

Novartis Investigative Site

Brescia, BS, Italy, 25123

Actively Recruiting

4

Novartis Investigative Site

Florence, FI, Italy, 50134

Actively Recruiting

5

Novartis Investigative Site

Palermo, PA, Italy, 90146

Actively Recruiting

6

Novartis Investigative Site

Roma, RM, Italy, 00161

Actively Recruiting

7

Novartis Investigative Site

Bassano del Grappa, VI, Italy, 36061

Actively Recruiting

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Research Team

N

Novartis Pharmaceuticals

CONTACT

N

Novartis Pharmaceuticals

CONTACT

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

1

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