Phase 2
N=25
A Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia
Autoimmune Hemolytic Anemia
Bottom Line
View on ClinicalTrials.gov: NCT03538041 ↗Enrolled (actual)
25
Serious AEs
44.0%
Results posted
Sep 2022
Primary outcome: Primary: Percentage of Participants Attaining a Complete Response at Any Visit From Week 6 to Week 12 — 20.0; 40.0 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Parsaclisib (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Incyte Corporation
- Primary completion
- Aug 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants Attaining a Complete Response at Any Visit From Week 6 to Week 12 |
20.0; 40.0 | — |
| PRIMARY Percentage of Participants Attaining a Partial Response at Any Visit From Week 6 to Week 12 |
60.0; 66.7 | — |
| PRIMARY Number of Participants With Any Treatment-emergent Adverse Event (TEAE) |
10; 15 | — |
| SECONDARY Percentage of Participants Attaining a Complete Response During Post-Baseline Visits |
0.0; 6.7; 10.0; 6.7; 22.2; 21.4 | — |
| SECONDARY Percentage of Participants Attaining a Partial Response During Post-Baseline Visits |
50.0; 53.3; 50.0; 66.7; 44.4; 64.3 | — |
| SECONDARY Percentage of Participants Attaining a ≥ 2 g/dL Increase in Hemoglobin From Baseline |
0.0; 26.7; 10.0; 46.7; 22.2; 42.9 | — |
| SECONDARY Change From Baseline in Hemoglobin |
9.1; 8.7; 0.9; 1.2; 0.6; 1.5 | — |
| SECONDARY Percentage Change From Baseline in Hemoglobin |
10.1; 14.4; 6.0; 18.5; 9.9; 19.2 | — |
| SECONDARY Percentage of Participants Requiring Transfusions |
0.0; 6.7; 0.0; 0.0; 22.2; 0.0 | — |
| SECONDARY Percentage of Participants Who Achieved Normalization of Hemoglobin, Haptoglobin, Lactate Dehydrogenase (LDH), Reticulocyte Count, Total Bilirubin, Direct Bilirubin, and Indirect Bilirubin |
0.0; 6.7; 0.0; 6.7; 0.0; 14.3 | — |
| SECONDARY Percentage of Participants Requiring a Prednisone Dose Change (Increase or Decrease) |
0.0; 6.7; 20.0; 6.7; 0.0; 0.0 | — |
| SECONDARY Change From Baseline in the Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) Subscale Scores |
32.6; 30.1; 6.7; 8.2; 6.2; 5.4 | — |
| SECONDARY Mean Cmax of Parsaclisib |
94.9; 191; 107; 219; 199 | 0.2815 |
| SECONDARY Mean Tmax of Parsaclisib |
1.00; 1.00; 1.00; 1.04; 1.02 | 0.2921 |
| SECONDARY Mean Cmin of Parsaclisib |
20.0; 32.3; 14.2; 39.4; 25.5 | 0.1267 |
| SECONDARY Mean AUC0-4 of Parsaclisib |
260; 542; 287; 631; 524 | 0.2676 |
| SECONDARY Mean AUC0-t of Parsaclisib |
260; 553; 287; 633; 524 | — |
| SECONDARY Mean Clast of Parsaclisib |
59.6; 128; 62.4; 134; 138 | — |
| SECONDARY Mean Tlast of Parsaclisib |
3.85; 3.98; 3.83; 3.92; 3.84 | — |
| SECONDARY Change From Baseline in Reticulocyte Count |
240.7460; 268.1429; -6.8244; -37.9910; -17.2571; -55.4934 | — |
| SECONDARY Change From Baseline in Cardiolipin Immunoglobulin G (IgG) Antibody and Cardiolipin Immunoglobulin M (IgM) Antibody |
0.0; 5.8; 0.0; 1.7 | — |
| SECONDARY Change From Baseline in Cold Hemagglutinin Levels |
3200; 11206.67; -1760; 960 | — |
| SECONDARY Change From Baseline in Haptoglobin, Total Bilirubin, Direct Bilirubin, and Indirect Bilirubin |
0.00; 0.81; 0.65; 1.20; 0.00; 0.90 | — |
| SECONDARY Change From Baseline in Lactate Dehydrogenase (LDH) |
328.9; 469.0; 11.8; -85.1; 32.2; -82.8 | — |
| SECONDARY Change From Baseline in CH50 |
84.3; 69.5; 5.4; 17.2; 91.5; 67.6 | — |
| SECONDARY Change From Baseline in Complement C3 and Complement C4 |
0.96; 0.91; 0.20; 0.20; 0.04; 0.12 | — |
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.
Data sourced from ClinicalTrials.gov (NCT03538041). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.