A Phase 2, Open-Label Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 25
- 试验地点
- 12
- 主要终点
- Percentage of Participants Attaining a Complete Response at Any Visit From Week 6 to Week 12
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
- •Willingness to avoid pregnancy or fathering children.
排除标准
- •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.
研究组 & 干预措施
Parsaclisib 1 mg QD
Parsaclisib at 1 milligram (mg) once daily (QD) for 12 weeks followed by extension period, with a dose-increase option (to 2.5 mg QD) at Week 6 for participants who fulfill dose increase criteria.
干预措施: Parsaclisib (Drug)
Parsaclisib 2.5 mg QD
Parsaclisib at 2.5 mg QD for 12 weeks followed by extension period.
干预措施: Parsaclisib (Drug)
结局指标
主要结局
Percentage of Participants Attaining a Complete Response at Any Visit From Week 6 to Week 12
时间窗: 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.
Percentage of Participants Attaining a Partial Response at Any Visit From Week 6 to Week 12
时间窗: 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.
Number of Participants With Any Treatment-emergent Adverse Event (TEAE)
时间窗: up to 1638 days
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.
次要结局
- Percentage of Participants Attaining a ≥ 2 g/dL Increase in Hemoglobin From Baseline(up to 1638 days)
- Percentage Change From Baseline in Hemoglobin(Baseline; up to 1638 days)
- Percentage of Participants Attaining a Complete Response During Post-Baseline Visits(up to 1638 days)
- Percentage of Participants Attaining a Partial Response During Post-Baseline Visits(up to 1638 days)
- Percentage of Participants Requiring Transfusions(Baseline; up to 1638 days)
- Percentage of Participants Requiring a Prednisone Dose Change (Increase or Decrease)(up to 1638 days)
- Change From Baseline in the Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) Subscale Scores(Baseline; up to 1638 days)
- Mean Cmax of Parsaclisib(predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8)
- Change From Baseline in Hemoglobin(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(up to 1638 days)
- Mean Tmax of Parsaclisib(predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8)
- Mean Cmin of Parsaclisib(predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8)
- Mean AUC0-4 of Parsaclisib(predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8)
- Mean AUC0-t of Parsaclisib(predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8)
- Mean Clast of Parsaclisib(predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8)
- Mean Tlast of Parsaclisib(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(Baseline; up to 1638 days)
- Change From Baseline in Cardiolipin Immunoglobulin G (IgG) Antibody and Cardiolipin Immunoglobulin M (IgM) Antibody(Baseline; Week 12)
- Change From Baseline in Cold Hemagglutinin Levels(Baseline; Week 12)
- Change From Baseline in Haptoglobin, Total Bilirubin, Direct Bilirubin, and Indirect Bilirubin(Baseline; up to 1638 days)
- Change From Baseline in Lactate Dehydrogenase (LDH)(Baseline; up to 1638 days)
- Change From Baseline in CH50(Baseline; up to 1638 days)
- Change From Baseline in Complement C3 and Complement C4(Baseline; up to 1638 days)