ClinicalTrials.gov record NCT07229235
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
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
An observational study collecting real-world data on adults with PNH in Italy who start iptacopan or transitioned from the Managed Access Program (MAP) from January 2023. The study follows patients for up to 24 months after iptacopan start and uses a mix of retrospective, retro‑prospective and prospective data. No extra visits or procedures are imposed beyond routine care; questionnaires are collected when possible.
What it is studying. The study assesses hematologic response after starting iptacopan (notably change in hemoglobin at 12 months) and the proportion of patients who avoid red blood cell transfusions from Day 14 through Month 12. It also collects safety data (adverse events, infections, breakthrough hemolysis, vascular events), other lab changes, health-related quality of life, medication adherence, and health resource use over 12 and 24 months.
What the researchers hope to learn. To describe long-term hematologic response after iptacopan initiation (including change in hemoglobin at 12 and 24 months), the proportion of patients remaining transfusion‑free, safety outcomes (adverse events, serious adverse events, infections, breakthrough hemolysis, major adverse vascular events), changes in lab measures, patient-reported outcomes (PROMIS-29+2, WPAI:SHP), medication adherence (MARS-5), and healthcare resource use in real-world clinical practice.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- 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))
Exclusion criteria
- 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).
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
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.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
The study collects routine medical records and test results from the date iptacopan treatment starts and follows patients for up to 24 months or until iptacopan is stopped, death, withdrawal of consent, or loss to follow-up. No extra visits, examinations, or procedures are imposed beyond usual care. Questionnaires about quality of life, medication adherence, and work productivity may be requested at certain timepoints but patients will not be recalled specifically to complete them. The study will record treatment dates, laboratory and clinical results, transfusion history, and any adverse events from routine care.
How the study is structured. A local, multicenter observational cohort study using retrospective, retro‑prospective and prospective cohorts. Patients are indexed at the date they start iptacopan and followed for up to 24 months or until treatment stops or other study‑defined endpoints occur. Primary data are collected prospectively while earlier MAP-treated patients contribute retrospective data. No study-driven treatments or extra clinic visits are required; questionnaires are optional and collected at routine visits when possible.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
- The listed sites are in Italy. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on paroxysmal nocturnal hemoglobinuria
Important dates
- Study start
- Jan 13, 2026
- Primary completion
- Aug 31, 2028 (estimated)
- Study completion
- Aug 31, 2028 (estimated)
- Target enrollment
- 50 participants (estimated)
- Last updated on registry
- Jun 25, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Novartis Pharmaceuticals
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Novartis Pharmaceuticals 📞 +41613241111 ✉ novartis.email@novartis.com
- Novartis Pharmaceuticals
Participating sites (12)
- Novartis Investigative SiteRecruiting
Alessandria, AL, 15121 · Italy
- Novartis Investigative SiteRecruiting
Avellino, AV, 83100 · Italy
- Novartis Investigative SiteRecruiting
Brescia, BS, 25123 · Italy
- Novartis Investigative SiteRecruiting
Florence, FI, 50134 · Italy
- Novartis Investigative SiteRecruiting
Genova, GE, 16132 · Italy
- Novartis Investigative SiteRecruiting
Milan, MI, 20122 · Italy
- Novartis Investigative SiteRecruiting
Milan, MI, 20132 · Italy
- Novartis Investigative SiteRecruiting
Palermo, PA, 90146 · Italy
- Novartis Investigative SiteRecruiting
Roma, RM, 00161 · Italy
- Novartis Investigative SiteRecruiting
Roma, RM, 00168 · Italy
Terms used on this page · explanations by MedicalUpdates.ai
- primary objective
- The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected.
- informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- interventional
- A study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
- exclusion criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- Inclusion criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- adverse event
- Any unwanted medical problem that happens during a study, whether or not it was caused by the treatment. A 'serious' adverse event is one that meets specific criteria, such as causing death, being life-threatening, requiring or prolonging hospital admission, causing significant disability, or another medically important outcome.
- follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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