A Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia

NCT03538041 | Completed Phase 2 Results FDA Drug

• 3 other identifiers: INCB 50465-206Parsaclisib2017-003652-22
• Study Type: interventional
• Target: 25
• Locations: 4 countries
• Sites: 12

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.

Conditions

Autoimmune Hemolytic Anemia

Keywords

autoimmune hemolytic anemia; phosphatidylinositol 3-kinase (PI3K) inhibitor

Outcome Measures

Primary Outcomes (3)

• 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.

  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.

  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.

  up to 1638 days

Secondary Outcomes (23)

• 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 Attaining a ≥ 2 g/dL Increase in Hemoglobin From Baseline

  up to 1638 days

• Change From Baseline in Hemoglobin

  Baseline; up to 1638 days

• Percentage Change From Baseline in Hemoglobin

  Baseline; up to 1638 days

Study Arms (2)

Parsaclisib 1 mg QD

EXPERIMENTAL

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.

Drug: Parsaclisib

Parsaclisib 2.5 mg QD

EXPERIMENTAL

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

Drug: Parsaclisib

Interventions

Parsaclisib DRUG

Parsaclisib administered orally.

Also known as: INCB050465

Parsaclisib 1 mg QD; Parsaclisib 2.5 mg QD

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Incyte Corporation: lead

Study Sites (12)

Georgetown University Hospital

Washington D.C., District of Columbia, 20007, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Weill Medical College of Cornell University

New York, New York, 10021, United States

Location

Montefiore Medical Center

The Bronx, New York, 10467, United States

Location

University Health System Inc., Dba the University of Tn Medical Center

Knoxville, Tennessee, 37920, United States

Location

Allgemeines Krankenhaus Der Stadt Wien

Vienna, 01090, Austria

Location

Centre Hospitalier Universitaire Henri Mondor

Créteil, 94010, France

Location

Centre Hospitalier Regional Universitaire (Chru) de Lille

Lille, 59037, France

Location

Fondazione Irccs Ca Granda Ospedale Maggiore

Milan, 20122, Italy

Location

UNIVERSIT� DI NAPOLI FEDERICO II

Napoli, 80131, Italy

Location

AZIENDA OSPEDALIERO UNIVERSITARIA MAGGIORE DELLA CARIT� DI NOVARA

Novara, 28100, Italy

Location

Related Publications (1)

• Barcellini W, Pane F, Patriarca A, Murakhovskaya I, Terriou L, DeSancho MT, Hanna WT, Leopold L, Rappold E, Szeto K, Wei S, Jager U. Parsaclisib for the treatment of primary autoimmune hemolytic anemia: Results from a phase 2, open-label study. Am J Hematol. 2024 Dec;99(12):2313-2320. doi: 10.1002/ajh.27493. Epub 2024 Oct 22.

  PMID: 39435908 DERIVED

MeSH Terms

Conditions

Anemia, Hemolytic, Autoimmune; Hereditary Sensory and Autonomic Neuropathies

Interventions

parsaclisib

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Autoimmune Diseases; Immune System Diseases; Nervous System Malformations; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Results Point of Contact

• Title: Study Director
• Organization: Incyte Corporation

medinfo@incyte.com

1-855-463-3463

Study Officials

• Kathleen Butler, MD

  Incyte Corporation

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 15, 2018
• First Posted: May 25, 2018
• Study Start: November 21, 2018
• Primary Completion: August 5, 2021
• Study Completion: April 2, 2024
• Last Updated: July 11, 2025
• Results First Posted: September 22, 2022

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

FR (2); US (6); AU (1); IT (3)