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

NCT05676697 | Terminated Phase 1

• 1 other identifier: IIT2022067
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 2

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

Outcome Measures

Primary Outcomes (1)

• Overall response rate

  Percentage of patients with hematological response. Hematological response is evaluated by hemoglobin and other hemolysis-related laboratory indicators.

  6-12 weeks

Secondary Outcomes (7)

• Incidence of the adverse event

  12 weeks

• Complete response rate

  6-12 weeks

• Complete response with incomplete hemolysis recovery, CRi

  6-12 weeks

• Mean change from baseline in hemoglobin (Hgb) levels

  6-12 weeks

• Time to achieve partial response (PR)

  6-12 weeks

Study Arms (1)

PI3K Delta Inhibitor

EXPERIMENTAL

Drug: Linperlisib

Interventions

Linperlisib 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.

PI3K Delta Inhibitor

Eligibility Criteria

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

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead
• YL-Pharma: collaborator

Study Sites (2)

Zhoukou Central Hospital

Zhoukou, Henan, China

Location

Regenerative Medicine Center

Tianjin, Tianjin Municipality, China

Location

MeSH Terms

Conditions

Anemia, Hemolytic, Autoimmune

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Autoimmune Diseases; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 7, 2022
• First Posted: January 9, 2023
• Study Start: January 13, 2023
• Primary Completion: January 31, 2024
• Study Completion: February 6, 2024
• Last Updated: February 11, 2026

Record last verified: 2024-02

Data Sharing

• IPD Sharing: Will not share

Locations

CN (2)