Status and phase
Conditions
Treatments
Study type
Funder types
Identifiers
About
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).
Enrollment
Sex
Ages
Volunteers
Inclusion criteria
Exclusion criteria
Platelet count < 30000/μL or there is a need for platelet transfusions.
ANC < 500/μL.
Clinically significant laboratory abnormalities related to liver function, including:
Current evidence of biliary cholestasis.
Known aplastic anemia or other bone marrow failure that requires HSCT or other therapies, including anti-thymocyte globulin and immunosuppressants unless the dosage of immunosuppressant has been stable for at least 12 weeks before Day 1 and is expected to remain stable through Week 12.
History of a major organ transplant (eg, heart, lung, kidney, liver) or HSCT.
Known or suspected complement deficiency.
Active bacterial or viral infection, a body temperature > 38°C on 2 consecutive daily measures, evidence of other infection, or history of any febrile illness within 14 days prior to first study intervention administration.
Primary purpose
Allocation
Interventional model
Masking
6 participants in 1 patient group
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Central trial contact
Alexion Pharmaceuticals, Inc. (Sponsor)
Data sourced from clinicaltrials.gov
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