A Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia

Incyte

Status and phase: Completed; Phase 2

Conditions: Autoimmune Hemolytic Anemia

Treatments: Drug: Parsaclisib

Study type: Interventional

Funder types: Industry

Identifiers

NCT03538041

INCB 50465-206

Parsaclisib (Other Identifier)

2017-003652-22 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment: 25 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

---

25 participants in 2 patient groups

Parsaclisib 1 mg QD

Experimental group

Description:

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.

Treatment:

Drug: Parsaclisib

Parsaclisib 2.5 mg QD

Experimental group

Description:

Parsaclisib at 2.5 mg QD for 12 weeks followed by extension period.

Treatment:

Drug: Parsaclisib