PI3K Delta Inhibitor in Relapsed / Refractory Autoimmune Hemolytic Anemia Patients After Receiving Two or More Lines of Therapy

NCT ID: NCT05676697

Last Updated: 2026-02-11

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE1
• Total Enrollment: 4 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-01-13
• Study Completion Date: 2024-02-06

Brief Summary

This is a prospective, multicenter, single-arm, pilot study. The aim of this study is to evaluate the efficacy and safety of Linperlisib, the PI3K delta inhibitor for autoimmune hemolytic anemia patients who failed the second line therapy.

Conditions

Autoimmune Hemolytic Anemia; Failure of Two Rounds of Treatment

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

PI3K Delta Inhibitor

Group Type: EXPERIMENTAL

Linperlisib

Intervention Type: DRUG

The phosphatidylinositol 3-kinase delta (PI3Kδ) signaling pathway plays a critical role in the activation, proliferation, and tissue homing of self-reactive B cells that contribute to autoimmune diseases. B cells play an essential role in immune system function and dysfunction (e.g., autoimmunity) by producing antibodies and by acting as antigen-presenting cells (APCs) for T cells. Signaling via PI3K controls many essential B cell functions and is therefore a promising target for preventing aberrant B cell activation.

Interventions

Linperlisib

The phosphatidylinositol 3-kinase delta (PI3Kδ) signaling pathway plays a critical role in the activation, proliferation, and tissue homing of self-reactive B cells that contribute to autoimmune diseases. B cells play an essential role in immune system function and dysfunction (e.g., autoimmunity) by producing antibodies and by acting as antigen-presenting cells (APCs) for T cells. Signaling via PI3K controls many essential B cell functions and is therefore a promising target for preventing aberrant B cell activation.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Male or female age ≥ 18 years
• Diagnosis of primary warm antibody hemolytic anemia (AIHA).
• Hemoglobin < 100g/L
• Refractory to or relapsed after at least 2 prior treatment line.
• ECOG performance status ≤ 2
• Willing and able to comply with the requirements for this study and written informed consent.

Exclusion Criteria

• Neutrophils counts < 0.5×10^9/L or platelet counts < 50 x 10^9/L
• Diagnosis of any of the following diseases: Cold agglutinin disease, cold agglutinin syndrome, mixed AIHA, paroxysmal cold hemoglobinuria (PCH).
• Diagnosis of the active stage of the connective tissue or systemic autoimmune rheumatic diseases (SARDs)
• History of lymphoproliferative neoplasms
• Had other inherited or acquired hemolytic diseases.
• Secondary AIHA caused by drugs or infection
• Previously received organ or stem cell transplantation.
• Had malignant tumor within 5 years before enrollment, exclusive of cured basal or squamous cell skin cancer, superficial bladder cancer, prostate intraepithelial tumor, cervical carcinoma in situ or other indolent tumors
• Patients with HBV, HCV, HIV or other infections that require treatment.
• Abnormal liver function: two consecutive examinations with an interval of ≥1 week suggest that ALT and AST are 2.5 times higher than the upper limit of normal values
• Renal impairment: creatinine clearance <60ml/min
• Any severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study, including clinically significant cardiac diseases, refractory hypertension, metabolic disorders and other diseases that seriously affect the function of the gastrointestinal tract.
• Had a history of any psychiatric diseases, cerebrovascular disease or cognitive sequelae of head injury.
• Received rituximab in 6 weeks before enrollment.
• Received attenuated vaccine 4 in weeks before enrollment
• Participation in another clinical trial within 4 weeks before the start of this trial
• Have an allergy to Linperlisib or any other part of this medicine.
• Previously treated with other PI3Kδ inhibitor.
• Pregnant or breast-feeding patients
• Patients considered to be ineligible for the study by the investigator for reasons other than the above

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

YL-Pharma

UNKNOWN

Sponsor Role: collaborator

Institute of Hematology & Blood Diseases Hospital, China

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Zhoukou Central Hospital

Zhoukou, Henan, China

Regenerative Medicine Center

Tianjin, Tianjin Municipality, China

Countries

China

Other Identifiers

IIT2022067

Identifier Type: -

Identifier Source: org_study_id