ClinicalTrials.gov record NCT07416162
A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy
A Post-marketing Surveillance of Fabhalta® (Iptacopan) in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) or C3 Glomerulopathy (C3G)
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.
This is a post‑marketing observational study collecting medical‑record data on adults treated with iptacopan for PNH or C3G. It aims to record adverse events and clinical/laboratory measures during routine care, with key assessments at baseline and around Week 24 and safety follow-up up to 2 years. Participants are grouped into separate PNH and C3G cohorts.
What it is studying. The study is collecting real‑world safety and effectiveness information about iptacopan when used in routine care for PNH and C3G. It measures the incidence of adverse events, serious adverse events, and adverse drug reactions, and records blood and kidney‑related laboratory measures such as hemoglobin, LDH, creatinine, estimated GFR, and urine protein, plus investigators' overall assessments of improvement.
What the researchers hope to learn. To describe the safety profile of iptacopan by measuring rates of adverse events, serious adverse events, and adverse drug reactions; to collect clinical and laboratory measures to evaluate effects on blood counts, hemolysis markers, kidney function, and proteinuria; and to summarize investigators' assessments of overall clinical change.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
This is a post-marketing surveillance study conducted as part of the Risk Management Plan (RMP) for South Korea, to evaluate the safety and effectiveness of iptacopan in real-world clinical settings for the treatment of either PNH or C3G in Korean patients. Prospective data will be collected from patient medical records to address the objectives for all eligible populations.
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Patients aged 18 years or older who have been diagnosed with PNH or C3G.
- Patients who have received vaccination in accordance with the approved Korean labeling prior to initiating treatment with iptacopan.
- Patients who are being treated or will be treated with iptacopan in accordance with the approved Korean labeling.
- Patients who have voluntarily provided consent for study participation (written informed consent).
Exclusion criteria
- Patients who fall under the contraindications for iptacopan administration according to approved Korean labeling.
- Patients for whom iptacopan administration is deemed inappropriate based on the investigator's judgment.
Study description and what participation involves
The registry record has no detailed description beyond the summary above.
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.
You would provide written consent and allow the study to collect prospective information from your medical record about your iptacopan treatment. The study records routine clinical measurements such as blood tests at baseline and Week 24, a random spot urine protein/creatinine ratio at Week 24, and an investigator assessment of overall improvement at the last visit. Safety information, including adverse events, will be collected for up to two years. The registry does not specify any additional invasive procedures beyond routine clinical care.
How the study is structured. A prospective observational cohort study using data from patients' medical records. People who are treated with iptacopan as part of routine care are followed; there are two observational cohorts (PNH and C3G). No study treatment is assigned—care follows the approved product labeling—and safety events are collected for up to 2 years with specific lab assessments at baseline and about Week 24.
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 South Korea. 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
- Jun 15, 2026
- Primary completion
- Feb 1, 2029 (estimated)
- Study completion
- Feb 1, 2029 (estimated)
- Target enrollment
- 21 participants (estimated)
- Last updated on registry
- Jun 26, 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 (3)
- Novartis Investigative SiteRecruiting
Jeollanam, 519763 · South Korea
- Novartis Investigative SiteRecruiting
Seoul, 03080 · South Korea
- Novartis Investigative SiteRecruiting
Seoul, 06591 · South Korea
Terms used on this page · explanations by MedicalUpdates.ai
- written 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.
- contraindications
- A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
- 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.
- Exclusion criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
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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