Paroxysmal Nocturnal Hemoglobinuria
Conditions
Keywords
PNH
Brief 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. 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 and 24 weeks.
Interventions
None listed
Sponsors
AstraZeneca
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety (adverse events (AEs), serious AEs (SAEs), adverse drug reactions (ADRs), serious ADRs (SADRs), unexpected AEs/ADRs), AESI | Patient data will be gathered for up to 24 weeks from the first dose of the study drug. | To assess the safety of Danicopan as add-on therapy to a C5 inhibitor (Eculizumab or Ravulizumab)in patients with PNH in Korea. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in hemoglobin (Hgb) at Week 12 and 24 | Effectiveness variables will be assessed at Week 12 and 24 or end of treatment (if treatment is discontinued before Week 24). | Hemoglobin (Hgb) will be summarized with descriptive statistics (mean, standard deviation, median, minimum, and maximum). |
| Change from baseline in absolute reticulocyte count (ARC) at Week 12 and 24 | Effectiveness variables will be assessed at Week 12 and 24 or end of treatment (if treatment is discontinued before Week 24) | Absolute reticulocyte count (ARC) will be summarized with descriptive statistics (mean, standard deviation, median, minimum, and maximum). |
| Change from baseline in FACIT Fatigue scores at Week 12 and 24 | Effectiveness variables will be assessed at Week 12 and 24 or end of treatment (if treatment is discontinued before Week 24) | FACIT Fatigue scores will be summarized with descriptive statistics (mean, standard deviation, median, minimum, and maximum). |
| Proportion of patients with transfusion avoidance* at Week 12 and 24 (* Transfusion Avoidance: Defined as remaining free from red blood cell transfusions) | Effectiveness variables will be assessed at Week 12 and 24 or end of treatment (if treatment is discontinued before Week 24) | 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. |
Countries
South Korea
Contacts
CONTACTAstraZeneca Clinical Study Information Center
Outcome results
None listed