Last Update: Jun 25, 2026 REAL-CARE: Real-world Effectiveness of iptacopAn in itaLian Patients With Paroxysmal noCturnAl HemoglobinuRia: an Observational Study ClinicalTrials.gov Identifier: [NCT07229235](https://clinicaltrials.gov/ct2/show/NCT07229235) Novartis Reference Number:CLNP023C1IT01 [See if you Pre-qualify](#trial-eligibility "See if you Pre-qualify") All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. ## Study Description 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. This is a local, multicenter, observational study with retrospective, retro-prospective and prospective cohorts. Adult patients with PNH either naïve to iptacopan or transitioning from the Managed Access Program (MAP) starting from January 2023 will be enrolled. Primary data will be collected prospectively; secondary data will be retrieved retrospectively from patients previously treated under MAP. A mix of primary and secondary data will be collected in retro-prospective patients. Patients will be indexed on the date of iptacopan initiation and will be followed up for 24 months or until iptacopan discontinuation (due to occurrence of an adverse event, lack of efficacy etc), clinical decision, death, administrative issues, consent withdrawal or loss to follow-up, whichever comes first. The enrollment period is planned to last 15 months, with potential adjustments based on enrollment progress. No treatment decision will be influenced by study participation. No extra visits, examinations, or procedures are imposed. Questionnaires should be collected at the indicated timepoints whenever possible, but, since the administration of the questionnaires represents an additional procedure not included in routine clinical care, these timepoints are considered indicative only, and patients will not be recalled specifically to complete questionnaires if a routine clinical visit is not scheduled. Condition Paroxysmal Nocturnal Hemoglobinuria (PNH) Overall Status Recruiting Number of Participants50 Start Date Jan 13, 2026 Completion Date Aug 31, 2028 Gender All Age(s) 18 Years - (Adult, Older Adult) ## 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). Italy #### Novartis Investigative Site Recruiting Alessandria,AL,15121,Italy #### Novartis Investigative Site Recruiting Avellino,AV,83100,Italy #### Novartis Investigative Site Recruiting Brescia,BS,25123,Italy #### Novartis Investigative Site Recruiting Florence,FI,50134,Italy #### Novartis Investigative Site Recruiting Palermo,PA,90146,Italy #### Novartis Investigative Site Recruiting Roma,RM,00161,Italy #### Novartis Investigative Site Recruiting Bassano del Grappa,VI,36061,Italy #### Novartis Investigative Site Recruiting Milan,MI,20132,Italy #### Novartis Investigative Site Recruiting Roma,RM,00168,Italy #### Novartis Investigative Site Recruiting Torino,TO,10126,Italy #### Novartis Investigative Site Recruiting Genova,GE,16132,Italy #### Novartis Investigative Site Recruiting Milan,MI,20122,Italy ## Worldwide Contacts If the location of your choosing does not feature any contact detail, please reach out using the information below. #### Novartis Pharmaceuticals Phone: [ +41613241111](tel:+41613241111) Email: