A Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia

NCT ID: NCT03538041

Last Updated: 2025-07-11

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

25 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-11-21

Study Completion Date

2024-04-02

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Autoimmune Hemolytic Anemia

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

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.

Group Type EXPERIMENTAL

Parsaclisib

Intervention Type DRUG

Parsaclisib administered orally.

Parsaclisib 2.5 mg QD

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

Group Type EXPERIMENTAL

Parsaclisib

Intervention Type DRUG

Parsaclisib administered orally.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Parsaclisib

Parsaclisib administered orally.

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

INCB050465

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Incyte Corporation

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Kathleen Butler, MD

Role: STUDY_DIRECTOR

Incyte Corporation

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Georgetown University Hospital

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

Site Status

University of Minnesota

Minneapolis, Minnesota, United States

Site Status

Washington University School of Medicine

St Louis, Missouri, United States

Site Status

Weill Medical College of Cornell University

New York, New York, United States

Site Status

Montefiore Medical Center

The Bronx, New York, United States

Site Status

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

Knoxville, Tennessee, United States

Site Status

Allgemeines Krankenhaus Der Stadt Wien

Vienna, , Austria

Site Status

Centre Hospitalier Universitaire Henri Mondor

Créteil, , France

Site Status

Centre Hospitalier Regional Universitaire (Chru) de Lille

Lille, , France

Site Status

Fondazione Irccs Ca Granda Ospedale Maggiore

Milan, , Italy

Site Status

UNIVERSIT� DI NAPOLI FEDERICO II

Napoli, , Italy

Site Status

AZIENDA OSPEDALIERO UNIVERSITARIA MAGGIORE DELLA CARIT� DI NOVARA

Novara, , Italy

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States Austria France Italy

References

Explore related publications, articles, or registry entries linked to this study.

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.

Reference Type DERIVED
PMID: 39435908 (View on PubMed)

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

Parsaclisib

Identifier Type: OTHER

Identifier Source: secondary_id

2017-003652-22

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

INCB 50465-206

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Leniolisib for Immune Dysregulation in PIDs
NCT06549114 RECRUITING PHASE2
Belimumab in Autoimmune Hepatitis
NCT06381453 RECRUITING PHASE2