The Safety and Efficacy of Zanubrutinib in Refractory/Relapsed Autoimmune Hemolytic Anemia

• Status: Recruiting
• Trial end date: 2025-12-31
• Target enrollment: 0
• Participant gender: All

Summary

The sample size of this study is calculated based on Simon's two-stage design. The first stage of the study enrolled a cohort of 12 patients. If after 12 weeks at least 6 patients achieved a response, then enrollment was expanded to a total of 26 patients. The null hypothesis was unaccepted if more than 14 of 26 patients achieved the response. Accounting for a 20% dropout rate, the estimated final sample size was 33 patients.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Institute of Hematology & Blood Diseases Hospital, China
• Treatments: Zanubrutinib

Criteria

Inclusion Criteria:

• Age from 6 to 70

• Diagnosis of Coombs-negative AIHA

• Diagnosis of warm AIHA, mixed AIHA or Evans syndrome.

• Meets the criteria of relapsed / refractory AIHA

• ECOG ≤ 3

• Willing and able to comply with the requirements for this study and written informed consent.

Exclusion Criteria:

• Diagnosis of any of the following diseases: Cold agglutinin disease, cold agglutinin syndrome, mixed AIHA, paroxysmal cold hemoglobinuria (PCH).

• Diagnosis of active stage of connective tissue disease.

• History of lymphoproliferative tumors or any other malignant.

• Diagnosis of other inherited or acquired hemolytic diseases.

• Secondary AIHA caused by drugs or infection.

• Previously received organ or stem cell transplantation.

• History of thrombosis or organ infarction.

• Received rituximab within 8 weeks before enrollment.

• Previously treated with BTK inhibitor ≥ 2 weeks.

• Received low-molecular-weight heparin or warfarin within 1 week before enrollment and need to continue the drug treatment.

• Received CYP3A4 Enzyme Inhibitors or Inducers within 1 week before enrollment and need to continue the drug treatment.

• Uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics, antiviral therapy, and/or other treatment), known human immunodeficiency virus (HIV), known evidence of active infectious hepatitis B, and/or known evidence of active hepatitis C.

• Any severe and/or uncontrolled medical conditions: refractory hypertension, clinically significant cardiac diseases, renal diseases, liver diseases and metabolic diseases, etc.

• History of mental illness.

• Participation in another clinical trial within 4 weeks before the start of this trial.

• Pregnant or breast-feeding patients.

• Patients considered ineligible for the study by the investigator for reasons other than the above.