Danicopan added to ravulizumab or eculizumab for PNH in Korea

Danicopan Regulatory Post-Marketing Study in Korea

This will see if adding Danicopan to ravulizumab or eculizumab is safe and helps people with PNH who are treated in routine care in Korea.

Quick facts

• Study type: Observational
• Enrollment: 8 (estimated)
• Sex: All
• Sponsor: AstraZeneca (Industry-sponsored)
• Drugs / interventions: Eculizumab, Ravulizumab
• Locations: 4 sites (Busan and 3 other locations)
• Trial ID: NCT07457151 on ClinicalTrials.gov

What this trial studies

This is a post-approval, observational program in Korea to collect real-world safety and effectiveness data on Danicopan when used as an add-on to C5 inhibitors (eculizumab or ravulizumab) for patients with paroxysmal nocturnal hemoglobinuria. Patients prescribed Danicopan in routine clinical practice at participating sites will be followed and data on adverse events, hemoglobin levels, transfusion need, and other effectiveness measures at around 12 weeks will be recorded. The study is non-interventional and designed to detect known safety signals or previously unrecognized adverse reactions under normal care conditions. Results will inform local post-marketing safety and usage patterns for Danicopan in Korea.

Who should consider this trial

Good fit: Patients with PNH in Korea who are eligible for and prescribed Danicopan as an add-on to eculizumab or ravulizumab and who provide informed consent are ideal candidates.

Not a fit: Patients not receiving Danicopan, those on other off-label indications, or those enrolled in concurrent interventional trials are unlikely to benefit from participation.

Why it matters

Potential benefit: If successful, this could confirm that adding Danicopan improves anemia control and reduces transfusion needs for some PNH patients already on a C5 inhibitor.

How similar studies have performed: Prior clinical trials of danicopan have shown improvements in hemolysis and hemoglobin as an add-on to C5 inhibitors, but real-world data in Korean clinical practice are limited.

Eligibility criteria

Inclusion Criteria:

1. Patients eligible for and treated with Danicpan as add-on therapy to a C5 inhibitor (Eculizumab or Ravulizumab) in patient with PNH in Korea
2. Provision of a signed and dated written informed consent by the patient or their legally acceptable representative

Exclusion Criteria:

1. Participation in any concurrent interventional trials during the period of study drug treatment
2. Other off-label indications according to the approved label in Korea

Where this trial is running

Busan and 3 other locations

• Research Site — Busan, South Korea (Recruiting)
• Research Site — Daegu, South Korea (Not_yet_recruiting)
• Research Site — Hwasun-gun, South Korea (Not_yet_recruiting)
• Research Site — Seoul, South Korea (Not_yet_recruiting)

Study contacts

• Study coordinator: AstraZeneca Clinical Study Information Center

Conditions Paroxysmal Nocturnal Hemoglobinuria; PNH