Paroxysmal Nocturnal HemoglobinuriaNCT07457151
Danicopan PMS in Korea
An observational study of Paroxysmal Nocturnal Hemoglobinuria, run by AstraZeneca at 4 sites in South Korea.
Trial summary
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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- How many people
- 27 participants
- Where
- 4 sitesSouth Korea
- Study dates
- 10 Apr 2026to 27 Jun 2028
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients currently treated with or initiating the study drug under approved local label and conditions of routine daily medical practice in Korea will be included in the study. This study will be conducted using a total surveillance method to investigate the safety and effectiveness of all patients being treated with the study drug.
Who may be able to join
- 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
Who cannot join
- Participation in any concurrent interventional trials during the period of study drug treatment
- Other off-label indications according to the approved label in Korea
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
AstraZeneca Clinical Study Information Center contact
South Korea4 sites, 3 recruiting
- Recruiting
Research Site
Busan
- Recruiting
Research Site
Hwasun-gun
- Recruiting
Research Site
Seoul
- Active, not recruiting
Research Site
Seoul
About this study
Official title: Danicopan Regulatory Post-Marketing Study in Korea
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.
Read the detailed description
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.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- PNH
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety (adverse events (AEs), serious AEs (SAEs), adverse drug reactions (ADRs), serious ADRs (SADRs), unexpected AEs/ADRs), AESI
To assess the safety of Danicopan as add-on therapy to a C5 inhibitor (Eculizumab or Ravulizumab)in patients with PNH in Korea.
Measured: Patient data will be gathered for up to 24 weeks from the first dose of the study drug.
Secondary outcomes (4)
Change from baseline in hemoglobin (Hgb) at Week 12 and 24
Hemoglobin (Hgb) will be summarized with descriptive statistics (mean, standard deviation, median, minimum, and maximum).
Measured: Effectiveness variables will be assessed at Week 12 and 24 or end of treatment (if treatment is discontinued before Week 24).
Change from baseline in absolute reticulocyte count (ARC) at Week 12 and 24
Absolute reticulocyte count (ARC) will be summarized with descriptive statistics (mean, standard deviation, median, minimum, and maximum).
Measured: Effectiveness variables will be assessed at Week 12 and 24 or end of treatment (if treatment is discontinued before Week 24)
Change from baseline in FACIT Fatigue scores at Week 12 and 24
FACIT Fatigue scores will be summarized with descriptive statistics (mean, standard deviation, median, minimum, and maximum).
Measured: Effectiveness variables will be assessed at Week 12 and 24 or end of treatment (if treatment is discontinued before Week 24)
Proportion of patients with transfusion avoidance* at Week 12 and 24 (* Transfusion Avoidance: Defined as remaining free from red blood cell transfusions)
The proportion of patients with transfusion avoidance will be summarized. Patients with transfusion avoidance will be considered to show effectiveness and the number and percentage of subjects corresponding to this classification will be presented, along with the 95% confidence interval (CI) for the percentage calculated using the Clopper-Pearson method.
Measured: Effectiveness variables will be assessed at Week 12 and 24 or end of treatment (if treatment is discontinued before Week 24)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Mar 2026
- Study start10 Apr 2026
- Primary completion27 Jun 2028 (estimated)
- Study completion27 Jun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies of the same condition, open and under way.
- More trials run by AstraZenecaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 27 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AstraZeneca
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07457151
- Sponsor's protocol ID
- D7332R00002
- Status last verified
- Oct 2026
- First submitted
- 13 Feb 2026
- Last updated
- 13 Oct 2026
- Results posted
- No
- Individual data sharing
- Yes: Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.