The primary objective of this study is to evaluate efficacy of danicopan as add-on treatment to ravulizumab or eculizumab as assessed by hemoglobin (Hgb) change from Baseline at Week 12 in pediatric participants with paroxysmal nocturnal hemoglobinuria (PNH) and clinically significant extravascular hemolysis (CS-EVH).
Confirmed diagnosis of PNH.
CS-EVH defined by: Anemia: Hgb ≤ 11.0 g/dL, and absolute reticulocyte count ≥ 100 × 109/L
Treated with ravulizumab or eculizumab for at least 12 weeks immediately preceding Day 1, the dose received should be stable during this period, and there should be no anticipated changes in dosage or interval during the first 12 weeks of this study.
all participants must be vaccinated against meningococcal infection from serogroups A, C, W, and Y and serogroup B within 3 years prior to, or at least 14 days prior to Day 1
Platelet count < 30000/μL or there is a need for platelet transfusions.
ANC < 500/μL.
ALT > 2 × ULN or ALT > 3 × ULN for participants with documented liver iron overload defined by serum ferritin values ≥ 500 ng/mL.
Direct bilirubin > 2 × ULN, unless, in the Investigator's opinion, is due to hemolysis or Gilbert's syndrome based on medical history.
Single group
Danicopan
DrugParticipants will receive danicopan on a weight-based dosing regimen.
Change From Baseline in Hemoglobin (Hgb) Concentration at Week 12
Maximum Plasma Concentration (Cmax) of Danicopan
Number of Participants With Transfusion Avoidance Through Weeks 12 and 24
Change From Baseline in Absolute Reticulocyte Count at Weeks 12 and 24
Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Generic Core Scales Score at Weeks 12 and 24
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Alexion Pharmaceuticals, Inc.
Lead sponsor
AstraZeneca
Collaborator
