ClinicalTrials.gov record NCT07457151
Danicopan PMS in Korea
Danicopan Regulatory Post-Marketing Study in Korea
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 post-marketing observational registry follows people with paroxysmal nocturnal hemoglobinuria (PNH) in Korea who are treated with Danicopan added to eculizumab or ravulizumab. It collects safety information and measures effectiveness (hemoglobin, reticulocyte count, fatigue scores, and transfusion avoidance) through about 24 weeks. The study uses a total-surveillance approach in routine clinical practice.
What it is studying. The study is testing the safety profile of Danicopan when used as an add-on to a C5 inhibitor (eculizumab or ravulizumab) in routine care in Korea, including known and previously unsuspected adverse reactions. It is also evaluating effectiveness at 12 and 24 weeks using hemoglobin, absolute reticulocyte count, FACIT Fatigue scores, and whether patients remain free from red blood cell transfusions.
What the researchers hope to learn. To characterize the safety of Danicopan as add-on therapy to a C5 inhibitor in patients with PNH in Korea and to evaluate effectiveness measures (hemoglobin, reticulocyte count, fatigue, and transfusion avoidance) at 12 and 24 weeks.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
As part of a post-approval commitment, the Korean health authority requests a study to characterize safety and effectiveness in patients who are treated with Danicopan as an add-on to ravulizumab or eculizumab in normal clinical practice settings. This study is designed to assess the known safety profile or identify previously unsuspected adverse reactions and to evaluate the effectiveness of Danicopan under conditions of routine daily medical practice in Korea.
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Patients eligible for and treated with Danicpan as add-on therapy to a C5 inhibitor (Eculizumab or Ravulizumab) in patient with PNH in Korea
- Provision of a signed and dated written informed consent by the patient or their legally acceptable representative
Exclusion criteria
- Participation in any concurrent interventional trials during the period of study drug treatment
- Other off-label indications according to the approved label in Korea
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
The objectives of this study are to assess the safety and effectiveness of Danicopan in a real world setting in patients who are prescribed with the study drug under the approved indication in Korea. Primary objective(s) To assess the safety of Danicopan as add-on therapy to a C5 inhibitor (Eculizumab or Ravulizumab)in patients with PNH in Korea. Secondary objective(s) To assess effectiveness of Danicopan as add- on therapy to a C5 inhibitor (Eculizumab or Ravulizumab) in patients with PNH at 12 and 24 weeks.
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.
Participants are patients who start or are already being treated with Danicopan as an add-on to a C5 inhibitor (eculizumab or ravulizumab) in routine care and must provide written informed consent. Study staff will collect safety information (adverse events, serious adverse events, adverse drug reactions, and adverse events of special interest) for up to 24 weeks from the first dose of Danicopan. Effectiveness will be assessed at Week 12 and Week 24 (or at treatment end if earlier) using blood tests for hemoglobin and absolute reticulocyte count, a FACIT Fatigue questionnaire, and records of any red blood cell transfusions. The registry record does not list any invasive procedures (for example, biopsies or spinal taps).
How the study is structured. This is a non-interventional, observational registry (cohort) that collects data from patients prescribed Danicopan under the approved Korean label. Patient data, including adverse events, will be gathered for up to 24 weeks from first dose. Effectiveness measures are assessed at baseline, Week 12 and Week 24 (or at end of treatment if earlier). The study uses total-surveillance methods in routine clinical practice.
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
- Apr 10, 2026
- Primary completion
- Jun 27, 2028 (estimated)
- Study completion
- Jun 27, 2028 (estimated)
- Target enrollment
- 27 participants (estimated)
- Last updated on registry
- Aug 14, 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
- AstraZeneca
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.
- AstraZeneca Clinical Study Information Center 📞 1-877-240-9479 ✉ information.center@astrazeneca.com
Participating sites (4)
- Research SiteRecruiting
Busan · South Korea
- Research SiteRecruiting
Hwasun-gun · South Korea
- Research SiteRecruiting
Seoul · South Korea
- Research SiteActive Not Recruiting
Seoul · 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.
- 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.
- study drug
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- 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.
- Primary objective
- The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected.
- Secondary objective
- An additional result the study measures alongside the primary outcome, for example quality of life or a laboratory value.
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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