Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)

Más betegség

• History of a major organ transplant or hematopoietic stem cell transplantation (HSCT)
• Participants with known aplastic anemia or other bone marrow failure that requires HSCT or other therapies including anti-thymocyte globulin and/or immunosuppressants
• Evidence of human immunodeficiency virus, hepatitis B, or active hepatitis C infection at screening

Laborértékek

• Laboratory abnormalities at screening, including:
  • Alanine aminotransferase \>2 x ULN (\>3 x ULN in case of patients with documented liver iron overload defined by serum ferratin values
  • 500 ng/ML)
  • Direct bilirubin \>2 x ULN (unless due to EVH or documented Gilbert's Syndrome)

Egyéb

• Known or suspected complement deficiency
• Current evidence of biliary cholestasis
• Estimated glomerular filtration rate of \<30 milliliters/minute/1.73 meter squared and/or are on dialysis

• Diagnosis of PNH
• Clinically Evident EVH defined by:
  • Anemia (Hgb ≤9.5 gram/deciliter) with absolute reticulocyte count ≥120 x 10\^9/liter
• Receiving an approved C5 inhibitor for at least 6 months prior to Day 1
• Platelet count ≥30,000/microliters (µL)
• Absolute neutrophil counts ≥500/μL
• Documentation of/or willingness to receive vaccinations for N. meningiditis and prophylactic antibiotics as required