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

Institute of Hematology & Blood Diseases Hospital, China

Status and phase

Enrolling

Phase 2

Conditions

Refractory/Relapsed Autoimmune Hemolytic Anemia

Treatments

Drug: Zanubrutinib

Study type

Interventional

Funder types

Other

Identifiers

NCT06021977

IHBDH-IIT20230711

Details and patient eligibility

About

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.

Enrollment

33 estimated patients

Sex

All

Ages

6 to 70 years old

Volunteers

No Healthy Volunteers

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.