A Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia

Sponsor
Incyte Corporation (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT03538041
Collaborator
(none)
25
12
2
57.6
2.1
0

Study Details

Study Description

Brief Summary

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.

Condition or Disease Intervention/Treatment Phase
Phase 2

Study Design

Study Type:
Interventional
Actual Enrollment :
25 participants
Allocation:
Non-Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Phase 2, Open-Label Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia
Actual Study Start Date :
Nov 21, 2018
Actual Primary Completion Date :
Aug 5, 2021
Anticipated Study Completion Date :
Sep 8, 2023

Arms and Interventions

Arm Intervention/Treatment
Experimental: Cohort 1

Parsaclisib at the protocol-defined dose for 12 weeks followed by extension period, with a dose-increase option at Week 6 for participants who fulfill dose increase criteria.

Drug: Parsaclisib
Parsaclisib administered orally.
Other Names:
  • INCB050465
  • Experimental: Cohort 2

    Parsaclisib at the protocol-defined dose for 12 weeks followed by extension period.

    Drug: Parsaclisib
    Parsaclisib administered orally.
    Other Names:
  • INCB050465
  • Outcome Measures

    Primary Outcome Measures

    1. Proportion of participants attaining a complete response (CR) [Week 6 to Week 12]

      CR defined as hemoglobin > 12 g/dL not attributed to transfusion effect and the normalization of hemolytic markers.

    2. Proportion of participants attaining a partial response (PR) [From Baseline to Week 6 to Week 12]

      PR defined as hemoglobin 10-12 g/dL or at least ≥ 2 g/dL increase from baseline not attributed to transfusion effect and the normalization of hemolytic markers.

    3. Number of participants with a treatment-emergent adverse event (TEAE) [Up to 28 weeks]

      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.

    Secondary Outcome Measures

    1. Proportion of participants attaining a CR during post baseline visits [Up to 28 weeks]

      CR defined as hemoglobin > 12 g/dL not attributed to transfusion effect and the normalization of hemolytic markers.

    2. Proportion of participants attaining a PR during post baseline visits [From Baseline up to 28 weeks]

      PR defined as hemoglobin 10-12 g/dL or at least ≥ 2 g/dL increase from baseline not attributed to transfusion effect and the normalization of hemolytic markers.

    3. Proportion of participants attaining an increase in hemoglobin from baseline [From Baseline up to 28 weeks]

      Participants attaining a ≥ 2 g/dL increase in hemoglobin from baseline.

    4. Change from baseline of hemoglobin [From Baseline up to 28 weeks]

      Hemoglobin level will be monitored throughout the study.

    5. Proportion of participants requiring transfusions [From Baseline up to 28 weeks]

      Transfusion requirements will be monitored throughout the study.

    6. Proportion of participants who achieve normalization of hemolytic markers [Up to 28 weeks]

      Hemolysis markers include hemoglobin, haptoglobin, LDH, reticulocyte count, total bilirubin, and direct/indirect bilirubin.

    7. Change of daily usage of prednisone [From Baseline up to 28 weeks]

      Prednisone use will be monitored throughout the study.

    8. Change from baseline in Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) sub-scale questionnaire [From Baseline up to 28 weeks]

      FACIT-F will be used to assess changes in patient-reported outcomes.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Georgetown University Hospital Washington District of Columbia United States 20007
    2 University of Minnesota Minneapolis Minnesota United States 55455
    3 Washington University School of Medicine Saint Louis Missouri United States 63110
    4 Montefiore Medical Center Bronx New York United States 10467
    5 Weill Medical College of Cornell University New York New York United States 10021
    6 University Health System Inc., Dba the University of Tn Medical Center Knoxville Tennessee United States 37920
    7 Allgemeines Krankenhaus Der Stadt Wien Vienna Austria 01090
    8 Centre Hospitalier Universitaire Henri Mondor Creteil France 94010
    9 Centre Hospitalier Regional Universitaire (Chru) de Lille Lille France 59037
    10 Fondazione Irccs Ca Granda Ospedale Maggiore Milan Italy 20122
    11 UNIVERSIT� DI NAPOLI FEDERICO II Napoli Italy 80131
    12 AZIENDA OSPEDALIERO UNIVERSITARIA MAGGIORE DELLA CARIT� DI NOVARA Novara Italy 28100

    Sponsors and Collaborators

    • Incyte Corporation

    Investigators

    • Study Director: Kathleen Butler, MD, Incyte Corporation

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Incyte Corporation
    ClinicalTrials.gov Identifier:
    NCT03538041
    Other Study ID Numbers:
    • INCB 50465-206
    • Parsaclisib
    First Posted:
    May 25, 2018
    Last Update Posted:
    May 18, 2022
    Last Verified:
    May 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Incyte Corporation
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of May 18, 2022