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.
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.
Study Type
OBSERVATIONAL
Enrollment
27
Research Site
Busan, South Korea
RECRUITINGResearch Site
Hwasun-gun, South Korea
RECRUITINGResearch Site
Seoul, South Korea
RECRUITINGSafety (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.
Time frame: Patient data will be gathered for up to 24 weeks from the first dose of the study drug.
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).
Time frame: 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).
Time frame: 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).
Time frame: 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.
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Research Site
Seoul, South Korea
ACTIVE_NOT_RECRUITINGTime frame: Effectiveness variables will be assessed at Week 12 and 24 or end of treatment (if treatment is discontinued before Week 24)