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.
Status:
Recruiting
Associated Drug(s):
Phase:
Phase 3
Gender:
Female
Male
Age Group:
18 years and older
Accepts Healthy Volunteers:
No
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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