Clinical trial NCT05073458: Study of the Efficacy and Safety of Parsaclisib in Participants With Primary Warm Autoimmune Hemolytic Anemia
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Warm Autoimmune Hemolytic Anemia (wAIHA)
Keywords: Warm Autoimmune Hemolytic Anemia (wAIHA)
Lead sponsor: Incyte Corporation
Sponsor class: INDUSTRY
First posted: 2021-10-11
Enrollment: 13
Sex: ALL
Minimum age (years): 18
Maximum age (years): 99
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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),

Detailed 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.

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.

Primary outcomes:
measure: Percentage of Participants Attaining a Durable Hemoglobin Response
timeFrame: up to Week 24
Secondary outcomes:
measure: Percentage of Participants With a ≥3-point Increase From Baseline in Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) Score at Week 24
timeFrame: Baseline; Week 24
measure: Percentage of Participants With a 50 Meter Increase From Baseline to Week 24 in a 6-minute Walk Test (6MWT)
timeFrame: Baseline; Week 24
measure: Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit
timeFrame: Baseline; Day 1; Weeks 8, 12, 16, 20, 24, 28, 32, 40, 48, 56, and every 16 weeks post-Week 56
measure: Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit
timeFrame: Baseline; Day 1; Weeks 8, 12, 16, 20, 24, 28, 32, 40, 48, 56, and every 16 weeks post-Week 56
measure: Change From Baseline in Hemoglobin at Each Post-Baseline Visit
timeFrame: Baseline; Day 1; Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, and every 8 weeks post-Week 56
measure: Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit
timeFrame: Baseline; Day 1; Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, and every 8 weeks post-Week 56
measure: Percentage of Participants Who Received Transfusions From Week 6 to Week 24 and From Week 24 to Week 48
timeFrame: Week 6 to Week 24; Week 24 to Week 48
measure: Change From Baseline in Daily Corticosteroid Dose at Week 24
timeFrame: Baseline; Week 24
measure: Percentage Change From Baseline in Daily Corticosteroid Dose at Week 24
timeFrame: Baseline; Week 24
measure: Percentage of Participants Who Required Rescue Therapy at Any Visit From Week 6 Through Week 24, and From Week 24 to Week 48
timeFrame: Week 6 to Week 24; Week 24 to Week 48
measure: Number of Participants With Any Treatment-emergent Adverse Event (TEAE)
timeFrame: up to 446 days
measure: Number of Participants With Any ≥Grade 3 TEAE
timeFrame: up to 446 days
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Investigative Site US005
state: California
city: Whittier
country: United States
facility: Investigative Site US004
state: California
city: Miami
country: United States
facility: Investigative Site US006
state: Florida
city: Indianapolis
country: United States
facility: Investigative Site US012
state: Indiana
city: Bronx
country: United States
facility: Investigative Site US007
state: New York
city: Bronx
country: United States
facility: Investigative Site US002
state: New York
city: Greenville
country: United States
facility: Investigative Site US003
state: North Carolina
city: Canton
country: United States
facility: Investigative Site US009
state: Ohio
city: Easton
country: United States
facility: Investigative Site US010
state: Pennsylvania
city: Knoxville
country: United States
facility: Investigative Site US001
state: Tennessee
city: Salzburg CET
country: Austria
facility: Investigative Site AT002
city: Vienna
country: Austria
facility: Investigative Site AT001
city: La Louviere
country: Belgium
facility: Investigative Site BE001
city: Liege
country: Belgium
facility: Investigative Site BE002
city: Edmonton
country: Canada
facility: Investigative Site CA001
state: Alberta
city: Lille Cedex
country: France
facility: Investigative Site FR002
city: Marseille
country: France
facility: Investigative Site FR003
city: Paris
country: France
facility: Investigative Site FR001
city: Essen
country: Germany
facility: Investigative Site DE001
city: ULM
country: Germany
facility: Investigative Site DE002
city: Haifa
country: Israel
facility: Investigative Site IL002
city: Nahariya
country: Israel
facility: Investigative Site IL001
city: Firenze
country: Italy
facility: Investigative Site IT003
city: Milan
country: Italy
facility: Investigative Site IT002
city: Novara
country: Italy
facility: Investigative Site IT001
city: Pavia
country: Italy
facility: Investigative Site IT004
city: Rome
country: Italy
facility: Investigative Site IT006
city: Trieste
country: Italy
facility: Investigative Site IT005
city: Fukuoka
country: Japan
facility: Investigative Site JP008
city: Isehara
country: Japan
facility: Investigative Site JP004
city: Nagoya
country: Japan
facility: Investigative Site JP006
city: Okayama
country: Japan
facility: Investigative Site JP009
city: Okayama
country: Japan
facility: Investigative Site JP002
city: Osakasayama-shi
country: Japan
facility: Investigative Site JP010
city: Saitama
country: Japan
facility: Investigative Site JP005
city: Sendai-shi
country: Japan
facility: Investigative Site JP007
city: Suita-shi
country: Japan
facility: Investigative Site JP001
city: Tokyo
country: Japan
facility: Investigative Site JP003
city: Rotterdam
country: Netherlands
facility: Investigative Site NL001
city: Legnica
country: Poland
facility: Investigative Site PL001
city: Lodz
country: Poland
facility: Investigative Site PL006
city: Nowy Sacz
country: Poland
facility: Investigative Site PL003
city: Opole
country: Poland
facility: Investigative Site PL005
city: Walbrzych
country: Poland
facility: Investigative Site PL004
city: Wroclaw
country: Poland
facility: Investigative Site PL002
city: Badalona
country: Spain
facility: Investigative Site ES006
city: Barcelona
country: Spain
facility: Investigative Site ES001
city: Madrid
country: Spain
facility: Investigative Site ES003
city: Murcia
country: Spain
facility: Investigative Site ES005
city: Tarragona
country: Spain
facility: Investigative Site ES004
city: Valencia
country: Spain
facility: Investigative Site ES002
city: Glasgow
country: United Kingdom
facility: Investigative Site GB002
city: London
country: United Kingdom
facility: Investigative Site GB006
city: Norwich
country: United Kingdom
facility: Investigative Site GB003
city: Plymouth
country: United Kingdom
facility: Investigative Site GB004
city: Reading
country: United Kingdom
facility: Investigative Site GB005
Study officials:
affiliation: Incyte Corporation
name: Kathleen Butler, MD
role: STUDY_DIRECTOR
References: