ALXN1830 in Patients With Warm Autoimmune Hemolytic Anemia

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Study of ALXN1830 in Patients With Warm Autoimmune Hemolytic Anemia

The main objective of the study is to evaluate the safety and efficacy of ALXN1830 compared to placebo in adult participants with warm autoimmune hemolytic anemia (WAIHA).

This study will consist of a 4-week Screening period, 13-week Primary Treatment period, and optional Extended Treatment period (up to 2 years).

Key Inclusion Criteria:

• Primary or secondary WAIHA, diagnosed at least 6 weeks prior to Screening
• Failed or not tolerated at least 1 prior WAIHA treatment regimen, for example, corticosteroids, rituximab, azathioprine, cyclophosphamide, cyclosporine, mycophenolate mofetil, danazol, or vincristine
• Hemoglobin < 10 g/dL and ≥ 6 g/dL
• Positive direct antiglobulin test (Coombs) (IgG positive, complement C3 [C3] positive or negative)
• Evidence of active hemolysis including any of the following: a) Lactate dehydrogenase (LDH) > upper limit of normal (ULN), b) Haptoglobin < lower limit of normal (LLN), c) Indirect bilirubin > ULN
• Total IgG > 500 mg/dL

Key Exclusion Criteria:

• Human immunodeficiency virus (HIV) infection (positive HIV-1 or HIV-2 antibody test)
• Positive hepatitis B surface antigen (HBsAg) or hepatitis C antibody test