NCT07229235Recruiting
REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
REAL-CARE: Real-world Effectiveness of iptacopAn in itaLian Patients With Paroxysmal noCturnAl HemoglobinuRia: an Observational Study
Source: ClinicalTrials.gov · Last updated 25 Jun 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 50 (estimated)
- First submitted
- 30 Oct 2025
- Start date
- 13 Jan 2026
- Primary completion date
- 31 Aug 2028
- Lead sponsor
- Novartis Pharmaceuticals
- NCT number
- NCT07229235
What you commit to
- Study type: Observational
- Planned participants: 50
- From 13 Jan 2026 to the primary completion date, 31 Aug 2028
- 10 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 1.Concomitant participation in an interventional clinical study related to PNH or its treatment.
Other
- No other inclusion/exclusion criteria apply other than the requirements stated in the Summary of Product Characteristics (SmPC).
Who the study is looking for
- 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))
Age in the registry: 18 years to - · Sex: All · Does not accept healthy volunteers
Sites (10)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Novartis Investigative Site Coordinator of this site? Confirm its status | Alessandria | Italy | Recruiting |
| Novartis Investigative Site Coordinator of this site? Confirm its status | Avellino | Italy | Recruiting |
| Novartis Investigative Site Coordinator of this site? Confirm its status | Bassano del Grappa | Italy | Recruiting |
| Novartis Investigative Site Coordinator of this site? Confirm its status | Brescia | Italy | Recruiting |
| Novartis Investigative Site Coordinator of this site? Confirm its status | Florence | Italy | Recruiting |
| Novartis Investigative Site Coordinator of this site? Confirm its status | Genova | Italy | Recruiting |
| Novartis Investigative Site Coordinator of this site? Confirm its status | Milan | Italy | Recruiting |
| Novartis Investigative Site Coordinator of this site? Confirm its status | Palermo | Italy | Recruiting |
| Novartis Investigative Site Coordinator of this site? Confirm its status | Roma | Italy | Recruiting |
| Novartis Investigative Site Coordinator of this site? Confirm its status | Torino | Italy | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.