Danicopan PMS in Korea
ConditionParoxysmal Nocturnal Hemoglobinuria
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorAstraZeneca
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
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 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.
Trial design
Design model
Cohort
Time perspective
Other
Treatments tested in this trial
Not listed
Trial groups
No trial groups listed
Trial outcomes
Primary outcomes
1
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.
Time frame
Patient data will be gathered for up to 24 weeks from the first dose of the study drug.Secondary outcomes
1
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).2
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)3
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)4
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.
Time frame
Effectiveness variables will be assessed at Week 12 and 24 or end of treatment (if treatment is discontinued before Week 24)Sponsors and contacts
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Source ClinicalTrials.gov