Actively Recruiting
REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria: an Observational Study
Led by Novartis Pharmaceuticals · Updated on 2026-05-18
50
Participants Needed
10
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the effectiveness and safety of iptacopan in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) in routine clinical practice. The study focuses on long-term hematological response, transfusion avoidance, and patient-reported outcomes. The main goal is to assess the change in hemoglobin levels and the proportion of patients who avoid red blood cell transfusions during the first year after starting iptacopan. This observational study includes adult patients who are either new to iptacopan treatment or transitioning from a Managed Access Program. Data will be collected retrospectively, prospectively, or as a combination, depending on the patient's treatment history. Patients will be followed for up to 24 months or until iptacopan is discontinued for various reasons. No extra visits or procedures beyond routine care are required, and questionnaires will be collected when possible during scheduled visits. Participants will be monitored through regular clinical assessments without any additional study-imposed visits. Researchers will collect data on hemoglobin levels, transfusion needs, and various laboratory measures over 12 and 24 months. Patient-reported outcomes and safety events will also be tracked. The study aims to gather real-world information on iptacopan's impact in clinical practice, with participation lasting up to two years or until treatment discontinuation.
CONDITIONS
Brief Title
REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Signed informed consent prior to any study assessment, except for deceased patients as per legal provisions
- Male or female
- At least 18 years of age
- Documented diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Followed at Italian clinical sites
- On treatment with iptacopan prescribed as part of routine medical care, either naive to treatment or previously treated under the Managed Access Program (MAP)
You will not qualify if you...
- Participation in another interventional clinical study related to PNH or its treatment
- No other criteria beyond the Summary of Product Characteristics (SmPC) apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 24 months or until iptacopan discontinuation, clinical decision, death, consent withdrawal, or loss to follow-up
Participants who undergo routine care with iptacopan are observed to collect clinical data and complete questionnaires at routine visits whenever possible.
Visits occur as part of routine clinical care; questionnaires are collected at routine visits but no extra visits are scheduled
Trial Site Locations
Total: 10 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
Milan, MI, Italy, 20132
Actively Recruiting
6
Novartis Investigative Site
Palermo, PA, Italy, 90146
Actively Recruiting
7
Novartis Investigative Site
Roma, RM, Italy, 00161
Actively Recruiting
8
Novartis Investigative Site
Roma, RM, Italy, 00168
Actively Recruiting
9
Novartis Investigative Site
Torino, TO, Italy, 10126
Actively Recruiting
10
Novartis Investigative Site
Bassano del Grappa, VI, Italy, 36061
Actively Recruiting
Research Team
N
Novartis Pharmaceuticals
N
Novartis Pharmaceuticals
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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