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

Warm Autoimmune Hemolytic Anemia (wAIHA) Clinical Trial

— PATHWAY  

Official 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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT05073458
• Other study ID #: INCB 50465-309
• Status: Terminated
• Phase: Phase 3
• Start date: March 15, 2022
• Est. completion date: April 29, 2024

Study information

• Verified date: June 2024
• Source: Incyte Corporation
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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),

Description:

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 13
• Est. completion date: April 29, 2024
• Est. primary completion date: October 17, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 99 Years

Eligibility

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.

Related Conditions & MeSH terms

• Anemia
• Anemia, Hemolytic
• Anemia, Hemolytic, Autoimmune
• Hemolysis
• Warm Autoimmune Hemolytic Anemia (wAIHA)

Intervention

Drug:
• parsaclisinib
parsaclisib will be administered QD orally
• placebo
placebo will be administered QD orally follwed by Parsaclisinib in the open label period

Locations

Country	Name	City	State
Austria	Investigative Site AT002	Salzburg CET
Austria	Investigative Site AT001	Vienna
Belgium	Investigative Site BE001	La Louviere
Belgium	Investigative Site BE002	Liege
Canada	Investigative Site CA001	Edmonton	Alberta
France	Investigative Site FR002	Lille Cedex
France	Investigative Site FR003	Marseille
France	Investigative Site FR001	Paris
Germany	Investigative Site DE001	Essen
Germany	Investigative Site DE002	ULM
Israel	Investigative Site IL002	Haifa
Israel	Investigative Site IL001	Nahariya
Italy	Investigative Site IT003	Firenze
Italy	Investigative Site IT002	Milan
Italy	Investigative Site IT001	Novara
Italy	Investigative Site IT004	Pavia
Italy	Investigative Site IT006	Rome
Italy	Investigative Site IT005	Trieste
Japan	Investigative Site JP008	Fukuoka
Japan	Investigative Site JP004	Isehara
Japan	Investigative Site JP006	Nagoya
Japan	Investigative Site JP002	Okayama
Japan	Investigative Site JP009	Okayama
Japan	Investigative Site JP010	Osakasayama-shi
Japan	Investigative Site JP005	Saitama
Japan	Investigative Site JP007	Sendai-shi
Japan	Investigative Site JP001	Suita-shi
Japan	Investigative Site JP003	Tokyo
Netherlands	Investigative Site NL001	Rotterdam
Poland	Investigative Site PL001	Legnica
Poland	Investigative Site PL006	Lodz
Poland	Investigative Site PL003	Nowy Sacz
Poland	Investigative Site PL005	Opole
Poland	Investigative Site PL004	Walbrzych
Poland	Investigative Site PL002	Wroclaw
Spain	Investigative Site ES006	Badalona
Spain	Investigative Site ES001	Barcelona
Spain	Investigative Site ES003	Madrid
Spain	Investigative Site ES005	Murcia
Spain	Investigative Site ES004	Tarragona
Spain	Investigative Site ES002	Valencia
United Kingdom	Investigative Site GB002	Glasgow
United Kingdom	Investigative Site GB006	London
United Kingdom	Investigative Site GB003	Norwich
United Kingdom	Investigative Site GB004	Plymouth
United Kingdom	Investigative Site GB005	Reading
United States	Investigative Site US002	Bronx	New York
United States	Investigative Site US007	Bronx	New York
United States	Investigative Site US009	Canton	Ohio
United States	Investigative Site US010	Easton	Pennsylvania
United States	Investigative Site US003	Greenville	North Carolina
United States	Investigative Site US012	Indianapolis	Indiana
United States	Investigative Site US001	Knoxville	Tennessee
United States	Investigative Site US005	Los Angeles	California
United States	Investigative Site US006	Miami	Florida
United States	Investigative Site US004	Whittier	California

Sponsors (1)

Lead Sponsor	Collaborator
Incyte Corporation

Countries where clinical trial is conducted

United States,  Austria,  Belgium,  Canada,  France,  Germany,  Israel,  Italy,  Japan,  Netherlands,  Poland,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Proportion of participants attaining a durable hemoglobin response	Proportion of participants attaining a durable hemoglobin response, defined as hemoglobin = 10 g/dL with an increase from baseline of = 2 g/dL not attributed to rescue therapy at = 3 of the 4 available visits at Week 12 and/or later during the 24-week double-blind treatment period.	Up to Week 24
Secondary	Proportion of participants with a = 3-point increase in FACIT-F score	Increase is measured by Functional Assessment of Chronic Illness Therapy - Fatigue questionnaire. The FACIT-F is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function over the past 7 days.	Up to Week 24
Secondary	Proportion of participants with a 50 m increase in a 6MWT	Defined as an increase of 50 m using the Six-minute walk test, a self-paced measurement of the distance that a participant can quickly walk on a flat, hard surface in a period of 6 minutes.	Up to Week 24
Secondary	Change in FACIT-F score	Change will be measured by Functional Assessment of Chronic Illness Therapy - Fatigue questionnaire. The FACIT-F is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function over the past 7 days.	Up to 3 years
Secondary	Percent Change in FACIT-F	will be measured by Functional Assessment of Chronic Illness Therapy - Fatigue questionnaire. The FACIT-F is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function over the past 7 days.	Up to 3 years
Secondary	Change in hemoglobin	Changes will be measured and compared in the hematology panel.	Up to 3 years
Secondary	Percentage change in hemoglobin	Percentage change will be measured and compared in the hematology panel.	Up to 3 years
Secondary	Proportion of participants who received transfusions	Proportion of participants who received transfusions.	Up to 48 weeks
Secondary	Change in corticosteroid dose from baseline	Change from baseline of daily corticosteroids dose	Up to Week 24
Secondary	Percentage change from baseline in daily corticosteroid dose	Percentage change from baseline of daily corticosteroids dose	Up to Week 24
Secondary	Proportion of participants who required rescue therapy at any visit	Rescue therapy will include new/increased dose of corticosteroids, transfusions, intravenous immunoglobulin (IVIG), and Erythropoietin.	Up to 48 weeks
Secondary	Number of Participants with Treatment Emergent Adverse Events (TEAE)	Defined as any adverse event either reported for the first time or worsening of a pre-existing event after first dose of study drug.	Up to 3 years