Clinical trial NCT03538041: A Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia
Official title: A Phase 2, Open-Label Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Autoimmune Hemolytic Anemia
Keywords: autoimmune hemolytic anemia
phosphatidylinositol 3-kinase (PI3K) inhibitor
Lead sponsor: Incyte Corporation
Sponsor class: INDUSTRY
First posted: 2018-05-25
Enrollment: 25
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility 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.

Primary outcomes:
measure: Percentage of Participants Attaining a Complete Response at Any Visit From Week 6 to Week 12
timeFrame: Week 6 to Week 12
measure: Percentage of Participants Attaining a Partial Response at Any Visit From Week 6 to Week 12
timeFrame: Week 6 to Week 12
measure: Number of Participants With Any Treatment-emergent Adverse Event (TEAE)
timeFrame: up to 1638 days
Secondary outcomes:
measure: Percentage of Participants Attaining a Complete Response During Post-Baseline Visits
timeFrame: up to 1638 days
measure: Percentage of Participants Attaining a Partial Response During Post-Baseline Visits
timeFrame: up to 1638 days
measure: Percentage of Participants Attaining a ≥ 2 g/dL Increase in Hemoglobin From Baseline
timeFrame: up to 1638 days
measure: Change From Baseline in Hemoglobin
timeFrame: Baseline; up to 1638 days
measure: Percentage Change From Baseline in Hemoglobin
timeFrame: Baseline; up to 1638 days
measure: Percentage of Participants Requiring Transfusions
timeFrame: Baseline; up to 1638 days
measure: Percentage of Participants Who Achieved Normalization of Hemoglobin, Haptoglobin, Lactate Dehydrogenase (LDH), Reticulocyte Count, Total Bilirubin, Direct Bilirubin, and Indirect Bilirubin
timeFrame: up to 1638 days
measure: Percentage of Participants Requiring a Prednisone Dose Change (Increase or Decrease)
timeFrame: up to 1638 days
measure: Change From Baseline in the Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) Subscale Scores
timeFrame: Baseline; up to 1638 days
measure: Mean Cmax of Parsaclisib
timeFrame: predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8
measure: Mean Tmax of Parsaclisib
timeFrame: predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8
measure: Mean Cmin of Parsaclisib
timeFrame: predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8
measure: Mean AUC0-4 of Parsaclisib
timeFrame: predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8
measure: Mean AUC0-t of Parsaclisib
timeFrame: predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8
measure: Mean Clast of Parsaclisib
timeFrame: predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8
measure: Mean Tlast of Parsaclisib
timeFrame: predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8
measure: Change From Baseline in Reticulocyte Count
timeFrame: Baseline; up to 1638 days
measure: Change From Baseline in Cardiolipin Immunoglobulin G (IgG) Antibody and Cardiolipin Immunoglobulin M (IgM) Antibody
timeFrame: Baseline; Week 12
measure: Change From Baseline in Cold Hemagglutinin Levels
timeFrame: Baseline; Week 12
measure: Change From Baseline in Haptoglobin, Total Bilirubin, Direct Bilirubin, and Indirect Bilirubin
timeFrame: Baseline; up to 1638 days
measure: Change From Baseline in Lactate Dehydrogenase (LDH)
timeFrame: Baseline; up to 1638 days
measure: Change From Baseline in CH50
timeFrame: Baseline; up to 1638 days
measure: Change From Baseline in Complement C3 and Complement C4
timeFrame: Baseline; up to 1638 days
Other outcomes:
Locations:
city: Washington
country: United States
facility: Georgetown University Hospital
state: District of Columbia
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Bronx
country: United States
facility: Montefiore Medical Center
state: New York
city: New York
country: United States
facility: Weill Medical College of Cornell University
state: New York
city: Knoxville
country: United States
facility: University Health System Inc., Dba the University of Tn Medical Center
state: Tennessee
city: Vienna
country: Austria
facility: Allgemeines Krankenhaus Der Stadt Wien
city: Creteil
country: France
facility: Centre Hospitalier Universitaire Henri Mondor
city: Lille
country: France
facility: Centre Hospitalier Regional Universitaire (Chru) de Lille
city: Milan
country: Italy
facility: Fondazione Irccs Ca Granda Ospedale Maggiore
city: Napoli
country: Italy
facility: UNIVERSITï¿½ DI NAPOLI FEDERICO II
city: Novara
country: Italy
facility: AZIENDA OSPEDALIERO UNIVERSITARIA MAGGIORE DELLA CARITï¿½ DI NOVARA
Study officials:
affiliation: Incyte Corporation
name: Kathleen Butler, MD
role: STUDY_DIRECTOR
References: