This is a single-arm long-term extension study that will enroll participants with PNH who have completed participation in Alexion-sponsored clinical studies with danicopan as an add on therapy to a C5i.
The total duration of the study will be up to 3 years. Eligible participants must complete all study assessments on the parent protocol before starting this study. All participants entering this study will receive danicopan as an add-on to a background C5i therapy. The only allowed C5i therapies are eculizumab and ravulizumab.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
80
Oral tablet
Participants Experiencing Treatment-emergent Adverse Events (TEAEs) And Serious TEAEs
Time frame: Up to 3 years
Change In Hemoglobin Values
Time frame: Up to 3 years
Proportion of patients with Hgb increase ≥ 2g/dL in the absence of transfusion over time
Time frame: Up to 3 years
Change In Absolute Reticulocyte Count
Time frame: Up to 3 years
Change In Lactate Dehydrogenase (LDH)
Time frame: Up to 3 years
Proportion Of Participants With LDH ≤ 1.5 × Upper Limit Of Normal
Time frame: Up to 3 years
Proportion Of Participants With Transfusion Avoidance
Time frame: Up to 3 years
Change In Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scores
Total scores range from 0 to 52, with higher scores indicating better QoL.
Time frame: Up to 3 years
Change In The European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Core 30 Scale (QLQ-C30) Scores
Thirty questions related to QoL, with the first 28 questions scored on a 4-point scale (1 = not at all to 4 = very much) and the final 2 questions that probe the participant's overall health and QoL scored on a scale of 1 (very poor) to 7 (excellent).
Time frame: Up to 3 years
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Research Site
Los Angeles, California, United States
Research Site
Chicago, Illinois, United States
Research Site
Baltimore, Maryland, United States
Research Site
Kansas City, Missouri, United States
Research Site
Long Island City, New York, United States
Research Site
Cleveland, Ohio, United States
Research Site
Dallas, Texas, United States
Research Site
Porto Alegre, Brazil
Research Site
Rio de Janeiro, Brazil
Research Site
Toronto, Ontario, Canada
...and 35 more locations
Participants Experiencing TEAEs Leading To Discontinuation
Time frame: Up to 3 years