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

• Register: JPRN
• Last refreshed on: 17 October 2023
• Main ID: JPRN-jRCT2051210140
• Date of registration: 24/12/2021
• Prospective Registration: Yes
• Primary sponsor: Ueda Eiji
• Public title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy and Safety of Parsaclisib in Participants With Primary Warm Autoimmune Hemolytic Anemia
• Scientific title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy and Safety of Parsaclisib in Participants With Primary Warm Autoimmune Hemolytic Anemia - PATHWAY
• Date of first enrolment: 30/12/2021
• Target sample size: 100
• Recruitment status: Not Recruiting
• URL: https://jrct.niph.go.jp/latest-detail/jRCT2051210140
• Study type: Interventional
• Study design: randomized controlled trial, double blind, placebo control, parallel assignment, treatment purpose
• Phase: 3

Countries of recruitment

Austria, Belgium, Canada, France, Germany, Israel, Italy, Japan, Netherlands, Poland, Spain, U.S.A, United Kingdom

Contacts

• Name: Medical Information Center
• Affiliation: Incyte Biosciences Japan G.K.

• Name: Eiji Ueda
• Affiliation: Incyte Biosciences Japan G.K.

Key inclusion & exclusion 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.

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

Health Condition(s) or Problem(s) studied

Warm Autoimmune Hemolytic Anemia (wAIHA)

Intervention(s)

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

Primary Outcome(s)

Proportion of participants attaining a durable hemoglobin response

Secondary Outcome(s)

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)

Secondary ID(s)

NCT05073458

Ethics review

Status: Approval
Approval date:
Contact:

[e-mail]

Institutional Review Board of Osaka University Hospital (The first approved IRB is shown)

[phone]

[e-mail]