Study of the Efficacy and Safety of Parsaclisib in Participants With Primary Warm Autoimmune Hemolytic Anemia (PATHWAY)
Warm Autoimmune Hemolytic Anemia (wAIHA)
About this trial
This is an interventional treatment trial for Warm Autoimmune Hemolytic Anemia (wAIHA) focused on measuring Warm Autoimmune Hemolytic Anemia (wAIHA)
Eligibility Criteria
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.
Sites / Locations
- Investigative Site US005
- Investigative Site US004
- Investigative Site US006
- Investigative Site US012
- Investigative Site US007
- Investigative Site US002
- Investigative Site US003
- Investigative Site US009
- Investigative Site US010
- Investigative Site US001
- Investigative Site AT002
- Investigative Site AT001
- Investigative Site BE001
- Investigative Site BE002
- Investigative Site CA001
- Investigative Site FR002
- Investigative Site FR003
- Investigative Site FR001
- Investigative Site DE001
- Investigative Site DE002
- Investigative Site IL002
- Investigative Site IL001
- Investigative Site IT003
- Investigative Site IT002
- Investigative Site IT001
- Investigative Site IT004
- Investigative Site IT006
- Investigative Site IT005
- Investigative Site JP008
- Investigative Site JP004
- Investigative Site JP006
- Investigative Site JP009
- Investigative Site JP002
- Investigative Site JP010
- Investigative Site JP005
- Investigative Site JP007
- Investigative Site JP001
- Investigative Site JP003
- Investigative Site NL001
- Investigative Site PL001
- Investigative Site PL006
- Investigative Site PL003
- Investigative Site PL005
- Investigative Site PL004
- Investigative Site PL002
- Investigative Site ES006
- Investigative Site ES001
- Investigative Site ES003
- Investigative Site ES005
- Investigative Site ES004
- Investigative Site ES002
- Investigative Site GB002
- Investigative Site GB006
- Investigative Site GB003
- Investigative Site GB004
- Investigative Site GB005
Arms of the Study
Arm 1
Arm 2
Experimental
Placebo Comparator
Group A: Parsaclisib
Group B: Placebo followed by Parsaclisib
Participants will receive parsaclisib for 24 weeks (double-blind period). Participant who completed the double-blind period and tolerating the study treatment upon investigator's opinion will continue into open-label period for an additional 24 weeks. Participants may then continue to receive parsaclisib in a long-term extension period.
Participants will receive placebo for 24 weeks (double-blind period). Participants who completed the double-blind period will receive parsaclisib in the 24 week open-label period. Participants may then continue to receive parsaclisib in a long-term extension period.