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Completed

NCT Number: NCT03538041

A Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia

The purpose of this study is to evaluate the safety and efficacy of parsaclisib administered orally to participants with autoimmune hemolytic anemia (AIHA) who have decreased hemoglobin and evidence of ongoing hemolysis that requires treatment intervention.

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Key information

Conditions

Autoimmune Hemolytic Anemia Anemia Anemia, Hemolytic Anemia, Hemolytic, Autoimmune Autoimmune Diseases Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hereditary Sensory and Autonomic Neuropathies Heredodegenerative Disorders, Nervous System Immune System Diseases Nervous System Diseases Nervous System Malformations Neurodegenerative Diseases Neuromuscular Diseases Peripheral Nervous System Diseases Polyneuropathies

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Allgemeines Krankenhaus Der Stadt Wien, Vienna, Austria

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Diagnosis of AIHA based on the presence of hemolytic anemia and serological evidence of anti-erythrocyte antibodies, detectable by the direct antiglobulin test.
  • Participants who have disease progression after treatment with standard therapies that are known to confer clinical benefit, or who are intolerant to treatment, or who refuse standard treatment. There is no limit to the number of prior treatment regimens.
  • Hemoglobin 7 to 10 g/dL.
  • No evidence of a lymphoproliferative malignancy or other autoimmune-related underlying conditions.
  • Eastern Cooperative Oncology Group performance status of 0 to 2.
  • Willingness to avoid pregnancy or fathering children.

Exclusion criteria

  • Pregnant or breastfeeding women.
  • Concurrent conditions and history of other protocol-specified diseases.
  • ANC < 1.5 × 10^9/L.
  • Platelet count < 100 × 10^9/L.
  • Severely impaired liver function.
  • Impaired renal function with estimated creatinine clearance less than 45 mL/min.
  • Anti-phospholipid antibodies positive or elevated anti-streptolysin antibodies.
  • Positive serology test results for hepatitis B surface antigen or core antibody, or hepatitis C virus antibody with detectable RNA at screening, consistent with active or chronic infection.
  • Known HIV infection or positivity on immunoassay.
  • History or presence of an abnormal ECG that, in the investigator's opinion, is clinically meaningful.
  • Known hypersensitivity or severe reaction to parsaclisib or its excipients.

Treatment and study plan

Parsaclisib

Drug

Parsaclisib administered orally.

Other names: INCB050465

Primary outcomes

  1. Percentage of Participants Attaining a Complete Response at Any Visit From Week 6 to Week 12

    Time frame: Week 6 to Week 12

    A complete response was defined as hemoglobin >12 grams per deciliter (g/dL) not attributed to a transfusion effect and the normalization of hemolytic markers. No transfusion effect was defined as > 1 week since the last transfusion.

  2. Percentage of Participants Attaining a Partial Response at Any Visit From Week 6 to Week 12

    Time frame: Week 6 to Week 12

    A partial response was defined as hemoglobin 10-12 g/dL or at least a 2 g/dL increase from Baseline not attributed to a transfusion effect and the normalization of hemolytic markers. No transfusion effect was defined as > 1 week since the last transfusion.

  3. Number of Participants With Any Treatment-emergent Adverse Event (TEAE)

    Time frame: up to 1638 days

    An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related, that occurred after a participant provided informed consent. Abnormal laboratory values or test results occurring after informed consent constituted AEs only if they induced clinical signs or symptoms, were considered clinically meaningful, required therapy, or required changes in the study drug. Anemia and transfusions should not have been reported as AEs unless they represented a clinically meaningful decrease from Baseline in hemoglobin. A TEAE was defined as any AE either reported for the first time or the worsening of a pre-existing event after the first dose of study drug.

Secondary outcomes

  1. Percentage of Participants Attaining a Complete Response During Post-Baseline Visits

    Time frame: up to 1638 days

    A complete response was defined as hemoglobin >12 g/dL) not attributed to a transfusion effect and the normalization of hemolytic markers. No transfusion effect was defined as > 1 week since the last transfusion.

  2. Percentage of Participants Attaining a Partial Response During Post-Baseline Visits

    Time frame: up to 1638 days

    A partial response was defined as hemoglobin 10-12 g/dL or at least a 2 g/dL increase from Baseline not attributed to a transfusion effect and the normalization of hemolytic markers. No transfusion effect was defined as > 1 week since the last transfusion.

  3. Percentage of Participants Attaining a ≥ 2 g/dL Increase in Hemoglobin From Baseline

    Time frame: up to 1638 days

    Hemoglobin levels were assessed throughout the study.

  4. Change From Baseline in Hemoglobin

    Time frame: Baseline; up to 1638 days

    Change from Baseline was calculated as the post-Baseline value minus the Baseline value.

  5. Percentage Change From Baseline in Hemoglobin

    Time frame: Baseline; up to 1638 days

    Percentage change from Baseline was calculated as: ([post-Baseline value minus the Baseline value] / Baseline value) x 100.

  6. Percentage of Participants Requiring Transfusions

    Time frame: Baseline; up to 1638 days

    A participant was defined to have required a transfusion if his or her last transfusion was within 7 days of the visit date.

  7. Percentage of Participants Who Achieved Normalization of Hemoglobin, Haptoglobin, Lactate Dehydrogenase (LDH), Reticulocyte Count, Total Bilirubin, Direct Bilirubin, and Indirect Bilirubin

    Time frame: up to 1638 days

    Normalization was determined by the Investigator based on normal ranges for the clinical reference laboratory.

  8. Percentage of Participants Requiring a Prednisone Dose Change (Increase or Decrease)

    Time frame: up to 1638 days

    Prednisone use was monitored throughout the study.

  9. Change From Baseline in the Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) Subscale Scores

    Time frame: Baseline; up to 1638 days

    The FACIT-F subscale is a 13-item instrument designed to assess fatigue/tiredness and its impact on daily activities and functioning in a number of chronic diseases. Participants were asked to respond to 13 statements that people with the illness have said are important on the following scale: 0, not at all; 1, a little bit; 2, somewhat; 3, quite a bit; 4, very much. Participants were asked to indicate the response as it applied to the last 7 days. The total fatigue subscale score ranges from 0 to 52; a higher score indicates more severe impact on daily activities and functioning.

  10. Mean Cmax of Parsaclisib

    Time frame: predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8

    Cmax was defined as the maximum observed concentration. Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2.

  11. Mean Tmax of Parsaclisib

    Time frame: predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8

    tmax was defined as the time to the maximum concentration. Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2.

  12. Mean Cmin of Parsaclisib

    Time frame: predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8

    Cmin was defined as the minimum observed concentration over the dose interval. Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2.

  13. Mean AUC0-4 of Parsaclisib

    Time frame: predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8

    AUC0-4 was defined as the area under the concentration-time curve from time = 0 to 4 hours postdose. Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2.

  14. Mean AUC0-t of Parsaclisib

    Time frame: predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8

    AUC0-t was defined as the area under the concentration-time curve from time = 0 to the last measureable concentration at time = t. Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2.

  15. Mean Clast of Parsaclisib

    Time frame: predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8

    Clast was defined as the last measurable concentration (above the quantification limit). Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2.

  16. Mean Tlast of Parsaclisib

    Time frame: predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8

    Tlast was defined as the time of the last measurable concentration (above the quantification limit). Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2.

  17. Change From Baseline in Reticulocyte Count

    Time frame: Baseline; up to 1638 days

    Change from Baseline was calculated as the post-Baseline value minus the Baseline value

  18. Change From Baseline in Cardiolipin Immunoglobulin G (IgG) Antibody and Cardiolipin Immunoglobulin M (IgM) Antibody

    Time frame: Baseline; Week 12

    Change from Baseline was calculated as the post-Baseline value minus the Baseline value.

  19. Change From Baseline in Cold Hemagglutinin Levels

    Time frame: Baseline; Week 12

    Change from Baseline was calculated as the post-Baseline value minus the Baseline value.

  20. Change From Baseline in Haptoglobin, Total Bilirubin, Direct Bilirubin, and Indirect Bilirubin

    Time frame: Baseline; up to 1638 days

    Change from Baseline was calculated as the post-Baseline value minus the Baseline value.

  21. Change From Baseline in Lactate Dehydrogenase (LDH)

    Time frame: Baseline; up to 1638 days

    Change from Baseline was calculated as the post-Baseline value minus the Baseline value.

  22. Change From Baseline in CH50

    Time frame: Baseline; up to 1638 days

    Change from Baseline was calculated as the post-Baseline value minus the Baseline value.

  23. Change From Baseline in Complement C3 and Complement C4

    Time frame: Baseline; up to 1638 days

    Change from Baseline was calculated as the post-Baseline value minus the Baseline value.

Sponsors and collaborators

Lead sponsor

Incyte Corporation

Industry

Registry information

Official study title

A Phase 2, Open-Label Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia

Important dates

Study start
2018
Primary completion
2021
Study completion
2024
First posted
May 25, 2018
Registry last updated
Jul 11, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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