Phase 3 Study of Danicopan Added to Ravulizumab or Eculizumab for Children Aged 12 to 17 With Paroxysmal Nocturnal Hemoglobinuria and Significant Extravascular Hemolysis

What this Trial Is About

Researchers are evaluating danicopan as an add-on treatment to ravulizumab or eculizumab in pediatric participants aged 12 to 17 years who have paroxysmal nocturnal hemoglobinuria PNH with clinically significant extravascular hemolysis CS-EVH. The main goal is to assess the change in hemoglobin levels from the start of the study to Week 12, focusing on how adding danicopan affects this blood condition. Participants receive danicopan based on their weight during a 12-week open-label treatment period, followed by up to a one-year open-label long-term extension. The study does not include randomization or blinding, and all participants continue their stable dose of ravulizumab or eculizumab throughout the trial. During the study, researchers monitor hemoglobin changes, reticulocyte counts, plasma danicopan levels, quality of life, fatigue, and other blood markers at various points up to Week 64. Safety and tolerability are followed closely through laboratory tests and questionnaires. The total participation includes the initial 12-week treatment and the optional extended follow-up period.

Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis