Refractory/Relapsed Autoimmune Hemolytic Anemia
Conditions
Brief 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.
Interventions
DRUGZanubrutinib
Zanubrutinib was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity.
Sponsors
Institute of Hematology & Blood Diseases Hospital, China
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to 70 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Overall response rate | within 12 weeks | The percentage of patients achieved complete response, complete response with incomplete hemolysis recovery, and partial response. |
Secondary
| Measure | Time frame |
|---|---|
| Incidence of adverse events and severe adverse events | within 24 weeks |
| Relapse free survival rate | within 48 weeks |
Countries
China
Contacts
Primary ContactWeiwang Li, PhD
Backup ContactJingyu Zhao, MPH
Outcome results
None listed