Study of the Efficacy and Safety of Parsaclisib in Participants With Primary Warm Autoimmune Hemolytic Anemia

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy and Safety of Parsaclisib in Participants With Primary Warm Autoimmune Hemolytic Anemia

The purpose of this study is to evaluate the efficacy and safety of parsaclisib compared with placebo in participants with Primary Warm Autoimmune Hemolytic Anemia (wAIHA),

Prospective participants must have primary wAIHA as well as other protocol-defined criteria. After participants have been determined to be eligible for the study, they will be randomized to 2:1, with stratification factor of corticosteroid dose and hemoglobin (Hgb <9 g/dL or ≥ 9 g/dL). Once a participant has completed the week 24 assessments in the double-blind period, the participant will have the opportunity to receive parsaclisib in the open-label treatment which will last up to another 24 weeks. Participants may then continue to receive parsaclisib in a long-term extension period.

Inclusion Criteria:

• Diagnosis of primary warm AIHA.
• Participants who have at least 1 unsuccessful prior therapy for warm AIHA or unable to receive or tolerate other therapies.
• Hemoglobin ≥ 6.5 to < 10 g/dL with symptoms of anemia at screening.
• FACIT-F score ≤ 43 at screening.
• Willingness to avoid pregnancy or fathering children.
• Willingness to receive PJP prophylaxis.
• Further inclusion criteria apply.

Exclusion Criteria:

• Women who are pregnant, breastfeeding or who are planning a pregnancy.
• Diagnosis of other types of AIHA (CAD, cold agglutinin syndrome, mixed-type AIHA or paroxysmal cold hemoglobinuria).
• Secondary warm AIHA from any cause or diagnosis of Evans syndrome.
• Splenectomy less than 3 months before randomization.
• Participants with a history or ongoing significant illness as assessed by the investigator.
• Participants with a current of medical history of a malignancy within the past 5 years except basal or squamous cell skin cancer that has been removed and considered cured, or superficial bladder cancer, prostate intraepithelial neoplasm, carcinoma in situ of the cervix, or other noninvasive or indolent malignancy.
• Participants know to be infected with HIV, Hepatitis B, or hepatitis C.
• Chronic or current active infectious disease requiring systemic antibiotics, antifungal, or antiviral treatment or exposure to a live vaccine.
• Participants with laboratory values outside of the protocol defined ranges.
• Further exclusion criteria apply.