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).
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
6
Participants will receive danicopan on a weight-based dosing regimen.
Research Site
Saskatoon, Saskatchewan, Canada
RECRUITINGResearch Site
Paris, France
RECRUITINGResearch Site
Leeds, United Kingdom
RECRUITINGResearch Site
London, United Kingdom
RECRUITINGChange From Baseline in Hemoglobin (Hgb) Concentration at Week 12
Time frame: Baseline, Week 12
Maximum Plasma Concentration (Cmax) of Danicopan
Time frame: Day 1 up to Week 12
Number of Participants With Transfusion Avoidance Through Weeks 12 and 24
Time frame: Weeks 12 and 24
Change From Baseline in Absolute Reticulocyte Count at Weeks 12 and 24
Time frame: Baseline, Weeks 12 and 24
Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Generic Core Scales Score at Weeks 12 and 24
Time frame: Baseline, Weeks 12 and 24
Change from Baseline in Pediatric Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Weeks 12 and 24
Time frame: Baseline, Weeks 12 and 24
Acceptability and Palatability Questionnaire Score
Time frame: Week 2
Change from Baseline in Hgb Concentration at Week 24
Time frame: Baseline, Week 24
Change from Baseline in Serum Alternative Pathway (AP) Activity
Time frame: Baseline up to Week 64
Change from Baseline in Plasma Bb Concentrations
Time frame: Baseline up to Week 64
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