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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 : July 27, 2018
Sponsor:
Information provided by (Responsible Party):
Incyte Corporation

Brief Summary:
The purpose of this study is to evaluate the safety and efficacy of INCB050465 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: INCB050465 Phase 2

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 : December 2019
Estimated Study Completion Date : December 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia

Arm Intervention/treatment
Experimental: Cohort 1
INCB050465 at the protocol-defined dose for 12 weeks, with a dose-increase option at Week 6 for participants who fulfill dose increase criteria.
Drug: INCB050465
INCB050465 administered orally.

Experimental: Cohort 2
INCB050465 at the protocol-defined dose for 12 weeks.
Drug: INCB050465
INCB050465 administered orally.




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 INCB050465 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
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
United States, Missouri
Washington University School of Medicine in St. Louis Not yet recruiting
Saint Louis, Missouri, United States, 63110
United States, New York
Montefiore Medical Center Not yet recruiting
Bronx, New York, United States, 10467
Austria
Medizinische Hochschule Innsbruck Not yet recruiting
INN, Austria, 6020
Medical University of Vienne, Klinische Abteilung fuer Haematologie und Haemostaseologie Not yet recruiting
Wien, Austria, 1090
France
Hopital Henri Mondor Recruiting
Créteil, France, 94000
Centre Hospitalier Regional Universitaire (CHRU) de Lille - Hopital Claude Huriez Not yet recruiting
Lille, France, 59037
"Département de Médecine Interne, Service de Médecine Interne 1 Hôpital de la Timone" Not yet recruiting
Marseille, France, 13385
Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Not yet recruiting
Milano, Italy, 20122
A.O.U. Federico II Not yet recruiting
Napoli, Italy, 80131
Azienda Ospedaliero Universitaria Maggiore della Carità di Novara Not yet recruiting
Novara, Italy, 28100
Sponsors and Collaborators
Incyte Corporation
Investigators
Study Director: Kathleen Butler, MD Incyte Corporation

Responsible Party: Incyte Corporation
ClinicalTrials.gov Identifier: NCT03538041     History of Changes
Other Study ID Numbers: INCB 50465-206
First Posted: May 25, 2018    Key Record Dates
Last Update Posted: July 27, 2018
Last Verified: July 2018
Individual Participant Data (IPD) Sharing Statement:
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:
autoimmune hemolytic anemia
phosphatidylinositol 3-kinase (PI3K) inhibitor

Additional relevant MeSH terms:
Anemia
Hemolysis
Anemia, Hemolytic
Anemia, Hemolytic, Autoimmune
Hematologic Diseases
Pathologic Processes
Autoimmune Diseases
Immune System Diseases