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

JRCT ID: jRCT2051210140

Registered date:24/12/2021

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Warm Autoimmune Hemolytic Anemia (wAIHA)
• Date of first enrollment: 30/12/2021
• Target sample size: 100
• Countries of recruitment: U.S.A,Japan,Austria,Japan,Belgium,Japan,Canada,Japan,France,Japan,Germany,Japan,Israel,Japan,Italy,Japan,Netherlands,Japan,Poland,Japan,Spain,Japan,United Kingdom,Japan
• Study type: Interventional

Intervention(s)

Group A: Parsaclisib (INCB050465) will be administered QD orally.
Group B: Placebo will be administered QD orally.

Outcome(s)

• Primary Outcome: Proportion of participants attaining a durable hemoglobin response

Secondary Outcome

1. Proportion of participants with a >= 3-point increase in FACIT-F score
2. Proportion of participants with a 50 m increase in a 6MWT
3. Change in FACIT-F score
4. Change in hemoglobin
5. Percentage change in hemoglobin
6. Proportion of participants who received transfusions
7. Change in corticosteroid dose from baseline
8. Percentage change from baseline in daily corticosteroid dose
9. Proportion of participants who required rescue therapy at any visit
10. Number of Participants with Treatment Emergent Adverse Events (TEAE)

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: <= 99age old
• Gender: Both

Include 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.

Exclude 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.

Related Information

• Primary Sponsor: Ueda Eiji
• Secondary ID(s): NCT05073458