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A Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia

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ClinicalTrials.gov Identifier: NCT03538041
Recruitment Status : Recruiting
First Posted : May 25, 2018
Last Update Posted : September 13, 2019
Sponsor:
Information provided by (Responsible Party):
Incyte Corporation

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
Autoimmune Hemolytic Anemia Drug: Parsaclisib Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 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 : July 2, 2018
Estimated Primary Completion Date : September 2020
Estimated Study Completion Date : September 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia

Arm Intervention/treatment
Experimental: Cohort 1
Parsaclisib at the protocol-defined dose for 12 weeks, with a dose-increase option at Week 6 or at Week 8 and an extension period for participants who fulfill dose increase criteria.
Drug: Parsaclisib
Parsaclisib administered orally.
Other Name: INCB050465

Experimental: Cohort 2
Parsaclisib at the protocol-defined dose for 12 weeks and an extension period for participants who fulfill dose increase criteria.
Drug: Parsaclisib
Parsaclisib administered orally.
Other Name: INCB050465




Primary Outcome Measures :
  1. Proportion of participants attaining a complete response (CR) [ Time Frame: 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) [ Time Frame: 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) [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: From Baseline up to 28 weeks ]
    Participants attaining a ≥ 2 g/dL increase in hemoglobin from baseline.

  4. Change from baseline of hemoglobin [ Time Frame: From Baseline up to 28 weeks ]
    Hemoglobin level will be monitored throughout the study.

  5. Proportion of participants requiring transfusions [ Time Frame: From Baseline up to 28 weeks ]
    Transfusion requirements will be monitored throughout the study.

  6. Proportion of participants who achieve normalization of hemolytic markers [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: From Baseline up to 28 weeks ]
    FACIT-F will be used to assess changes in patient-reported outcomes.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03538041


Contacts
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Contact: Incyte Corporation Call Center (US) 1.855.463.3463 medinfo@incyte.com
Contact: Incyte Corporation Call Center (ex-US) +800 00027423 globalmedinfo@incyte.com

Locations
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United States, District of Columbia
Georgetown University Georgetown Lombardi Comprehensive Cancer Center Division Hematology/Oncology Podium B Not yet recruiting
Washington, District of Columbia, United States, 20007
United States, Minnesota
University of Minnesota Not yet recruiting
Minneapolis, Minnesota, United States, 55455
United States, Missouri
Washington University School of Medicine in St. Louis Recruiting
Saint Louis, Missouri, United States, 63110
United States, New York
Montefiore Medical Center Department Of Oncology Recruiting
Bronx, New York, United States, 10467
Austria
Tirol Kliniken Withdrawn
Innsbruck, Austria, 6020
Medical University of Vienne Klinische Abteilung für Hämatologie und Hämostaseologie Recruiting
Vienna, Austria, 1090
France
Hôpital Henri Mondor Recruiting
Créteil, France, 94000
Hopital Claude Huriez CHRU de Lille Recruiting
Lille, France, 59037
Hopital de la Timone Withdrawn
Marseille, France, 13385
Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Recruiting
Milano, Italy, 20122
A.O.U. Federico II Recruiting
Napoli, Italy, 80131
AOU Maggiore della Carità Recruiting
Novara, Italy, 28100
Sponsors and Collaborators
Incyte Corporation
Investigators
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Study Director: Kathleen Butler, MD Incyte Corporation

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Responsible Party: Incyte Corporation
ClinicalTrials.gov Identifier: NCT03538041     History of Changes
Other Study ID Numbers: INCB 50465-206
Parsaclisib ( Other Identifier: Incyte Corporation )
First Posted: May 25, 2018    Key Record Dates
Last Update Posted: September 13, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Incyte Corporation:
autoimmune hemolytic anemia
phosphatidylinositol 3-kinase (PI3K) inhibitor
Additional relevant MeSH terms:
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Anemia
Anemia, Hemolytic
Anemia, Hemolytic, Autoimmune
Hemolysis
Hematologic Diseases
Pathologic Processes
Autoimmune Diseases
Immune System Diseases