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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
25
Parsaclisib administered orally.
Georgetown University Hospital
Washington D.C., District of Columbia, United States
University of Minnesota
Minneapolis, Minnesota, United States
Washington University School of Medicine
St Louis, Missouri, United States
Percentage of Participants Attaining a Complete Response at Any Visit From 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.
Time frame: Week 6 to Week 12
Percentage of Participants Attaining a Partial Response at Any Visit From 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.
Time frame: Week 6 to Week 12
Number of Participants With Any Treatment-emergent Adverse Event (TEAE)
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.
Time frame: up to 1638 days
Percentage of Participants Attaining a Complete Response During Post-Baseline Visits
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.
Time frame: up to 1638 days
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Weill Medical College of Cornell University
New York, New York, United States
Montefiore Medical Center
The Bronx, New York, United States
University Health System Inc., Dba the University of Tn Medical Center
Knoxville, Tennessee, United States
Allgemeines Krankenhaus Der Stadt Wien
Vienna, Austria
Centre Hospitalier Universitaire Henri Mondor
Créteil, France
Centre Hospitalier Regional Universitaire (Chru) de Lille
Lille, France
Fondazione Irccs Ca Granda Ospedale Maggiore
Milan, Italy
...and 2 more locations
Percentage of Participants Attaining a Partial Response During Post-Baseline Visits
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.
Time frame: up to 1638 days
Percentage of Participants Attaining a ≥ 2 g/dL Increase in Hemoglobin From Baseline
Hemoglobin levels were assessed throughout the study.
Time frame: up to 1638 days
Change From Baseline in Hemoglobin
Change from Baseline was calculated as the post-Baseline value minus the Baseline value.
Time frame: Baseline; up to 1638 days
Percentage Change From Baseline in Hemoglobin
Percentage change from Baseline was calculated as: (\[post-Baseline value minus the Baseline value\] / Baseline value) x 100.
Time frame: Baseline; up to 1638 days
Percentage of Participants Requiring Transfusions
A participant was defined to have required a transfusion if his or her last transfusion was within 7 days of the visit date.
Time frame: Baseline; up to 1638 days
Percentage of Participants Who Achieved Normalization of Hemoglobin, Haptoglobin, Lactate Dehydrogenase (LDH), Reticulocyte Count, Total Bilirubin, Direct Bilirubin, and Indirect Bilirubin
Normalization was determined by the Investigator based on normal ranges for the clinical reference laboratory.
Time frame: up to 1638 days
Percentage of Participants Requiring a Prednisone Dose Change (Increase or Decrease)
Prednisone use was monitored throughout the study.
Time frame: up to 1638 days
Change From Baseline in the Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) Subscale Scores
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.
Time frame: Baseline; up to 1638 days
Mean Cmax of Parsaclisib
Cmax was defined as the maximum observed concentration. Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2.
Time frame: predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8
Mean Tmax of Parsaclisib
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.
Time frame: predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8
Mean Cmin of Parsaclisib
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.
Time frame: predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8
Mean AUC0-4 of Parsaclisib
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.
Time frame: predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8
Mean AUC0-t of Parsaclisib
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.
Time frame: predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8
Mean Clast of Parsaclisib
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.
Time frame: predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8
Mean Tlast of Parsaclisib
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.
Time frame: predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8
Change From Baseline in Reticulocyte Count
Change from Baseline was calculated as the post-Baseline value minus the Baseline value
Time frame: Baseline; up to 1638 days
Change From Baseline in Cardiolipin Immunoglobulin G (IgG) Antibody and Cardiolipin Immunoglobulin M (IgM) Antibody
Change from Baseline was calculated as the post-Baseline value minus the Baseline value.
Time frame: Baseline; Week 12
Change From Baseline in Cold Hemagglutinin Levels
Change from Baseline was calculated as the post-Baseline value minus the Baseline value.
Time frame: Baseline; Week 12
Change From Baseline in Haptoglobin, Total Bilirubin, Direct Bilirubin, and Indirect Bilirubin
Change from Baseline was calculated as the post-Baseline value minus the Baseline value.
Time frame: Baseline; up to 1638 days
Change From Baseline in Lactate Dehydrogenase (LDH)
Change from Baseline was calculated as the post-Baseline value minus the Baseline value.
Time frame: Baseline; up to 1638 days
Change From Baseline in CH50
Change from Baseline was calculated as the post-Baseline value minus the Baseline value.
Time frame: Baseline; up to 1638 days
Change From Baseline in Complement C3 and Complement C4
Change from Baseline was calculated as the post-Baseline value minus the Baseline value.
Time frame: Baseline; up to 1638 days