A Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia
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 |
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Phase 2 |
Study Design
Arms and Interventions
Arm | Intervention/Treatment |
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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:
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Experimental: Cohort 2 Parsaclisib at the protocol-defined dose for 12 weeks followed by extension period. |
Drug: Parsaclisib
Parsaclisib administered orally.
Other Names:
|
Outcome Measures
Primary Outcome Measures
- 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.
- 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.
- 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
- 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.
- 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.
- 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.
- Change from baseline of hemoglobin [From Baseline up to 28 weeks]
Hemoglobin level will be monitored throughout the study.
- Proportion of participants requiring transfusions [From Baseline up to 28 weeks]
Transfusion requirements will be monitored throughout the study.
- 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.
- Change of daily usage of prednisone [From Baseline up to 28 weeks]
Prednisone use will be monitored throughout the study.
- 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
Inclusion Criteria:
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Diagnosis of AIHA based on the presence of hemolytic anemia and serological evidence of anti-erythrocyte antibodies, detectable by the direct antiglobulin test.
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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.
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Hemoglobin 7 to 10 g/dL.
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No evidence of a lymphoproliferative malignancy or other autoimmune-related underlying conditions.
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Eastern Cooperative Oncology Group performance status of 0 to 2.
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Willingness to avoid pregnancy or fathering children.
Exclusion Criteria:
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Pregnant or breastfeeding women.
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Concurrent conditions and history of other protocol-specified diseases.
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ANC < 1.5 × 10^9/L.
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Platelet count < 100 × 10^9/L.
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Severely impaired liver function.
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Impaired renal function with estimated creatinine clearance less than 45 mL/min.
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Anti-phospholipid antibodies positive or elevated anti-streptolysin antibodies.
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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.
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Known HIV infection or positivity on immunoassay.
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History or presence of an abnormal ECG that, in the investigator's opinion, is clinically meaningful.
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Known hypersensitivity or severe reaction to parsaclisib or its excipients.
Contacts and Locations
Locations
Site | City | State | Country | Postal Code | |
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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.- INCB 50465-206
- Parsaclisib