Danicopan PMS in Korea
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Paroxysmal Nocturnal Hemoglobinuria. Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- South Korea
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Danicopan Regulatory Post-Marketing Study in Korea
Overview
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.
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 weeks.
Primary outcome measures
- Safety (adverse events (AEs), serious AEs (SAEs), adverse drug reactions (ADRs), serious ADRs (SADRs), unexpected AEs/ADRs), AESI [Time frame: Patient data will be gathered for up to 12 weeks from the first dose of the study drug.]
Secondary outcome measures (4)
- Change from baseline in hemoglobin (Hgb) at Week 12 [Time frame: Effectiveness variables will be assessed at Week 12 or end of treatment (if treatment is discontinued before Week 12).]
- Change from baseline in absolute reticulocyte count (ARC) at Week 12 [Time frame: Effectiveness variables will be assessed at Week 12 orend of treatment (if treatment is discontinued before Week 12)]
- Change from baseline in FACIT Fatigue scores at Week 12 [Time frame: Effectiveness variables will be assessed at Week 12 orend of treatment (if treatment is discontinued before Week 12)]
- Proportion of patients with transfusion avoidance* at Week 12(* Transfusion Avoidance: Defined as remaining free from red blood cell transfusions) [Time frame: Effectiveness variables will be assessed at Week 12 orend of treatment (if treatment is discontinued before Week 12)]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
South Korea · 4 centers
- Research Site — Busan
- Research Site — Hwasun-gun
- Research Site — Seoul
- Research Site — Seoul
Identifiers
NCT: NCT07457151 · D7332R00002