Autoimmune hemolytic anemia

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

Incyte Study ID:
INCB 50465-309
CT.gov ID:
NCT05073458
Eudra ID:
2021-002844-66
EU CT Number:
N/A
Sponsor:
Incyte Corporation
Collaborator:
N/A
Study Contact Information:
1.855.463.3463 or [email protected]
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Results Available

Protocol
Available Languages: English
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Statistical Analysis Plan (SAP)
Available Languages: English
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Clinical Study Purpose

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),

Clinical Study Summary

MEDICAL CONDITION(S)
  • Autoimmune hemolytic anemia
    • PRODUCT
  • Drug: parsaclisinib
  • Drug: placebo
    • COLLABORATORS
    N/A
    DATE
    Mar 2022 - Apr 2024
    TYPE
    Interventional
    PHASE
    Phase 3
    SEX
    Female & Male
    AGE
    18 - 99 Years
    ACCEPTS HEALTHY VOLUNTEERS
    No

    Clinical Study Locations

    Name
    Contact Us
    Name
    Investigative Site US006
    MIAMI, FL, US, 33165
    Name
    Investigative Site JP001
    SUITA-SHI, Japan, 565-0871
    Name
    Investigative Site JP002
    OKAYAMA, Japan, 701-0192
    Name
    Investigative Site JP003
    TOKYO, Japan, 141-8625
    Name
    Investigative Site JP004
    ISEHARA, Japan, 259-1193
    Name
    Investigative Site JP005
    SAITAMA, Japan, 350-0495
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    Key Inclusion and 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.

    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).
    Requirements information
    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.

    Protocol Summary

    Incyte Study ID:
    INCB 50465-309
    Primary Purpose:
    Treatment
    Allocation:
    Randomized
    Study Design:
    Parallel Assignment
    Masking:
    Triple (Participant, Care Provider, Investigator)
    Interventions:
  • Drug: parsaclisinib
  • Drug: placebo
    • Enrollment:
    13
    Primary Outcome
    Open

    Proportion of participants attaining a durable hemoglobin response

    Timeframe: Up to Week 24

    Secondary Outcome
    Open

    Proportion of participants with a ≥ 3-point increase in FACIT-F score

    Timeframe: Up to Week 24

    Proportion of participants with a 50 m increase in a 6MWT

    Timeframe: Up to Week 24

    Change in FACIT-F score

    Timeframe: Up to 3 years

    Percent Change in FACIT-F

    Timeframe: Up to 3 years

    Change in hemoglobin

    Timeframe: Up to 3 years

    Percentage change in hemoglobin

    Timeframe: Up to 3 years

    Proportion of participants who received transfusions

    Timeframe: Up to 48 weeks

    Change in corticosteroid dose from baseline

    Timeframe: Up to Week 24

    Percentage change from baseline in daily corticosteroid dose

    Timeframe: Up to Week 24

    Proportion of participants who required rescue therapy at any visit

    Timeframe: Up to 48 weeks

    Number of Participants with Treatment Emergent Adverse Events (TEAE)

    Timeframe: Up to 3 years