Instructions
If you are interested in learning more about your possible participation in this clinical trial, please complete the form. Your information will be forwarded directly to the sponsoring company.
- Diagnosis of PNH
- Clinically Evident EVH defined by:
- Anemia (Hgb ≤9.5 gram/deciliter) with absolute reticulocyte count ≥120 x 10^9/liter
- At least 1 packed red blood cell or whole blood transfusion within 6 months prior to the start of the study
- Receiving an approved C5 inhibitor (eculizumab or ravulizumab) for at least 6 months prior to study Day 1
- Platelet count ≥30,000/microliters (µL)
- Absolute neutrophil counts ≥750/μL
- Documentation of/or willingness to receive vaccinations for N. meningiditis and prophylactic antibiotics as required
- 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
- Known or suspected complement deficiency
- Laboratory abnormalities at screening, including:
- Alanine aminotransferase >2 x ULN
- Direct bilirubin >2 x ULN (unless due to EVH or documented Gilbert's Syndrome)
- Known underlying bleeding disorders (eg, coagulation factor deficiencies, idiopathic thrombocytopenic purpura, Von Willebrand disease, etc.) or any conditions leading to anemia that are not primarily due to PNH.
- Current evidence of biliary cholestasis
- Estimated glomerular filtration rate <30 milliliters/minute/1.73 meter squared and/or are on dialysis
- Evidence of human immunodeficiency virus, hepatitis B, or active hepatitis C infection at screening