Safety and Efficacy Study of PI3K Delta Inhibitor in Autoimmune Hemolytic Anemia Patients Who Failed Two-round Therapy

• Status: Not yet recruiting
• Trial end date: 2024-12-31
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Institute of Hematology & Blood Diseases Hospital
• Collaborator: YL-Pharma

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